All posts by medical

The Neuroscience of Choice – Newsmax

December 22, 2020 medical

Misplacing your keys, forgetting someone's name at a party, or coming home from the market without the most important item these are just some of the many common memory slips we all experience from time to time.

The international bestseller that provides pioneering brain-enhancement strategies, memory exercises, a healthy brain diet, and stress reduction tps for enhancing cognitive function and halting memory loss.

Dr. Gary Small,author of The Mind Health Report newsletter, is a professor of psychiatry and aging and director of the UCLA Longevity Center at the Semel Institute for Neuroscience and Human Behavior. Dr. Small, one the nations top brain health experts, frequently appears on The Today Show, Good Morning America, and The Dr. Oz Show. He is co-author with his wife Gigi Vorgan of many popular books, including The New York Times best-seller, The Memory Bible, and The Alzheimers Prevention Program.

Let's face it without a decent mind, you have no quality of life. With Dr. Gary Small's Mind Health Report, you'll gain greater health, happiness, and fulfillment in your relationships, personal life, work life or retirement, and more. Dr. Small fills every issue with the latest advancements in brain research from the far-reaching frontiers of neuroscience and psychiatry. You'll not only read about breakthrough techniques for rejuvenating your brain health, but also see actual case studies from Dr. Small, one of the nation's leading brain and aging experts and director of the UCLA Longevity Center.

Each month, you'll embark on a new journey into the world of your brain. You'll discover the latest on topics such as Alzheimer's disease and memory loss, anxiety and depression, diet advice for a healthy brain, natural supplements and drugs that aid mental functioning and lessen pain and fatigue, and much more.

More:
The Neuroscience of Choice - Newsmax

Neuroscience

Drug helps mice with autism-linked mutation recognize others – Spectrum

December 22, 2020 medical

Ion flow: A protein (red) that ferries potassium out of nerve cells abounds in a small part of the brains memory hub linked to social behavior.

Courtesy of Macayla Donegan

A drug that has been tested in clinical trials as a treatment for depression restores social memory in a mouse model of 22q11.2 deletion syndrome, according to a new study. The findings hint that the drug might also be useful to treat social cognitive difficulties in people with conditions such as autism, experts say.

People who are missing one copy of a chromosomal region known as 22q11.2 have heart abnormalities, distinctive facial features and an increased risk of schizophrenia and other psychiatric conditions. About 16 percent have autism. People with the syndrome also have a smaller-than-average hippocampus, a structure that functions as the brains memory hub.

The findings extend what researchers know about the role of the hippocampus in social behavior by suggesting that a small region of the hippocampus known as CA2 springs to life when an animal encounters an individual it hasnt met before.

A strength of the study is that it describes the basic biology of a brain circuit, shows how that circuit is disrupted in a mouse model and identifies a therapeutic target to reverse those disruptions, says Anthony LaMantia, professor of developmental disorders and genetics at Virginia Polytechnic Institute and State University in Blacksburg, who was not involved in the work. This is one of the best papers sort of going from soup to nuts that has come out.

Previous studies showed that CA2 is crucial for social memory, the ability to recognize and remember others. But we really didnt have a good handle on what type of information CA2 was providing to the rest of the brain, says study leader Steven Siegelbaum, professor of neuroscience and pharmacology at Columbia University.

Siegelbaums team recorded the electrical activity of CA2 neurons in six wildtype mice, as well as that of neurons in the neighboring CA1 region in three of the animals.

Most of the cells in CA2 show little activity as a mouse explores its cage or objects placed in it, the team found. But the cells become more active when another mouse is present. And some of the cells are even more active when that mouse is a stranger. The findings appeared in October in Nature Neuroscience.

In order to remember an individual, you first have to recognize whether you know them or you dont, and we think that CA2 is initially providing this novelty signal, Siegelbaum says.

The team fed information about the activity of CA1 and CA2 neurons into a machine-learning algorithm. Based on CA2 activity, the algorithm could decode when a mouse was interacting with another mouse, and whether that mouse was a stranger. Based on activity in CA1 a region known for making mental maps the algorithm could identify a mouses location in the cage.

The algorithm is a clever approach, says Enrico Cherubini, professor of neuroscience at the International School for Advanced Studies in Trieste, Italy, who was not involved in the work. Mice move during social interactions, so it can be difficult to distinguish place-related neural activity from that related to social behavior.

Because mice navigate by sight and recognize each other largely by smell, whereas people depend on sight for both functions, it does raise the issue of whether or not you can really make the comparisons, LaMantia says.

The hippocampus is known to be able to integrate multiple sources of sensory information, Siegelbaum says.

The researchers also recorded the activity of CA2 neurons in five 22q11.2 mice. These mice are known to have deficits in social memory, although they are just as interested in socializing as wildtype mice are. The model mice also show a loss of a certain type of neuron in CA2, as do postmortem brains of people with schizophrenia.

The CA2 neurons in the 22q11.2 mice are less active than those in controls, regardless of what the mice are doing, the researchers found. The cells dont become as active when the mice interact with other mice, and they dont show the marked increase in activity when theres a new mouse around.

Instead, the cells activity tends to reflect where the mouse is in the cage similar to the typical behavior of CA1 neurons. The gain of function was surprising, Siegelbaum says.

The researchers had evidence that the abnormally sluggish firing of CA2 neurons in the 22q11.2 mice could be due to overactivity of TREK-1, a protein that aids the flow of potassium ions out of the cell. (Too much potassium ion outflow inhibits a neuron from firing.)

So they injected the mice with spadin, a drug that blocks TREK-1 activity. The treatment normalized the activity of the CA2 neurons. It also enabled the mice to remember other mice that they had met before.

The researchers also used a genetic technique to turn down the activity of TREK-1 in the CA2 region. This method, too, corrects social memory problems in the 22q11.2 mice.

Siegelbaum plans similar studies to investigate CA2 function in other mouse models with genetic alterations linked to autism, such as those affecting SHANK3 and CNTNAP2.

See more here:
Drug helps mice with autism-linked mutation recognize others - Spectrum

Neuroscience

A brain and behavior expert explains how to give better gifts – Business Insider – Business Insider

December 22, 2020 medical

Q: Do you have advice on how to find a good gift for a family member who already has everything and they don't need anything?

Giving gifts is easy if you pay attention throughout the year. It is very likely that the person you're giving gifts to, at some point, mentioned something they wanted/needed during the year. If you had listened and made a note you will have had the answer already.

So, as a first tip for the future, start a mini diary where you make a note every time someone says they want/need/miss something. Come Christmas you'll find that you have a lot of ideas in your diary. This not only makes things easier, it also highly impresses the recipient to know that you've been paying attention.

This is true, by the way, even if by now they actually already gotten the Bluetooth speaker they mentioned in July, or the silk bedding that came up in the conversation in March.

So now, let's assume you don't have this diary and have no inside information. What now?

For someone who has everything, there are a couple of answers. One quite popular these days is to gift a shared experience (i.e., a dinner you cook, a kayaking trip you go to together, or a spa day you arrange for the two of you).

Moran Cerf. Moran Cerf

Even if they had that experience before, they haven't had it with you. So the experience context is new, and assuming they like you is seen as a gift.

If you want to go for something material, and you believe the person really does have everything, or can afford to have everything they want, one thing you can do is aim for something in your budget that is the "top of a category they wouldn't buy themselves." John Ruhlin, author of "Giftology," has the example of buying an expensive "box" for a friend.

Read more: The lucrative lives of professional Christmas decorators, who charge as much as $80,000 to deck a house's halls and say business is booming this year

He suggests that if you were going to spend $50 on a gift, and the person really loves watches, for example, then buying them a $50 watch may not do the job compared to the ones a watch collector may own. But getting them a box for a watch that costs $50 may actually prove quite fancy.

So instead of getting a cheaper thing in category A, you get an expensive item in category B, a cheaper category. You get the top item in a different domain rather than a lower item in a domain you cannot afford. Instead of a fancy new game console, you can buy the best game for it; or instead of an expensive power tool, you get a really good level.

Thinking about the brain in that context, the way our brain resolves internal challenges is often not by identifying the optimal solution, but rather by reframing the problem. This is a key tool in emotion regulation and in therapy that allows our brain to overcome problems without always solving them.

Q: This year for Christmas, we're not to be there in person for the gift-giving, so I'm wondering: Is there any advice on how to make the people mostly kids, but not only who receive the gift associate it with me? (Sorry about the vanity, but I hate that my nieces don't know the gift came from me, after all the effort.)

One way to make a gift associated with you rather than, say, Santa is to put yourself in the gift. Here are a few ways.

If the gift has a visual component (i.e., a picture frame, or an external hard drive) you can add something of yours to it. If it's a picture frame you can put a picture of you and your nieces in it when you gift it. They may replace the picture, but the initial link is generated in their episodic memory. If it is a hard drive, you may leave a file that you want them to have: maybe a movie that you want them to watch, or a folder with some app they like.

Research in neuroscience shows that only a single association between an item and a person is needed to create a memory, IF it is the first association created for an experience meaning, if your nieces get a picture of you and them in the frame you will be registered with this frame even after the replaced your initial content.

Read more: Sleep-tracking ring Oura is beloved by some of the biggest names in tech. We asked 7 investors and execs how it's helped them revamp their routines to sleep better and live healthier.

And one last thought on the nieces' gift.

There are quite a few studies in brain development and memory, and the unfortunate bottom line is that it's likely that kids until the age of 2 will not really form explicit memories. They will form impressions (and events leave marks on them, and define trajectories of their personality development), but most likely won't remember much of the gift or your involvement in it at this stage.

So think of the gift as a way to shape their future self, and to start building a path together. And if you optimize for explicit memory, save some of the planned expenses for the next year or two, when they are more likely to code your role in the summoning of the Barbie Dollhouse.

Moran Cerf is a professor of neuroscience and business who explores how we can harness our understanding of the brain to improve our behavior, our business, and society. He's a former hacker, a science consultant to Hollywood films and TV shows, and the founder of a number of companies.

View original post here:
A brain and behavior expert explains how to give better gifts - Business Insider - Business Insider

Genetics

Correlation and Causation: What Can Genetics and Genomics Tell Us about COVID-19? – BioNews

December 22, 2020 medical

21 December 2020

Session three of the Progress Educational Trust (PET) annual conference explored the genetic and genomic links to susceptibility to severe COVID-19. Sarah Norcross, director at PET, opened the session with the unfortunate news that one of the speakers Dr Kri Stefnsson was unable to participate in the session due to illness.

The session was chaired by Dr Roger Highfield, science director at the Science Museum Group mild-mannered and a clear speaker, he chaired the session with ease. Dr Highfield introduced the first speaker Dr Sharon Moalem a scientist and physician who specialises in genetics. Dr Moalem is a bestselling author, with titles including: 'How Sex Works', 'Inheritance: How Our Genes Change Our Lives and Our Lives Change Our Genes', and 'The Better Half: On the Genetic Superiority of Women' (see BioNews 1050).

Dr Moalem focused his talk on the law of homogameity, and whether COVID-19 illustrates the genetic/genomic resiliency of women. He began by briefing the audience on the basics of mammalian genetics, how we have 46 chromosomes, one pair of which is sex chromosomes, containing either XX for a female, termed homogametic, or XY for a male, termed heterogametic. A system has arisen in females, where in each cell one X chromosomes is 'turned off', called X-inactivation or silencing. However, it has now been discovered that X-inactivation is not complete, and that about 25 percent of the second X chromosome is still active. Dr Moalem explained that this allows females to have more 'genetic horse power' within each of her cells.

X-linked conditions, such as fragile X, red-green colour blindness and Duchene muscular dystrophy, are more common in males, as men do not have another X chromosome. Females do not simply have a 'back up X', but in some cases their non-faulty gene produces and shares the required protein, essentially rescuing the cells containing the faulty gene, which would have died. In other cases, cells with such a mutation do not survive, but cell lines with the healthy X proliferate to compensate. This leads to tissues with an uneven distribution of active Xs, called X-skewing.

Moving onto COVID-19, why is the mortality rate for males higher than that for females? Dr Moalem proposed the law of homogameity, which predicts that the homogametic sex has a survival advantage across the life course. Females have a greater genetic diversity due to having an extra X-chromosome, which equates to 1000 more genes. The X chromosome contains many immune-related genes eg, TLR7 which is used by certain cells in the body to detect for single-stranded RNA viruses, like SARS-CoV-2, the virus which leads to COVID-19. As females have two variations of TLR7 they have two different immune cell populations to help detect the virus. However, there is a cost to homogameity increased autoimmunity, with 'long COVID' proving to be four times more common in women.

In the USA there is currently no requirement for drug approval from the FDA to use both male and female cells. Scientists can use just one sex, yet females' cells work in a corporative way. Dr Moalem believes there should be a completely separate drug approval process as many drugs behave differently in men and women.

Dr Highfield returned to introduce the second speaker Dr Qian Zhang a research associate at the St Giles Laboratory of Human Genetics of Infectious Diseases, at Rockefeller University in New York City. Dr Zhang's research specialises in inborn errors of immunity (IEIs) inherited disorders that impair normal immune development and function.

Dr Zhang focused her talk on type I interferon immunity in patients with life-threatening COVID-19 and began by explaining how early on in the pandemic it became clear that people infected with SARS-CoV-2 responded differently. Most were asymptomatic and developed either no or very mild clinical symptoms. A small proportion of patients developed life-threatening disease. This phenomenon is seen in all infectious diseases from bacteria, fungi and viruses.

Dr Zhang and her research team studied whether the same genetic mutations already known to be associated with life-threatening influenza infections also increase the risk of life-threatening COVID-19 pneumonia. There are three genes, TLR3, IRF7 and IRF9, in the type I interferon pathway that are mutated in people who develop life-threatening influenza. In addition, ten further genes, IFNAR1, IFNAR2, STAT1, STAT2, IRF3, UNC93B, TRIF, NEMO, TRAF3 and TBK1, are reported to affect severity of other viral infections.

Type I interferon is a cytokine and has 17 different subtypes, which lead to the stimulation of several hundred interferon-stimulated genes that have an antiviral function. Hence, if this pathway is disrupted by a genetic mutation, viruses are able to gain a foothold more easily.

Dr Zhang's team sequenced the whole of the genome of over 600 severe COVID-19 patients to determine whether they had mutations in any of these 13 genes and discovered over 118 variants, of which, 24 resulted in loss of function.

Four patients with autosomal recessive mutations causing a complete loss of function in IRF7 and IFNAR1had never been hospitalised before contracting COVID-19, much to Dr Zhang's surprise. IRF7 is a transcription factor that amplifies the antiviral signal of type I interferon, and IFNAR1 is one of two proteins that make the receptor for type I interferons. Patients with these mutations are unable to mount an interferon response to COVID-19 infection.

Dr Zhang reassuringly explained that these mutations are rare, less than one in 1000 in the population, as such they cannot explain why there are so many people dying of COVID-19. This led Dr Zhang to the second part of her research: studying whether auto-antibodies against type I interferons lead to the same phenotype as these rare mutations.

Over ten percent of patients with life-threatening COVID-19 make auto-antibodies against two of the type I interferons. These neutralising auto-antibodies can entirely block the protective effect of type I interferons, which may be the cause of severe COVID-19.

Surprisingly, 95 percent of patients with these auto-antibodies were male and only six were female. One of these female patients had incontinentia pigmenti (IP), which is caused by a NEMO mutation on the X chromosome, leading to skewed X-inactivation a perfect example of the genetic diseases Dr Moalem was discussing earlier in the session. Even though such patients have two X chromosomes, most of the tissues in their bodies express just one X chromosome, and so these females are more similar to males in terms of susceptibility.

Combining both parts of her research, Dr Zhang's team discovered that selected patients with TLR3 and IRF7 mutations could simply be treated with type I interferon. However, this treatment did not work for patients with IFNAR1 mutations because the receptor is absent, but treatment with wildtype IFNAR1 was successful. Unfortunately, neither treatment worked for patients with auto-antibodies.

In her opinion COVID-19 could be considered an X-linked disease, even though the candidate on the X-chromosome has yet to be discovered and furthermore, type I interferon immunity is essential to control COVID-19 infection.

Dr Highfield returned to update the audience on Dr Stefnsson's research, investigating the genetic code of each COVID-19 infection in Iceland, giving an insight into the origins, and how the infection was caught, spread and mutated. Surprisingly, a large number of cases came from the UK. Similar research in the UK has detected 1356 independent introductions of the virus, mostly due to inbound international travel a third came from Spain, over a quarter from France and 14 percent from Italy.

The session provoked interesting discussions within the Q&As, with the first asking Dr Moalem whether females are less severely affected by other viruses, which he concluded as true, particularly for influenza. But even for HIV-1, women are much better at clearing the virus and have a much lower viral load.

With a personal interest, I took the opportunity to ask Dr Zhang whether patients with type I interferon IEIs were more susceptible to severe COVID-19, as such a disease affects members of my own family. Dr Zhang confirmed that such patients are more likely to suffer with severe COVID-19 and had now tested 20 IP patients discovering at least a quarter had high levels of autoantibodies to type I interferons. She warned that these females should be very cautious and shield as much as possible, as any patient with auto-antibodies is very difficult to treat. Reassuringly, patients with certain genetic mutations that do not have high levels of autoantibodies to type I interferon can simply be treated early with interferon injections.

I will leave with a final comment from Dr Moalem: 'Men are more biologically fragile when compared to women.' Whoever said that women were the weaker sex?

PET would like to thank the sponsor of this session, the Anne McLaren Memorial Trust Fund, and the other sponsors of its conference - the Edwards and Steptoe Research Trust Fund, ESHRE, Wellcome, the European Sperm Bank, Ferring Pharmaceuticals, the London Women's Clinic, Merck, Theramex, Vitrolife and the Institute of Medical Ethics.

Continue reading here:
Correlation and Causation: What Can Genetics and Genomics Tell Us about COVID-19? - BioNews

Genetics

Bryan Sykes obituary – The Guardian

December 22, 2020 medical

The human geneticist Bryan Sykes, who has died aged 73, pushed forward the analysis of inherited conditions such as brittle bone disease and double-jointedness, and was one of the first to extract DNA from ancient bone.

The same Bryan Sykes, holder of a personal chair at Oxford University, analysed hair supposedly taken from mythical hominids such as the Bigfoot and Yeti, and announced the results in a three-part television series. His delight in science and enthusiasm for communicating it to popular audiences were both aspects of an expansive personality that alternately inspired and exasperated his colleagues.

Sykes was not the only one to realise that the ability to read sequences of DNA code opened up the possibility of tracing human ancestry to our early origins. He was exceptional, however, in seeing that the wider public would connect emotionally to these stories if the dry details of the science could be presented accessibly. His book The Seven Daughters of Eve (2001) proposed that every living European could trace his or her ancestry to one of seven women living between 8,500 and 45,000 years ago. They, in turn, would share descent from a single Eve, who lived in Africa even earlier. He gave the seven women names and, anticipating peoples desire to know which tribe they belonged to, the same year set up the first direct-to-consumer genetic testing company, Oxford Ancestors, as an Oxford University spinout.

Sykes began this work long before modern methods of whole-genome DNA sequencing were available. When, in the late 1980s, he, Erica Hagelberg and Robert Hedges of Oxfords Research Laboratory for Archaeology first extracted DNA from bones up to 12,000 years old, they opted to focus on mitochondrial DNA (mtDNA). There are more than 1,000 mitochondria in each cell but only one nucleus (where most of our DNA resides), increasing the chances of retrieving mtDNA. But Sykes soon appreciated that it has another property. It is inherited largely unchanged in the maternal line over thousands of years, while nuclear DNA is mixed with every generation. To test whether it would be possible to use mtDNA to trace distant ancestors, Sykes first confirmed that domesticated golden hamsters from numerous locations, which he had heard were all descended from a single wild-caught female, had the same signature in their mtDNA.

Sykes went on to use this method to solve the mystery of the origins of islanders scattered throughout the Pacific Ocean: whether they had arrived from the Americas, as Thor Heyerdahl had suggested on the basis of the 1947 voyage of the Kon-Tiki raft, or from Asia. Receiving hospital treatment on Raratonga in the Cook Islands after a motorcycle accident while on holiday in the mid-90s, Sykes realised he could resolve this uncertainty using mtDNA. He went on to collect samples from Pacific islands and Pacific Rim countries, and established that Polynesia was in fact entirely settled from Asia.

In 1987 he won a British Association for the Advancement of Science media fellowship that enabled him to spend seven weeks working with Channel 4 News. The lessons he learned about what makes a good story came to the fore in Seven Daughters and his subsequent books.

Adams Curse (2003) drew some controversial conclusions about the influence of the Y chromosome on male behaviour, but also covered studies that traced descent via Y chromosomes. These pass from father to son, like British surnames, though without the uncertainty introduced by nonpaternity events. When the chairman of the pharmaceutical company GlaxoSmithKline, Sir Richard Sykes, wondered if the two of them might be related, Bryan collected DNA from dozens of Sykeses in Britain. He discovered that more than half of them shared the same unusual Y chromosome variant, suggesting a single founding father in Yorkshire in the 13th or 14th century.

His collaboration with enthusiasts searching for the Bigfoot and Yeti raised eyebrows even higher. Hairs from bits of mystery creatures that had long lain in museums and temples made their way to his lab. The three-part Channel 4 series Bigfoot Files (2015) maintained the suspense to the end, but all the samples proved to come from known animal species. A hasty claim that a Yeti specimen was a match to a prehistoric polar bear proved to be a case of mistaken identity. For Sykes it was all education as entertainment he never seriously believed that such creatures existed, but sought to encourage curiosity rather than squashing it.

Born in London, Bryan was the son of Frank Sykes, an accountant, and his wife, Irene. He attended the independent boys school Eltham college, near his home in south-east London, and developed passions for the natural world, experiments and inventions. He also excelled at cross-country running, rugby and swimming.

He studied biochemistry at the University of Liverpool, and did a PhD at the University of Bristol on the connective tissue protein elastin. He arrived at Oxford in 1973 as a research fellow in the Nuffield department of orthopaedic surgery, continuing to work on elastin and collagen. By the time he was appointed lecturer in molecular pathology in 1987, he was deploying new genetic techniques to explore inherited disorders of bone and connective tissue. His collagen genetics group moved from orthopaedic surgery to Oxfords newly established Institute of Molecular Medicine, founded by the geneticist Sir David Weatherall, who was an important mentor. He was appointed to a personal chair in human genetics in 1997, and formally retired in 2016.

Sykess expertise in bone led to his involvement in the effort to extract DNA from ancient specimens. As his interest in studies of human populations developed, he recruited lab members who worked in that area alongside those who continued his pathological studies. Colleagues remember the lab as being unusually collaborative, though occasionally disrupted by TV cameras, and Sykes himself as encouraging and supportive. He took them all to Scotland in 1998 to assist with the collection of samples for his work on prehistoric migration into Britain (published as Blood of the Isles, 2006). A keen fisherman, he got out his rods in the bar of their hotel to teach them how to cast a fly.

Sykes was extremely smart and a brilliant communicator, with a streak of mischief: he didnt turn a hair when Italian colleagues casually invited him to access the bone store at Pompeii by climbing over a fence (they had arrived before opening time), and there was always champagne in the lab when anyone published a paper.

Sykes met Sue Foden when she was a student in Oxford, and they were married in 1978. Though the marriage was annulled in 1984, he and Sue remained close and had a son, Richard, born in 1991. His later marriage to Janis Wilson ended in divorce. In 2007 he collaborated with the Danish artist Ulla Plougmand on an exhibition featuring the seven daughters of Eve, and their subsequent relationship lasted until the end of his life. In later years, as his health deteriorated, Bryan was increasingly supported and cared for by Sue. She, Ulla and Richard survive him.

Bryan Clifford Sykes, geneticist, born 9 September 1947; died 10 December 2020

See the article here:
Bryan Sykes obituary - The Guardian

Genetics

British scientists find worrying changes in the coronavirus – Los Angeles Times

December 22, 2020 medical

In the United Kingdom and several other countries, a disconcerting cluster of genetic changes has been detected in the coronavirus that causes COVID-19, prompting British Prime Minister Boris Johnson to all but cancel Christmas in Britain.

At least 1,619 samples of the virus collected from infected Britons contained a distinctive set of 17 genetic alterations, including three that appear to make the virus easier to transmit from person to person and improve its ability to sneak past the immune systems defenses.

The changes in the coronaviruss RNA were detailed in a report by British researchers who use time-stamped genetic sequences of the virus to track the pandemics progress. The COVID-19 Genomics UK Consortium said the unexpectedly large number of changes, and their potential influence on key parts of the virus, require urgent laboratory characterisation and enhanced genomic surveillance worldwide.

The same collection of RNA alterations has been detected in viral samples culled from four other countries, the British geneticists added.

Newsletter

Get our free Coronavirus Today newsletter

Enter Email Address

Sign Me Up

You may occasionally receive promotional content from the Los Angeles Times.

Several of the changes appear to alter the virus spike protein, which the coronavirus uses to pick the lock to human cells and convert them into factories for its own production. Two of the changes appear to make cells in the respiratory tract and elsewhere more vulnerable to invasion, and they increased the virus infectivity and its virulence in mice.

While both mutations have been seen separately, theyve only been seen together in the new clutch of cases, the British researchers said.

Thats a potential problem because all five of the COVID-19 vaccines furthest along in development train the immune system to target the spike protein. In theory, these genetic changes might alter the protein enough to erode the effectiveness of vaccines made by Pfizer and Moderna both of which are being rolled out in the U.S. as well as three others close behind them.

In a report last week, the British researchers wrote that there is currently no evidence that this variant (or any other studied to date) has any impact on disease severity, or that it will render vaccines less effective, although both questions require further studies performed at pace.

Over the weekend, a panel of vaccine experts pressed the U.S. Centers for Disease Control and Prevention on whether the British strain would alter the effectiveness of the Moderna vaccine that received emergency use authorization from the Food and Drug Administration on Friday. Officials assured committee members that Moderna and other vaccine makers will perform deep sequencing on these so-called breakthrough cases to detect whether such changes have occurred.

The developments caused Johnson to renew lockdown measures across much of England, including travel restrictions and the shuttering of pubs, gyms, theaters and hair salons. He said the moves were taken in response to findings that the RNA changes could make the virus up to 70% more transmissible.

This is spreading very fast, the prime minister said.

By Sunday, France, Germany, Italy, the Netherlands, Belgium, Austria, Ireland and Bulgaria all said they would ban incoming flights from the U.K.

Samples of the SARS-CoV-2 virus that include some or all of 17 changes were first detected in samples from two Britons on Sept. 20. One was collected from a person in Kent in southeastern England, and the other came from a person in the London metropolitan area the next day.

By Friday, viral samples with roughly the same genetic fingerprint represented 6% of all viral samples sequenced in the United Kingdom since Nov. 1. (Researchers at the U.K. genetics consortium sequence roughly 10% of viral samples from all Britons who test positive for coronavirus infections.)

Emma Hodcroft, a geneticist working with a viral tracking group called Nextstrain, said the family of changes has also been detected in samples in Denmark and Australia, which were probably imported from Britain. Belgium and Italy are also following up on reports that the mutation has been detected within their borders.

Researchers in South Africa also have detected infections that bear some of the same changes, including ones in the virus spike protein.

Those who have followed the virus genetic journey during the pandemic were quick to note that not all alterations should set off alarm bells. As with any family tree, the virus spins off distinct lineages as it spreads across time and space, and each one is thought to pick up one or two changes a month.

As a result, the coronavirus has incorporated thousands of modifications since it emerged late last year.

While such incremental changes can gradually shift a virus behavior over time, none has yet been seen to suddenly make this coronavirus more dangerous to those it infects or to boost its ability to jump from person to person.

But this case may be different, the British researchers warned.

The abrupt appearance of so many samples with so many of the same potentially significant changes is, to date, unprecedented in the global virus genomic data for the COVID-19 pandemic, they wrote.

While scientists have been aware of some of these genetic variants since September, theyve circulated far more widely in recent weeks. The researchers hypothesized that the cluster of changes may have been prompted by the virus efforts to overcome the suppressive effects of antiviral drugs or antibody-rich blood plasma donated by people who have recovered from COVID-19.

The researchers noted that alterations tend to accumulate very quickly in people who take months to clear a viral infection, as well as in people with weakened immune systems. As their infections linger, those people become incubators for multiple versions of the virus that sicken them simultaneously, allowing them to swap and share their genetic variants.

Other scientists who have tracked the pandemics twists and turns said its not entirely clear that the genetic changes either singly or collectively have made the virus more transmissible.

The early assessments of transmissibility appear to be inferred in part from the growth of samples collected around Kent and London. But Dr. Marc Suchard, a UCLA biomathematician, cautioned that population density, different rates of mask-wearing and social distancing, and other factors can influence how fast a particular virus spreads.

One might say the evidence is suggestive of increased transmissibility, Suchard said. But nailing that down will require a careful scrubbing of epidemiological data and laboratory testing, he said.

Along the way, he added, scientists may discover that as the virus becomes better at spreading, it could also become less dangerous a tradeoff that has taken the bite out of other epidemics.

Dr. Arturo Casadevall, a Johns Hopkins University microbiologist, said that if the genetic changes will have any effect on COVID-19 vaccines, it will be minimal.

I am not alarmist about this, Casadevall said.

Even changes that significantly alter the virus spike protein dont affect plenty of other targets, called epitopes, that antibodies use to recognize and kill virus, he said.

So for the virus to completely defeat vaccine, it would have to change in many, many places where the virus binds to cells, he said. And that is a very low probability event.

That view is shared by Trevor Bedford, a geneticist at the Fred Hutchinson Cancer Research Center who has tracked the coronavirus genetic meanderings.

Im not concerned that these variants will significantly reduce vaccine efficacy in the 2021 rollout, Bedford said in a Twitter thread. The strong immune response to the mRNA vaccines would suggest that a large antigenic change would be needed to significantly reduce efficacy.

Cell Biology

Follow the Money: Spatial Omics, CAR-NK Cells, AI-Powered Biology – Bio-IT World

December 22, 2020 medical

December 21, 2020 | Funding updates around the life sciences including cash for a handheld mass spec device, Series D for oncolytic immunotherapy, a new company launch in allogenic CAR-NK cells, AI-powered cell classification, a 3-D sequencing platform, and more.

$170M: Israeli Digital Health First VC Firm

OTV (formerly Olive Tree Ventures), Israels digital health first venture capital firm, today announced the closing of a fund with a total value of $170M. OTV also announced their new name and the appointment of a new Head of Asia Pacific to spearhead the funds expansion into the regions market. OTV is the only venture capital fund in Israel whose primary focus is digital health, specializing in supporting their portfolio companies reach maturity, refine execution, tackle regulatory hurdles and ensure a global imprint on validated products. Over the course of the past five years, OTV has prioritized investment in digital health companies that develop cutting-edge solutions to todays most pressing healthcare problems. OTVs portfolio includes some of the worlds highest-profile digital health leaders, including TytoCare, Lemonaid Health, Emedgene, Scopio and Donisi Health.

$116M: Handheld, Desktop Mass Spec

908 Devices, which provides mass spectrometry devices for forensic and scientific research, raised the proposed deal size for its upcoming IPO. The Boston, MA-based company now plans to raise $116 million by offering 6.3 million shares at a price range of $18 to $19. The company had previously filed to offer the same number of shares at a range of $15 to $17. At the midpoint of the revised range, 908 Devices will raise 16% more in proceeds than previously anticipated. 908 Devices provides handheld and desktop mass spectrometry devices that are used to interrogate unknown and invisible materials, providing actionable answers to directly address critical problems in life sciences research, bioprocessing, industrial biotech, forensics and adjacent markets. Since its inception, the company has sold more than 1,200 handheld and desktop devices to over 300 customers in 32 countries, including 18 of the top 20 pharmaceutical companies by 2019 revenue, as well as numerous domestic and foreign government agencies and leading academic institutions.

$91M: Series C for Digital, Decentralized Trials

Medable has announced $91 million in Series C funding to accelerate the life sciences industrys shift to digital and decentralized clinical trials. The round was led by Sapphire Ventures, with follow-on investment from existing investors GSR Ventures, PPD, and Streamlined Ventures. The funding brings Medables total capital raised to more than $136 million. Medables flexible and modular software platform enables clinical leaders to shift from clinic-centric to patient-centric research strategies. The platform provides a unified experience for patients and clinicians, enabling recruitment, remote screening, electronic consent, clinical outcomes assessment (eCOA), eSource, telemedicine, and connected devices. Medable has seen rapid eCOA adoption, driving the field forward with enhancements including connected devices and telemedicine. The COVID-19 pandemic has driven demand for remote clinical trial technologies and Medable is enabling complex research protocols to be conducted remotely through its platform. By minimizing the need for in-person site visits, Medable customers have achieved unprecedented results including 3X faster enrollment and over 90% retention rates.

$58M: Series B for Healthcare Ecosystem Platform

H1, a global platform for the healthcare ecosystem, announced today that it has closed a $58 million Series B round of funding. The round was co-led by IVP and Menlo Ventures, which led the Series A round in April 2020. Transformation Capital, Lux Capital, Lead Edge Capital, Novartis dRx and YC also participated. H1 has created the largest healthcare platform to forge connections in the healthcare ecosystem. The H1 team has taken a unique approach to building the platform that combines AI, human powered engineering, third-party data sources, and government partnerships, to create the largest platform of healthcare professionals, currently spanning over 9 million healthcare professions around the globe.

$50M: Series C for Somatic Cancer, Wellness Platform

Congenica has announced the completion of its Series C funding round, raising $50 million. The round was co-led by Tencent and Legal & General and included other new investors Xeraya, Puhua Capital and IDO Investments. Existing investors Parkwalk, Cambridge Innovation Capital and Downing also participated. The funding is aimed at accelerating international market development and driving further expansion of Congenicas product platform into somatic cancer, wellness and through partnerships with pharmaceutical companies. Furthermore, the company will deliver capabilities including the ability to integrate with existing electronic health systems and deliver automated interpretation.

$47M: Series D for Novel Oncolytic Immunotherapies

CG Oncology has closed a $47 million Series D preferred stock financing led by new investor Kissei Pharmaceutical Co., Ltd., with participation from existing investors ORI Healthcare Fund, Camford Capital and Perseverance Capital Management. The financing will support the advancement of CG Oncologys late-stage clinical programs for its lead oncolytic immunotherapy, CG0070, including an ongoing global Phase 3 trial (BOND3) with CG0070 as a monotherapy for the treatment of BCG-unresponsive, Non-Muscle Invasive Bladder Cancer (NMIBC), and a combination Phase 2 study (CORE1) of CG0070 with KEYTRUDA (pembrolizumab) in the same indication. In addition, a Phase 1b study (CORE2) is currently ongoing with CG0070 in combination with OPDIVO (nivolumab) as a neoadjuvant immunotherapy for Muscle-Invasive Bladder Cancer (MIBC) in cisplatin-ineligible patients.

$42M: Series A for Allogeneic CAR-NK Cell Therapies

Catamaran Bio has launched with $42 million in financing. Sofinnova Partners and Lightstone Ventures co-led the Series A round that is part of the launch financing, with participation by founding investor SV Health Investors, as well as Takeda Ventures and Astellas Venture Management. Proceeds will be used to advance the companys two lead chimeric antigen receptor (CAR)-NK cell therapy programs. In addition, funding will expand the companys TAILWIND Platform, an integrated and proprietary suite of technologies for designing, genetically engineering, and manufacturing allogeneic CAR-NK cell therapies.

$34M: Series B for glycoproteomic powered ovarian cancer diagnostic

InterVenn Biosciences has raised $34M in a Series B fundraising. The latest round was led by Anzu Partners with full participation of Genoa Ventures, Amplify Partners, and True Ventures; Xeraya Capital and the Ojjeh Family joined the syndicate as well. Funds will be used to commercialize the companys High-Throughput-Glycoproteomic powered diagnostic for ovarian cancer; to service increasing partnership platform demand; and to accelerate development efforts for the immuno-oncology treatment response and colorectal cancer indications. InterVenn has demonstrated that analysis of protein glycosylation, the most common and most complex form of post-translational protein modification, is a highly effective way of discovering uniquely informative biomarkers. This breakthrough was made possible given the companys development of its AI neural network for high throughput analysis (PiP) and enabling software that powers the proprietary Vista research product for over a dozen different oncology indications.

$24M: Series A for Molecular Cartography

Resolve Biosciences has completed a $24 million Series A financing round and appointed Co-founder Jason T. Gammack as Chief Executive Officer. The Series A financing round was led by PS Capital Management and MasterMind Advisory Services and included participation from Alafi Capital, John Shoffner, and High Tech Grnderfonds. The company will use the proceeds to accelerate product development and drive industry adoption of its Molecular Cartography technology. The company's Molecular Cartography platform is a groundbreaking multi-analyte and highly multiplex spatial analysis technology that enables scientists to resolve the most daunting biological challenges in areas such as oncology, neuroscience, and infectious disease. It produces deep contextual datasets that illuminate molecular interactions at subcellular resolution, while preserving the sample tissue. The initial applications of Resolve's Molecular Cartography platform deliver the highest-resolution view of transcriptomic activity and provide the ability to interrogate hundreds of genes in a single run. Future solutions will add DNA, protein, and metabolomic data layers. Unlike current approaches, Resolve's technology provides the required sensitivity, specificity, and workflow convenience to elucidate the cell's complex transcriptional landscape.

$23M: Series A for Traumatic Brain Injury Test

BRAINBox Solutions announced the initial closing on a $23 million Series A financing to support the clinical development of the company's BRAINBox TBI (Traumatic Brain Injury) Test to aid both in the diagnosis and prognosis of mild TBI (concussion). BioVentures Investors led the financing round and was joined by the Tauber Foundation, the Virginia Tech Carilion Innovation and Seed Funds, Genoa VC, Pharmakon Holdings LLC, Astia Angels and additional qualified investors, including Kevin Love, professional basketball player and mental health advocate. BRAINBox TBI is the first test designed to assist in both the diagnosis and prognosis of concussion. The multi-marker and multi-modality test, which can be used in either point-of-care or clinical laboratory settings, includes a panel of blood biomarkers as well as advanced digital neurocognitive testing in partnership with BrainCheck, Inc. Using proprietary AI algorithms, BRAINBox TBI combines the results of the test components and patient reported outcomes to generate a single, objective score for diagnosis up to 96 hours from the time of injury and a prognosis report for likely injury-related symptoms up to three months post-event.

$23M: Series B for Digital, Computational Pathology Solutions

Proscia has secured $23 million in Series B funding led by Scale Venture Partners, with participation from Hitachi Ventures, the strategic corporate venture capital arm of Hitachi, Ltd., bringing its funding total to $35 million. The company will use the investment to accelerate its global growth and strengthen its position of leadership in transforming cancer research and diagnosis at a time when demand for modernizing pathology is higher than ever. With its Concentriq software platform, Proscia is accelerating the transformation to digital pathology, which centers around high-resolution images of tissue biopsies, as the new standard of care. Concentriq combines enterprise scalability with powerful AI applications to help laboratories, health systems, and life sciences companies unlock new insights, accelerate breakthroughs, and improve patient outcomes.

$21.5M: Series B for Software, Data Products for Biomarker Discovery

Ovation.io has raised $21.5 million in Series B funding led by SignalFire with participation from Madrona Venture Group, Borealis Ventures, StageDotO Ventures and industry veteran David Shaw. Ovations suite of software and data products make it easier for molecular diagnostic labs to bring innovative tests to the patients that need them. Developed by scientists to help laboratories accelerate adoption of molecular diagnostics, Ovation is a turn-key, cloud-based platform with configurable, out-of-the-box workflows for molecular tests and seamless integrations to support the needs of a rapidly growing lab. With this latest round of funding, Ovation will work with its network of participating labs to develop insights that can help life-science companies reduce the time and cost associated with biomarker discovery.

$20M: Series A for AI-Powered Cell Classification

Deepcell has closed its Series A round of financing with $20 million, led by Bow Capital and joined by Andreessen Horowitz, which led its $5 million seed round. The new funding will allow Deepcell to develop its microfluidics-based technology, continue building a cell morphology atlas of more than 400 million cells, and drive a hypothesis-free approach to cell classification and sorting. Spun out of Stanford University in 2017, Deepcell is using deep learning and big data to classify and isolate individual cells from a sample. The technology combines advances in AI, cell capture, and single-cell analysis to sort cells based on detailed visual features, delivering novel insights through an unprecedented view of cell biology. The Deepcell platform maintains cell viability for downstream single-cell analysis and can be used to isolate virtually any type of cell even those occurring at frequencies as low as one in a billion to offer access to rare cells and atypical cell states that will help advance precision medicine research.

$20M: Series B for Spatial Omics

Rebus Biosystems has closed a $20 million Series B financing round, led by Illumina Ventures and joined by Lifecore Partners, Ncore Ventures, Xolon Invest, CTK Investments, Ray Co., Ltd., Seegene Medical Foundation, LabGenomics Co., Ltd., and Timefolio Asset Management. At the heart of the Rebus Biosystems spatial omics solution is the companys patented Synthetic Aperture Optics (SAO) system, which provides the resolution and sensitivity of a 100X oil lens, but with the breadth and depth of a 20X air lens. Data is captured 100 times faster than with other imaging based spatial omics methods that rely on 100x lenses and z-stacking. Speed and ease of use of the system is further improved by integration with custom microfluidics and image processing.

$14.8M: Scaled Up Production of Portable PCR Diagnostic Device

QuantuMDx Group Limited, a UK-based life sciences company, is investing over 11 million to scale up production to mass manufacture its flagship diagnostic device, Q-POC and disposable test cassette. QuantuMDx accelerated development, scale-up and manufacture of Q-POCits rapid point-of-care testing systemearlier this year in response to the COVID-19 pandemic.Q-POC is a portable, PCR device offering rapid, sample-to-answer, molecular diagnostic testing at the point of care, with results in approximately 30 minutes. The Q-POC system comprises a sample collection kit, single-use test cassette and analyzer. The companys first commercial assay for Q-POC will detect SARS-CoV-2, the virus causing COVID- 19, and Flu A & B, providing a powerful rapid PCR diagnostics and surveillance tool for clinicians and public health officials.QuantuMDx has worked with British development partner Cambridge Design Partnership to undertake pilot manufacturing of Q-POCTM, and is now working with Cogent Technology, as the company scales for volume manufacturing.

$14M: Novel Treatment for Ischemic Stroke

BrainsGate has secured $14 million in a new investment round at a pre-money valuation of $147 million. New investor, BNP Joint Capital Fund, and the Impact investment and consulting firm SPERO led the round alongside existing investors, Elron, Medtronic, Agate, Pitango, and Cipio. The new investment is expected to fund BrainsGate through its pre-market approval (PMA) and enable it to achieve volume production readiness and apply for coverage from the Centers for Medicare & Medicaid Services (CMS) for its Ischemic Stroke System (ISS). BrainsGates therapy involves a less than 5 minutes procedure in which a neurostimulator implant is injected into an existing canal. The implant stimulates a nerve center that augments collateral blood flow to improve stroke patients outcomes in a 24-hour window. In May 2020, BrainsGate received marketing approval in Europe (CE marking) of its ISS product. BrainsGates PMA application was filed with the US FDA in February 2020. The company plans to initiate commercialization activities subject to the PMA being approved.

$10.6M: UK Investment Firm for Life Sciences

Intuitive Investments Group, a closed-end investment company focused on the life sciences sector, has raised 7.85 million (before expenses) in its AIM float by placing 39,250,000 new Ordinary Shares at 20p. The net proceeds of the Placing will be used by the Company to invest in fast growing and/or high potential Life Sciences businesses, based predominantly in the UK, wider Europe and the US, chosen from an identified pipeline of investment opportunities. Investments will be focused on diagnostics and healthcare, medical devices, tools and technologies and bio-therapeutics and pharmaceuticals

$7.4M: BARDA Extension For Point-of-Care Infection Diagnostics

Inflammatix has announced a contract extension of $7.4 million from the Biomedical Advanced Research and Development Authority (BARDA) to further develop its point-of-care test and system to diagnose infection by reading the immune system. The contract is part of a BARDA contract worth up to $72 million, if all options are exercised. The new funding will support continued development and commercialization of Inflammatixs sample-to-answer, point-of-care Myrna test system, which is designed to read RNA using machine learning and produce results in under 30 minutes, as well as continued development of the ViraBac EZ test (formerly known as HostDx Fever), which reads gene expression patterns in the immune system to identify whether a suspected infection is bacterial or viral, enabling physicians to quickly and accurately determine when to prescribe antibiotics. The test will use a fingerstick collection and capillary blood sample, and is designed for use in primary care, urgent care and other outpatient clinical settings.

$6.6M: Series A for Robotics, Machine Learning in Biology

Trailhead Biosystems announced its $6.6 million Series A financing. The company has developed a proprietary platform to perform systems-level interrogation of complex biological problems, ranging from the generation of industrially-scalable manufacturing conditions for specialized human cells to combinatorial drug discovery in cancer and anti-viral therapies. Trailhead robotically conducts the largest dimensioned experiments in biology, using machine learning to discover critical process parameters and combinatorial inputs that explain biological responses. Trailhead aims to rapidly develop the capability of creating specialized human cell types at high purity for regenerative medicine and therapeutic purposes at an industrial scale, addressing current industry needs for highly specialized cells used in drug discovery and modeling of human diseases. Through strategic partnerships, the cells provided by the company will be used to address multiple areas of clinical need. The company currently develops products that target type I diabetes, Parkinson's disease, Multiple Sclerosis, Alzheimer's disease, and blood disorders, among others.

$6.1M: 3-D Sequencing Platform

DNA sequencing instigator Single Technologies announced the completion of a heavily oversubscribed 5 million share issue to existing and new shareholders. Among the new investors are Jens von Bahr, Rothesay Ltd, Carl-Henric Svanberg funded Cygnus Montanus Trust, Professor Ulf Landegren and Andreas Ehn. The funds will be used to accelerate development of the companys 3-D sequencing platform. Stockholm Corporate Finance acted as financial advisor. The new funding will enable the company to finalize automating its 3-D sequencing process, make it more robust and improve quality for both Whole Genome Sequencing (WGS) and transcriptomics applications. The ambition is to open the first data sequencing production site in Stockholm by 2022.

$5M: Gates Grant for At-Home COVID-19 Test

Sherlock Biosciences has received a $5 million grant from the Bill & Melinda Gates Foundation to continue to advance INSPECTR, its instrument-free, synthetic biology-based molecular diagnostics platform. In addition to advancing the INSPECTR platform development to be as sensitive as gold-standard PCR tests, the funding will support the development of an over-the-counter disposable product, similar to an at-home pregnancy test, that can be used to detect SARS-CoV-2, the virus that causes COVID-19. Pending approval, the company says they will launch this product in mid-2021. INSPECTR, which stands for Internal Splint-Pairing Expression Cassette Translation Reaction, uses synthetic biology to enable the creation of instrument-free diagnostic tests that can be conducted at home, at room temperature. INSPECTR can be adapted to work on a simple paper strip test or to provide an electrochemical readout that can be read with a mobile phone. It can also be adapted for use in laboratory or point-of-care settings.

$4M: Prostate Health Center at Mount Sinai

Mount Sinai has received a $4 million donation from Lizzie and Jonathan Tisch to support prostate health and the Milton and Carroll Petrie Department of Urology at Mount Sinai. The prostate program will be named The Lizzie and Jonathan Tisch Center for Prostate Health. The medical services provided at the Lizzie and Jonathan Tisch Center for Prostate Center include state-of-the-art prostate cancer screening and imaging technologies, precision urology, focal therapy, targeted biopsies, robotics for prostate cancer surgery, prostate cancer fusion biopsy, and active surveillance. Additionally, prostate cancer experts such as Dr. Tewari; Avinash Reddy, MD; Sujit Nair, PhD; Robert Valenzuela; MD; Michael Palese, MD; and Steven Kaplan, MD, are available to see patients at the newly named Center.

$2.9M: NIH Grant for Human Placental Stem Cells

Human placental stem cells may have the potential to regenerate heart tissue after a heart attack, according to Mount Sinai researchers who have received a $2.9 million grant from the National Institutes of Health to study them. Their findings could lead to new therapies for repairing the heart and other organs. Hina W. Chaudhry, MD, Director of Cardiovascular Regenerative Medicine at the Icahn School of Medicine at Mount Sinai, is the Principal Investigator for this four-year award. Dr. Chaudhry and a team of investigators previously discovered that mouse placental stem cells can help the hearts of mice recover from injury that could otherwise lead to heart failure. They identified a specific type of placental stem cells, called Cdx2 cells, as the most effective in making heart cells regenerate.

$2M: Cryo Solutions for Cell, Gene Therapy

GlycoNet has secured a $2-million USD equity investment for PanTHERA CryoSolutions (PanTHERA), a Canadian biotechnology start-up. The investment came from US-based investor Casdin Capital and bioproduction tools supplier BioLife Solutions Inc. In addition to an up-front investment, subject to closing conditions, BioLife will provide an additional $2 million to support product development over the next 24 months in exchange for exclusive, worldwide marketing and distribution rights to the technology for use in cell and gene therapy applications. The core technology from PanTHERA was created out of an academic research collaboration between the University of Ottawa and the University of Alberta. During cryopreservation of biological materials, the uncontrolled growth of ice causes cell injury and death. PanTHERA's solution is to develop ice recrystallization inhibitors (IRIs) to control the growth of ice and prevent this damage from occurring, ultimately resulting in superior cellular products after thawing.

Read the original here:
Follow the Money: Spatial Omics, CAR-NK Cells, AI-Powered Biology - Bio-IT World

Cell Biology

Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) – GlobeNewswire

December 22, 2020 medical

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

Excerpt from:
Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire

Cell Biology

Better Than MDA: PTA Whole Genome Amplification Has More Info, Fewer Errors – BioSpace

December 22, 2020 medical

BioSkryb has developed a more robust technology for whole genome sequencing that accurately and uniformly captures more than 95% of the genomes of single cells.

Dubbed primary template-directed amplification (PTA), this is the foundational technology behind BioSkrybs ResolveDNA kits.

PTA lets researchers see genetic changes that other WGA technologies lose, Chuck Gawad, M.D., Ph.D., co-founder and director of BioSkryb, told BioSpace.

Its all based around resolution, so ResolveDNA is analogous to providing a better microscope but for the genome, said co-founder and CEO Jay West, Ph.D.

Kits contain the core technology (including proprietary nucleotides) and a product solution (the enzymes and reagents) to help researchers amplify the genome of samples as small as a single cell to prepare and analyze the data, looking at the whole genome of a species. Cell barcodes also can be attached to the amplification products for pooling and downstream analysis.

The companys initial focus is around oncology.

Theres a lot of the genetic diversity in tumors that isnt captured by standard sequencing, so researchers havent seen enormous utility from sequencing, Gawad said.

He began tackling the problem as a post-doc at Stanford University and continued the work in his own lab at St. Jude Childrens Research Hospital, where he finally teased out a solution. It was a simple idea, but became quite powerful.

As Gawad and colleagues explained in a recent research paper, Accurate Genomic Variant Detection in Single Cells with Primary Template-Directed Amplification, PTA takes advantage of the processivity, strong strand displacement activity, and low-error rate of phi29 polymerase used in (multiple displacement amplification) MDA. Howeverexonuclease-resistant terminators are incorporated into the reaction, creating small double-stranded amplification products that undergo limited subsequent amplification. This transforms the reaction from exponential into a quasilinear process, with more of the amplification occurring from the primary template.

This approach allowed DNA proportions to be maintained after amplification and the genome to be copied evenly, thus reducing artifacts, Gawad said. Were capturing more information from the genome, with fewer errors.

Importantly, PTA technology can be dropped into labs without affecting workflow. It takes about an hour of hands-on time and requires only a few steps, he added.

Analysis is performed on Trailblazer, BioSkrybs cloud-based bioinformatics platform.

Before BioSkryb launched the ResolveDNA and Trailblazer technology, the company took it to key opinion leaders and major laboratories that are well-versed in WGA.

They were overwhelmingly positive, Gawad said. When comparing PTA results to their prior WGA data, They found that a lot of artefacts in the data e disappeared and gaps were filled in, so they have a new level of detail, which has led to novel insights.

ResolveDNA is commercialized, but the company is still in its innovation phase, West said. Were releasing products for research use only. We have a long-term mission to improve patient outcomes, but first we have to move into the market and have clinical validation of the product.

Eventually, West envisions clinical applications, such as enabling physicians to select therapies based on the genomics of tumor cells.

Cancer is a tough disease, West said. Some tumor cells dont respond to a given therapy. They evade and repopulate, causing a relapse. When that happens, the genetic composition of the tumor has changed. Thats hard to assess using standard technology.

PTA may allow those changes to be identified and annotated for better-focused therapies.

BioSkryb developed DMEM, a tool to map genome-wide interactions of mutagens, as part of its mission to develop new applications for WGA. As Gawad explained, DMEM lets researchers measure the magnitude, genomic locations and nucleotide context of ENU-induced mutations in living human cells. It offers base pair resolution for living single cells, making it valuable in measuring off-target actions for drug candidates.

This is a marked contrast to the widely-used Ames test. That test, developed in the 1970s, tests chemicals against a particular strain of Salmonella typhimurium to determine toxicity.

How that is related to human genetics is unknown, but it probably only partially captures the effects of mutagens in human cells, Gawad said.

As West said, We are passionate about translational science asking biological questions that have real clinical impact. Single-cell genomics applications include the ability to perform single-cell evaluations of biopsies, including:

Other applications include:

To expand applications, BioSkryb recently announced a grant program to fund two researchers up to $20,000 each to demonstrate that their research can fuel new discoveries in single-cell genomics and help improve human health. Applications will be accepted through January 29, 2021 and awarded March 1 at The Advances in Genome Biology and Technology (AGBT) 2021 General Meeting.

At BioSkryb, West continued, Were focused on supporting the research market broadly. ResolveDNA can be used for high-throughput, parallel reactions in microfluidic devices or emulsions. To that end, the company is working to increase throughput beyond its current 24 & 96-reaction/cell format. It also may combine transcriptomic amplification and analysis to its genomic model, allowing researchers to gain insights into the relationships among cell state, genotype, and cellular phenotype.

Follow this link:
Better Than MDA: PTA Whole Genome Amplification Has More Info, Fewer Errors - BioSpace

Cell Biology

Top 15 Biotech Companies Shaking up the Scene in… – Labiotech.eu

December 22, 2020 medical

Cambridge has a large biotech scene that is starting to rival that of its US namesake. Here are the top 15 biotech companies in Cambridge you should know about.

Cambridge is a small but vibrant city that is well known for its university and for being a center of excellence for life sciences and health-related research. The Wellcome Sanger Institute, where part of the human genome was sequenced, is on its outskirts, next to the European Bioinformatics Institute. There are also a number of world-class research institutes in the vicinity including Addenbrookes Hospital.

While the city has historically had more of an academic research focus, in recent years it has become a major biotech and pharma hub. Antibody development is one of the strengths of the city thanks to the legacy of Cambridge Antibody Technologies, which pioneered the technology of phage display and developed Humira, the worlds best-selling drug. Newer therapeutic areas are also represented, such as RNA silencing, cell and gene therapies, and the microbiome, as well as companies using artificial intelligence to improve drug discovery.

As there are so many excellent companies in the region, it was hard to choose only 15. After consultation with local experts, here are our choices in no particular order.

Founded: 2010

Kymab is one of several Cambridge companies focusing on antibody therapies, such as F-Star Therapeutics or Crescendo Biologics. The company was spun out of the Wellcome Sanger Institute to rapidly develop bispecific antibodies using genetically engineered mice.

Kymab has eight products in its pipeline targeting cancer, hemophilia, and several immune disorders. Its most advanced candidate is a treatment for atopic dermatitis that recently reported successful results from a phase II trial.

The company has raised almost 200M to date. It has an ongoing patent dispute with US biotech Regeneron; in June, the US patent office rejected a fifth request by Regeneron to invalidate Kymabs US patents and the UK Supreme Court ruled in favor of Kymab.

Founded: 2009

Founded by Nobel prize winner and Cambridge professor Sir Greg Winter, Bicycle Therapeutics develops a new class of drugs called bicyclic peptides, which blend the ability of traditional antibodies to hunt normally undruggable proteins with the manufacturing ease of small molecule drugs.

Its technology caught the eye of AstraZeneca, which struck a 1B deal to tackle respiratory, cardiovascular, and metabolic diseases in 2016. Two years later, the companys first peptide began clinical trials to treat solid tumors that express high levels of a protein called metalloproteinase.

In 2019, the company listed on the Nasdaq stock exchange in a downsized IPO of 50M. This year the company made a deal with Genentech worth up to 1.6B to develop novel cancer immunotherapies.

Founded: 2011

Mission Therapeutics is developing small molecule drugs to inhibit deubiquitylating enzymes, which regulate the degradation of proteins within the cells. These enzymes are involved in a wide range of conditions including mitochondrial diseases, kidney injury, pulmonary fibrosis, and Parkinsons disease.

Mission Therapeutics raised 75M in one of the biggest rounds of European biotech back in 2016. This year, it raised an additional 12.4M and struck a drug development deal with Pfizer. The company currently has four programs in its pipeline, four of them in preclinical development.

Founded: 2013

Another spin-off from the Wellcome Sanger Institute, Congenica uses genomic analysis to assist in the diagnosis of rare diseases. The company has partnerships with many UK hospitals for genomic diagnostics and works with UCB in Belgium in drug discovery programs.

Congenica has developed a clinical genome analysis platform called Sapientia that uses machine learning to produce diagnostic reports from genomic data collected in hospitals and other research institutions around the world.

Last year, the company raised funds to support the use of its rare disease diagnostics platform by the newly formed NHS Genomic Medicine Service. This was topped up with a 41M Series C last month to drive the companys international expansion.

Founded: 2016

Artios Pharma is developing drugs that inhibit an enzyme called DNA polymerase theta, which plays a role in multiple DNA repair processes. The levels of this enzyme are typically higher in several types of tumors, including breast, ovarian and lung cancers, than in healthy tissue.

The company had an oversubscribed 73M Series B round in 2018. Earlier this month, the company signed a 5.7B megadeal with Merck KGaA to develop DNA damage repair cancer therapeutics. Its first clinical trial in humans is planned for 2021.

Founded: 2014

Healx uses artificial intelligence to analyze extensive medical research data with the goal of repurposing existing drugs to treat rare diseases. The companys most advanced program, a drug repurposed for fragile X syndrome, took less than two years to reach clinical trials instead of the normal five-to-seven years this process usually takes.

The company raised a 50.8M Series B round in October 2019. Shortly after, Healx started the Rare Treatment Accelerator program, allowing patient advocacy groups a chance to work with the company to find new treatments for rare diseases.

Healx has a mission to advance 100 rare disease treatments towards the clinic by 2025. Since the start of the pandemic, the company has been using its technology to search for treatments for Covid-19.

Founded: 2019

Cyted is applying artificial intelligence to the development of diagnostics. The company is collaborating with Medtronic to develop an early detection esophageal cancer test. Medtronics technology is based on a device that can be swallowed like a pill, invented by one of Cyted founders before starting the company.

Cyted raised 9.6M in February this year and acquired Pathognomics, a provider of digital pathology and clinical diagnostic laboratory services that will allow the company to scale up and roll out its diagnostic services.

Founded: 2015

Storm Therapeutics was spun out of the University of Cambridge to treat cancer and other diseases using a technology to modulate RNA-modifying enzymes, such as RNA methyltransferase.

The company raised 34M in Series A funding in 2019. Its most advanced drug candidate is now ready to enter clinical trials as a treatment for leukemia.

There has been increasing interest in companies working in the RNA space since the first RNA interference therapy was approved in 2018 and the recent approval of the first Covid-19 vaccine using messenger RNA technology in the UK and Canada.

Founded: 2012

Cambridge Epigenetix was founded by Cambridge professor Sir Shankar Balasubramanian, who co-invented the technology behind next-generation sequencing now used by DNA sequencing giant Illumina.

Cambridge Epigenetix pioneered epigenetic tests for the early detection of cancer and other diseases. Its technology is able to reveal information about the activity of disease-related genes not available using genetic analysis alone. The company has raised approximately 50M to date.

Founded: 2016

Mogrify uses an algorithm to predict what chemicals are needed to convert any human cell type into another cell type.

The company raised a 14.5M Series A last year and signed a licensing agreement with genomic medicine company Sangamo earlier this year to help the US company scale up the production of off-the-shelf cell therapies for inflammatory and autoimmune diseases.

In October, Mogrify announced the launch of a platform that can model the epigenetic state of cells and provide important information on switches that can change cell biology.

Founded: 2016

Previously known as Elpis Biomed, Bit Bio was founded by neurosurgeon and cell therapy specialist Mark Kotter, who is also a Cambridge academic. The company is creating cell therapies through cellular reprogramming.

Bit Bios approach is to screen large datasets for cocktails of proteins that are needed to turn stem cells into the desired cell type. The company then genetically engineers the stem cells so that they switch on the production of these proteins when given an antibiotic.

The company has so far raised 44.4M to support the development of this technology.

Founded: 2015

Evonetix is working on a fast, scalable, high fidelity approach to DNA synthesis. The company is currently developing a DNA on a chip technology that will allow DNA to be made simultaneously at many different points on a silicon chip via electronic programming.

The company has so far raised approximately 36M. Earlier this year, Evonetix partnered with imec, a Dutch research and innovation hub active in the fields of nanoelectronics and digital technologies. This will increase the production of Evonetixs chips, enabling the platform to be manufactured at a commercial scale.

Founded: 2016

Microbiotica was established in 2016 as a spin-out from the Wellcome Sanger Institute. The company uses clinical data and detailed microbiome profiling to develop therapeutics and diagnostic biomarkers

In 2018, Microbiotica entered into a collaboration with Genentech worth up to 452M to develop microbiome-based treatments for inflammatory bowel disease. The company is also developing treatments for cancer and Clostridium difficile infections

In June, Microbiotica started a collaboration with Cancer Research UK and Cambridge University Hospitals NHS Foundation Trust to identify and develop combinations of microbiome therapeutics and biomarkers for cancer patients receiving immune checkpoint inhibitor therapy.

Founded: 2016

NodThera is targeting several inflammatory conditions affecting the liver, lungs, and bowels.These conditions involve excessive activation of a multi-protein complex called the NLRP3 inflammasome, which has received a lot of interest from big pharma over the past year. This complex regulates the release of two proinflammatory proteins that normally help the body to get rid of infection or heal tissue damage.

The company raised 48.7M in Series B funding in the summer. Its most advanced candidate is now in phase I clinical trials.

Founded: 2009

CN Bio Innovations develops human organ-on-a-chip technology for testing and developing therapies to treat human diseases. Having initially operated in stealth mode, the company is now selling products and services and vocal about it.

CN Bio works with academic bioengineering groups at MIT and Imperial College London and provides its technology to the US FDA. Last month, the company launched a commercial drug metabolism and safety toxicity testing service for researchers and companies to assess liver toxicity of various compounds.

Cover illustration by Anastasiia Slynko

Visit link:
Top 15 Biotech Companies Shaking up the Scene in... - Labiotech.eu