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Immunology

Early Immunological Studies Show Universally Effective Antibodies Against SARS-CoV-2 in Recovered Humans – Technology Networks

The first round of results from an immunological study of 149 people who have recovered from COVID-19 show that although the amount of antibodies they generated varies widely, most individuals had generated at least some that were intrinsically capable of neutralizing the SARS-CoV-2 virus.

Antibodies vary widely in their efficacy. While many may latch on to the virus, only some are truly neutralizing, meaning that they actually block the virus from entering the cells.

Since April 1, a team of immunologists, medical scientists, and virologists, has been collecting blood samples from volunteers who have recovered from COVID-19. The majority of the samples they have studied showed poor to modest neutralizing activity, indicating a weak antibody response. However, a closer look revealed everyones immune system is capable of generating effective antibodiesjust not necessarily enough of them. Even when neutralizing antibodies were not present in an individuals serum in large quantities, researchers could find some rare immune cells that make them.

This suggests just about everybody can do this, which is very good news for vaccines, says Michel C. Nussenzweig, head of the Laboratory of Molecular Immunology at Rockefeller. It means if you were able to create a vaccine that elicits these particular antibodies, then the vaccine is likely to be effective and work for a lot of people.

Moreover, the researchers identified three distinct antibodies that were shown to be the most potent of the bunch in neutralizing the virus. They are working to develop them further into therapeutic and preventive drugs.

The findings are shared on BioRxiv ahead of submission to peer-reviewed scientific journals. Nussenzweigs collaborators include, Davide F. Robbiani, Marina Caskey, Paul Bieniasz, Theodora Hatziioannou, and Charles M. Rice.

From the beginning of April and over 5 weeks, 149 people who had recovered from COVID-19 visited The Rockefeller Hospital to donate plasma, the portion of the blood that contains the antibodies, and the immune B-cells that produce them. The participants had experienced symptoms for an average of 12 days, and had their first symptoms on average 39 days before plasma donation.

Bieniasz and Hatziioannous team used an essay they had developed to test the neutralizing activity of the plasma samples. This involved mixing the plasma with a pseudo SARS-CoV-2 virus and measuring how well this mixture could still infect human cells in a dish.

In 33 percent of donors, the neutralizing activity of plasma was below detectable levels. Its possible that for many in this group, their immune systems first line of defense had resolved the infection quickly, before the antibody-producing cells were called in.

The majority of the plasma samples showed poor to modest neutralizing activity. And for 1 percent of donors it was remarkably high. Like in other diseases, everyone responds differently, says Robbiani, research associate professor at Laboratory of Molecular Immunology. Some people have poor response, some average. And then there is a fraction of people that are exceptional responders.

Those elite responders are crucial to the teams plans. The high numbers of neutralizing antibodies in their serum makes it possible for researcher to catch the rare B cells that make them. They can then clone the antibodies from those cells, and use them to emulate the same strong defense in other people.

Out of the numerous antibodies generated by elite responders who had the best performing plasma, the team identified 40 that neutralized the virus, and zeroed in on three that could do so even at very low concentrations. The team has cloned these most potent antibodies and is now working to develop them for clinical use.

Neutralizing antibodies found in this study bind to at least three distinct sites on the receptor-binding domain (RBD) subunit of the spike protein, which is what SARS-CoV-2 uses to gain entry to host cells. A second look at the low-performing plasma samples revealed they also contained these RBD-binding antibodies, albeit in small quantities.

We now know what an effective antibody looks like and we have found similar ones in more than one person, Robbiani says. This is important information for people who are designing and testing vaccines. If they see their vaccine can elicit these antibodies, they know they are on the right track.

ReferenceConvergent Antibody Responses to SARS-CoV-2 Infection in Convalescent Individuals. Davide F. Robbiani et al. bioRxiv, doi: https://doi.org/10.1101/2020.05.13.092619.

This article is based on research findings that are yet to be peer-reviewed. Results are therefore regarded as preliminary and should be interpreted as such. Find out about the role of the peer review process in research here. For further information, please contact the cited source.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Early Immunological Studies Show Universally Effective Antibodies Against SARS-CoV-2 in Recovered Humans - Technology Networks

Immunology

IHW organises web summit on vasculitis care during COVID-19 organised – Express Healthcare

Rheumatologists, immunologists highlighted that rheumatoid diseases caused by malfunctioning of immune system need continuous care, voluntary discontinuation of prescribed medicine will cause more harm than good

At a time when fear of coronavirus infection is adding to worries of patients living with chronic diseases, rheumatologists and immunologists at the IHW Councils web summit on vasculitis care during COVID-19 highlighted that rheumatoid diseases caused by malfunctioning of the immune system need continuous care and voluntary discontinuation of prescribed medicine will cause more harm than good.

Vasculitis is a combination of diseases. Though it is not very common in India but the impact of the disease can be very serious. Though the medicines prescribed for vasculitis are immunosuppressant in nature, they do not make the patient more vulnerable to COVID-19 infection. It will be highly recommended that vasculitis patients should not stop their medicines until they have confirmed COVID-19 infection and are advised by doctors to do so as the mortality of vasculitis is far higher than COVID-19, saidDr Debasish Danda, Professor & Founder of Clinical Immunology & Rheumatology, CMC Vellore; President-Elect, Asia Pacific League of Associations for Rheumatology (APLAR).

While highlighting a greater need for awareness about diseases like vasculitis, which has nearly 20 different types of conditions, Dr Danda said, Unfortunately, we do not know the right prevalence of the disease in India as there is no population-based study. According to facility-based studies conducted at the Christian Medical College, Vellore, vasculitis variant like Takayasu has seen 600 patients in 15 years and this is among the largest facility-based studies conducted anywhere in the world. Diseases like Takayasu, a form of vasculitis that harms the medium blood vessels, are often misdiagnosed as TB due to thesimilarity in symptoms such as fever and rapid weight loss.

Discussing the types of vasculitis,Dr Ramnath Misra, Head, Clinical Immunology & Rheumatology, KIMS, Bhubaneswar & Ex- Dean and Head Clinical Immunology, SGPGIMS, Lucknow,said, There are two types of vasculitis: firstly, primary vasculitis where the name of the vasculitis type depends on the size of the affected vessels. The most common types of vasculitis in this segment are Takayasu, Kawasaki, etc. The other type is known as secondary vasculitis which happens due to any existing disease in the patient. The number of patients affected by secondary vasculitis outnumbers the patients suffering from primary vasculitis.

Globally, May is observed as Vasculitis Awareness Month. Recently, a number of COVID-19-infected children from Europe and North America have shown multi-system the inflammatory condition with some features similar to those of Kawasaki disease and toxic shock syndrome, prompting intensive care arrangements.

Kawasaki is a medium vessel vasculitis that is almost exclusively seen in very young children, especially infants and those below five. Though there is no known cause of the disease, Kawasaki disease may have an association with coronavirus, as Northwestern Universitys Kawasaki disease expert Dr Anne Rowley suggests that serious illness and shock in children may have been caused by the mutation of the virus. Kawasaki is not a rare disease Japan sees 13,000 new cases annually, while at PGI Chandigarh, we get about 80-100 children every year with symptoms of Kawasaki disease, saidDr Surjit Singh, Head, Department of Paediatrics and Chief, Allergy Immunology Unit, Advanced Paediatrics Centre, PGIMER, Chandigarh & Principal Investigator, ICMR Centre for Advanced Research in Primary Immunodeficiency Diseases.

The common symptoms of Kawasaki disease are redness in eyes, lips, tongue as well as swelling in neck, hands and feet. Early detection of the disease is the single-most effective way to prevent any further damage it is advisable to diagnose the disease within 5-6 days, otherwise it may cause permanent damage to coronary arteries. In many cases adult heart attack patients were found to have Kawasaki disease as children that remained undiagnosed, Dr Singh added.

Talking about Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV),Dr Aman Sharma, Professor, Clinical Immunology and Rheumatology, Internal Medicine, PGIMER, Chandigarh, said, ANCA-associated vasculitis causes inflammation in small vessels and can even destroy them, damaging organs. We receive about 350-400 patients every year in PGIMER Chandigarh vasculitis is not as rare as we think it to be.

The virtual summit on managing vasculitis and coronavirus, organised by theIntegrated Health and Wellbeing (IHW) Council, was also attended by Dr Vishad Viswanath, Director, Institute of Rheumatology & Immunology Research, Thiruvananthapuram, Kerala and Dr Rohini Samant, Professor, Department of Rheumatology, Hinduja National Hospital, Mumbai.

Kamal Narayan, CEO, IHW Council, who moderated the session, said, Though vasculitis diseases are rare, there is a high possibility that they have substantial prevalence in India, given our large pool of population. Clinicians are almost unanimous in their views that it is not possible to prevent such diseases as there is no scientific knowledge on what causes these diseases. In such a condition, early and timely diagnosis of vasculitis diseases itself is a big prevention as it can help in effective treatment and management. Following our philosophy of enabling people to live a healthy life, we found it important to make more people aware about these diseases which can help in early reporting and diagnosis.

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IHW organises web summit on vasculitis care during COVID-19 organised - Express Healthcare

Immunology

Third-year UND medical student among volunteers on state penitentiary immunology teams – Grand Forks Herald

Espelien was one person on three four-person immunology teams to conduct mass testing at the North Dakota State Penitentiary two weeks ago. About half of those teams are made up of medical student volunteers, said John Hagan, a UND School of Medicine professor who also oversees medical care in Department of Corrections and Rehabilitation facilities. Espelien had worked in the prison one other time, on a mental health rotation as part of her education, but her latest task at the penitentiary - donning full-body personal protective equipment and administering COVID-19 tests to state prisoners in their pods - was unlike any she'd been asked to do before as a medical student.

"I was really excited to be able to talk to patients and do hands-on patient care for the first time in months," she said. "It was good to get back in the swing of things, and I really enjoyed talking to the residents there. A lot of them were joking and in fairly good spirits, with the whole thing going on."

It's widely acknowledged that prison and jail populations are particularly vulnerable to COVID-19. Due to the confined space, prisoners' inability to practice social distancing, and staff's limited ability to isolate sick inmates, once the virus is introduced to a prison population, it has been found to spread efficiently.

DOCR took quick steps early in the pandemic to prevent the spread of the virus in their facilities. They closed their doors to new intakes on March 13, and Hagan said North Dakota's prisons have reduced their population by about 15% - but mass testing is still essential for protecting those populations, he said.

Two weeks ago, Hagan put out a call to his medical students and residents looking for volunteers for the immunology teams in the prisons.

"We've actually had more requests to come out than we have slots," Hagan said. "I love the fact that these are adults who are in advanced learning, and they're eager to get out and take care of sick people. I'm pretty proud of them."

Espelien, an Albuquerque native who completed her undergraduate studies in San Diego, was drawn to North Dakota by the quality of UND's medical school and family ties in Park River, and, going into her fourth year of medical school, she aims to apply for an OB/GYN residency. Her work in the penitentiary was part of Hagan's internal medicine rotation. She said she's not aware of any other medical school that offers rotations in state prisons.

Her work with the immunology team began Wednesday, May 13, and lasted three days, the time it took to complete the mass tests in the North Dakota State Penitentiary. Mass tests also were completed in four other DOCR facilities, but only the testing event in the NDSP yielded any positive test results - four people in custody reportedly tested positive for the virus, according to the DOCR website.

"That'll be good information for them to know, and they'll be able to isolate those residents and, hopefully, keep all the fellow residents and staff members safe," Espelien said. "I think it's important to keep doing this epidemiologic testing at the penitentiaries to make sure that we can keep any outbreaks controlled and make sure that this vulnerable patient population is protected."

Four positive results were initially reported in the Missouri River Correctional Center in Bismarck as well, but the case count in MRCC was updated to zero after the North Dakota Department of Health reported that malfunctioning testing equipment yielded 82 false positives statewide last week.

Espelien said tasks were split among the team members. One person screened patients' temperatures and asked questions about symptoms, and another team member ensured samples were labeled properly and the correct paperwork was filled out. Espelien was one of the team members who administered the swab tests.

Residents of the penitentiary had the option to either take the test or decline the test and isolate for 14 days. Espelien said that, though a few people weren't happy about the tests, which can be uncomfortable to take, she said everyone was pleasant and had generally positive attitudes.

"I think that there are just some questions about how things are going outside of the penitentiary, since they only get to see what's on the news or what they hear from family members and staff," Espelien said. "So some of them were asking me questions about when antibody tests will be available, or how the throat swab is different from the nasal swab, and if one is better than the other. So I guess there was an air of curiosity, more than fear or worry or things like that."

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Third-year UND medical student among volunteers on state penitentiary immunology teams - Grand Forks Herald

Immunology

argenx Announces Positive Topline Results from Phase 3 ADAPT Trial of Efgartigimod in Patients with Generalized Myasthenia Gravis – GlobeNewswire

Regulated information Inside information

May 26, 2020Breda, the Netherlands / Ghent, Belgium argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, today announced positive topline data from the pivotal ADAPT trial of efgartigimod. ADAPT met its primary endpoint defined as percentage of responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score among acetylcholine receptor-antibody positive (AChR-Ab+) generalized myasthenia gravis (gMG) patients. Responders are defined as having at least a two-point improvement on the MG-ADL score for at least four consecutive weeks. Based on these results, argenx plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of 2020.

Highlights of topline ADAPT data

The efgartigimod data showed rapid and robust responses in people with gMG, as well as a favorable tolerability profile, said James F. Howard Jr., M.D., Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and principal investigator for the ADAPT trial. Patients with this devastating disease can experience chronic and potentially life-threatening muscle weakness that has a major impact on their quality of life, and more treatment options are needed. These data are very encouraging as they show efgartigimod has potential to make a meaningful impact on daily living activities, and we are hopeful they will lead to a new treatment being available for the gMG community.

With the ADAPT trial, we set out to evaluate efgartigimods ability to redefine the treatment paradigm for people living with gMG. The data showed that efgartigimod drove fast and deep responses, including in a proportion of patients who achieved minimal or no symptoms after treatment. In addition, we saw responses that lasted beyond eight or 12 weeks, supporting our plans to offer individualized dosing schedules that are purpose-fit to the variability in disease course that gMG patients experience, commented Wim Parys, M.D., Chief Medical Officer of argenx. Based on these data, we intend to submit a BLA for efgartigimod to the FDA before the end of the year, taking us one step closer to potentially making efgartigimod available to patients in 2021. All of us at argenx want to thank the patients and healthcare providers who participated in the ADAPT trial. ADAPT is the first pivotal trial of efgartigimod and these data further our confidence in its broad opportunity in other severe, IgG-mediated autoimmune diseases.

Additional ADAPT results, including secondary endpoints and prespecified analyses

Detailed data from the ADAPT trial will be submitted for presentation at a future medical meeting.

Phase 3 ADAPT Trial DesignThe Phase 3 ADAPT trial was a randomized, double-blind, placebo-controlled, multi-center, global trial evaluating the safety and efficacy of efgartigimod in patients with gMG. A total of 167 adult patients with gMG in North America, Europe and Japan enrolled in the trial and were treated. Enrolled patients had a confirmed gMG diagnosis and an MG-ADL total score of five or greater. Patients were on a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs, and were required to remain on that stable dose throughout the primary trial. Patients were eligible to enroll in ADAPT regardless of antibody status, including patients with AChR antibodies (AChR-Ab+) and patients where AChR antibodies were not detected.

Patients were randomized in a 1:1 ratio to receive efgartigimod or placebo for a total of 26 weeks as part of the primary trial. ADAPT was designed to enable an individualized treatment approach with an initial treatment cycle followed by a variable number of subsequent treatment cycles. Treatment cycles consist of four infusions of efgartigimod (10mg/kg IV) or placebo at weekly intervals. Retreatment with additional treatment cycles was initiated according to clinical response. The primary endpoint was the number of AChR-Ab+ patients who achieved a response on the MG-ADL score defined by at least a two-point improvement for four or more consecutive weeks.

After the 26-week primary ADAPT trial, patients were eligible to roll-over into an open-label extension, ADAPT Plus.

About Efgartigimod

Efgartigimod is a first-in-class antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.

About Myasthenia Gravis (MG)

MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. More than 85% of people with MG progress to generalized MG (gMG) within 18 months, where muscles throughout the body may be affected, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility. In more life-threatening cases, MG can affect the muscles responsible for breathing. Patients with confirmed AChR antibodies account for 80-90% of the total gMG population. There are approximately 65,000 people in the United States and 20,000 people in Japan living with the disease.

Conference Call DetailsManagement will host a conference call and webcast presentation today at 2:30 p.m. Central European Summer Time (CEST) / 8:30 a.m. Eastern Daylight Time (EDT). To participate in the conference call, please select your phone number below and use the confirmation code 6295982. The webcast may be accessed on the Investors page of the argenx website at http://www.argenx.com or by clicking here.

Dial-in numbers:Please dial in 510 minutes prior to 2:30 p.m. CEST / 8:30 a.m. EDT using the number and conference ID below.

Confirmation Code: 6295982

Belgium: +32 (0)2 793 3847Belgium: 0800 484 71France: +33 (0)1 7070 0781France: 0805 101 465Netherlands: +31 (0)2 0795 6614Netherlands: 0800 023 5015UK: +44 (0) 844 481 9752UK: 0800 279 6619US: +1646 741 3167US: 1877 870 9135

About argenxargenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx is translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx is evaluating efgartigimod in multiple serious autoimmune diseases, and cusatuzumab in hematological cancers in collaboration with Janssen. argenx is also advancing several earlier stage experimental medicines within its therapeutic franchises. argenx has offices in Belgium, the United States and Japan. For more information, visit http://www.argenx.com and follow us on LinkedIn at https://www.linkedin.com/company/argenx/.

Contacts:

Beth DelGiacco, Vice President, Investor Relations (US)+1 518 424 4980bdelgiacco@argenx.com

Joke Comijn, Director Corporate Communications & Investor Relations (EU)+32 (0)477 77 29 44+32 (0)9 310 34 19JComijn@argenx.com

Forward-looking Statements

The contents of this announcement include statements that are, or may be deemed to be, forward-looking statements. These forward-looking statements can be identified by the use of forward-looking terminology, including the terms believes, estimates, anticipates, expects, intends, may, will, or should and include statements argenx makes concerning the safety, tolerability and efficacy of efgartigimod and the results of the ADAPT trial; the timing of planned regulatory submissions with the FDA and, if approved, launch in the U.S.; the therapeutic and commercial potential of efgartigimod; the opportunity of efgartigimod in other severe, IgG-mediated autoimmune diseases; and the intended results of its strategy.By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenxs actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including argenxs expectations regarding its the inherent uncertainties associated with competitive developments, preclinical and clinical trial and product development activities and regulatory approval requirements; failure to demonstrate the safety, tolerability and efficacy of argenxs product candidates; final and quality controlled verification of data and the related analyses; the expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration and European Medicines Agency; the possibility of having to conduct additional clinical trials; argenxs reliance on collaborations with third parties; estimating the commercial potential of argenxs product candidates; argenxs ability to obtain and maintain protection of intellectual property for its technologies and drugs; argenxs limited operating history; and argenxs ability to obtain additional funding for operations and to complete the development and commercialization of its product candidates. A further list and description of these risks, uncertainties and other risks can be found in argenxs U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenxs most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.

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argenx Announces Positive Topline Results from Phase 3 ADAPT Trial of Efgartigimod in Patients with Generalized Myasthenia Gravis - GlobeNewswire

Immunology

Q32 Bio Launches to Develop Novel Therapeutics Targeting Regulators of Innate and Adaptive Immunity – P&T Community

CAMBRIDGE, Mass., May 27, 2020 /PRNewswire/ --Q32 Bio, a biotechnology company developing biologics to restore healthy immune regulation, today announced the company's pipeline and plans to enter the clinic this year with its lead candidate. Enabled by a $46M Series A financing led by Atlas Venture, and a world class team and Scientific Advisory Board, Q32 Bio is advancing a portfolio of biologics targeting the body's innate and adaptive immune systems. The company has a robust product pipeline including a monoclonal antibody antagonist of the interleukin-7 receptor (IL-7R), and a complement therapeutics platform that has generated fusion proteins that downregulate complement activity specifically in disease-affected tissues. The company is led by industry veterans Michael Broxson as Chief Executive Officer, and co-founder, Shelia Violette, Ph.D., as Chief Scientific Officer and President of Research.

Q32 Bio was seeded and incubated by Atlas Venture with foundational science from renowned researchers in immunology Michael Holers, M.D. and Joshua Thurman, M.D., both from the University of Colorado, and Steven Tomlinson, Ph.D. from the Medical University of South Carolina. Dr. Holers serves as chairman of the Scientific Advisory Board, which is comprised of distinguished global experts in autoimmune and inflammatory disease.

The subsequent Series A financing included Atlas Venture, OrbiMed Advisors, Abingworth, Sanofi Ventures, University of Colorado and Children's Hospital Colorado Center for Innovation.

"Autoimmune and inflammatory diseases are driven by dysregulation of the immune response," said Mr. Broxson. "Q32 Bio has a preeminent team with expertise in both innate and adaptive immunity, a board of directors and scientific advisory board made up of leaders in our field, and a syndicate of blue-chip investors. This gives us a running start in developing therapies that may improve and save lives. I am thrilled to join Q32 Bio and look forward to advancing our first two immune-regulating therapies into first-in-human studies planned for 2020 and 2021."

Integrated Approach to Restoring Healthy Immune Regulation Q32 Bio's most advanced program, ADX-914, is a fully human anti-IL-7R antibody licensed from Bristol Myers Squibb (NYSE: BMY) that re-regulates adaptive immune function. The cytokine IL-7 has demonstrated the ability to drive several T-cell mediated pathological processes. It stimulates T-effector and T-memory cells, lowering the threshold-response to disease related antigens; inhibits the immunosuppressive function of T-regulatory cells; and promotes pathogenic autoantibody production. Inhibition of IL-7R signaling has the potential to durably and safely restore healthy immune regulation in numerous autoimmune and inflammatory diseases. Q32 Bio is advancing plans for the first-in-human trial of ADX-914 while continuing to monitor the potential impact of COVID-19 on clinical operations. Pending confirmation that trial sites are able to operate and enroll patients safely, a biomarker-guided ADX-914 Phase 1 trial is planned to start in late 2020.

Q32 Bio is developing ADX-914 (formerly known as BMS-986265) as part of an exclusive worldwide licensing agreement with Bristol Myers Squibb. Under the terms of the agreement, Bristol Myers Squibb received an upfront payment and became a minority shareholder of Q32 Bio. Bristol Myers Squibb has the potential to earn additional milestone payments subject to the achievement of certain development, regulatory and sales-based milestones, as well as tiered annual net sales royalties. Q32 Bio is solely responsible for all future development and commercialization costs of ADX-914.

Q32 Bio's lead program in innate immunity, ADX-097, is based on a groundbreaking platform providing tissue-targeted regulation of the complement system. Complement is an integral component of the innate immune system that provides a first line of defense for clearing pathogens and removing damaged cells. In a wide range of autoimmune and inflammatory diseases the complement system becomes hyperactivated, causing the immune system to attack and damage otherwise healthy tissue. Q32 Bio's proprietary and unique platform has yielded a pipeline of novel protein therapeutics that provide potent inhibition of complement in diseased tissues without long-term systemic blockade a key differentiator versus current complement therapeutics. Q32 Bio has initiated IND-enabling activities for ADX-097 and first-in-human dosing is planned for 2H 2021.

"Given their broad roles in mediating immunity, the complement system and IL-7 signaling pathways are high potential arenas for drug development," said Dave Grayzel, MD, Partner, Atlas Venture and chairman of the Q32 Bio Board of Directors. "With a best-in-class IL-7R antibody and fusion proteins designed to target the complement system at sites of activation in a unique and differentiated manner, Q32 Bio is well positioned to make a meaningful impact for patients with serious inflammatory and autoimmune diseases."

Experienced Leadership TeamMichael Broxson brings over 20 years of industry experience to Q32 Bio. He joined from Goldfinch Bio, where he was Chief Business and Operating officer. Prior to Goldfinch he served in leadership roles in business development, new product planning, strategy and finance at Takeda Pharmaceuticals. He holds BA and MSPH degrees in Toxicology from Tulane University and an MBA from the University of Chicago's Booth School of Business and is a CFA charterholder.

Shelia Violette, Ph.D., joined Q32 Bio from Atlas Venture, where she was Entrepreneur in Residence and initially co-led the formation of Q32 Bio. Previously she served in leadership roles in the Immunology Research group at Biogen, joining Stromedix as Vice President of Research and returning to Biogen as Vice President of Research, spearheading the tissue injury and fibrosis therapeutic area. She holds a Ph.D. in Pharmacology from Yale University.

About Q32 BioQ32 Bio is a biotechnology company developing therapies targeting powerful regulators of the innate and adaptive immune systems, to re-balance immunity in severe autoimmune and inflammatory diseases. Q32 Bio's lead programs, focused on the IL-7R pathway and complement system, address immune dysregulation to help patients take back control of their lives. For more information, please visit http://www.Q32bio.com.

About Atlas VentureAtlas Venture is a leading biotech venture capital firm. With the goal of doing well by doing good, the company has been building breakthrough biotech startups for over 25 years. Atlas works side by side with exceptional scientists and entrepreneurs to translate high impact science into medicines for patients. Our seed-led venture creation strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. For more information, please visitwww.atlasventure.com.

About AbingworthAbingworth is a leading transatlantic life sciences investment firm. We help transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams and building world-class companies. With offices in Menlo Park (California), Boston and London, Abingworth has invested in approximately 160 life science companies, leading to more than 40 M&A/exits and over 65 IPOs since 1973.

About OrbiMed OrbiMed is a leading healthcare investment firm, with $13 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, utilizing a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed maintains offices in New York City, San Francisco, Shanghai, Hong Kong, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies.

About Sanofi VenturesSanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi's business footprint, and digital health solutions. For more information, visitwww.sanofiventures.com.

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Q32 Bio Launches to Develop Novel Therapeutics Targeting Regulators of Innate and Adaptive Immunity - P&T Community

Neuroscience

Denali unveils new way of crossing blood brain barrier as the big neuroscience bets enter its clinical years – Endpoints News

Five years ago, as much of pharma began leaving neuroscience, three big-name scientists from Genentech and some A-list investors, including ARCH and Flagship, made a $217 million bet that new genetic insights and a reliance on biomarkers could bring them success. They called it Denali Therapeutics.

Still, Denali faced the problem that neuroscience developers have faced for decades: How do you get a large molecule across the blood-brain barrier, a natural defense evolved precisely to keep them out? Enzyme replacement therapy, for instance, would be a great candidate to treat several neurological disorders, but enzymes cant cross the barrier.

Now, Denali thinks theyve solved the problem, or at least part of it. In a pair of papers published inScience Translational Medicine,the South San Francisco biotech detailed the invention of a new transport vehicle to sneak large molecules past the brains gates. So far, its been used in mice and monkeys, but they wont wait long to bring it to patients: A clinical trial using it to replace an enzyme lost in people with Hunters syndrome is set to begin this year, with proof-of-concept data expected to come before 2021.

The blood-brain barrier consists in part of tightly packed endothelial cells. Since certain molecules, such as insulin, cross the barrier by first binding to receptors on these cells and then being allowed through, scientists have long tried to build antibodies that can similarly bind to these receptors and shuttle across a therapeutic cargo. But the results, over several decades, have been less than transformative.

CEO and founder Ryan Watts has been part of that search since his Genentech days. The research method he and Denalis scientists came up with began with a process called directed evolution in which a protein is induced to mutate repeatedly, until it gives rise to a protein with the qualities you want to build a protein, called an FC fragment, that binds to whats called a transferrin receptor, a node that normally imports iron into the brain. In theory, there are numerous drugs one could then hook onto that Fc fragment, but Denali first tested it with an antibody-targeting enzyme called beta-secretase. The enzyme is linked to the build-up of amyloid plaques in people with Alzheimers, and the researchers showed their vehicle reduced the amount of amyloid in mice and monkeys.

In a second study, the researchers attached an enzyme called iduronate-2-sulfatase, the critical protein that people with Hunters syndrome are missing. Without it, sugars called glycosaminoglycans build up in cells, causing abnormalities in several different organs. Shire gained approval for an enzyme replacement therapy in 2006, but it only works outside the brain (the companys erstwhile efforts to improve cognitive function yielded little promise). Using the transport vehicle, though, Denali was able to get significantly increased brain penetration of the enzyme and reduce the pathology in mice and monkeys.

Denali played up the potential versatility of their approach over other blood-brain-barrier-crossing proposals, such as bispecific antibodies, saying you can attach a greater range of therapies to their vehicle. The company has over a dozen programs including a Parkinsons one now in the clinic but the first test of the vehicle will be later this year, in 16 kids with a rare disease whose worst symptoms remain untreated.

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Denali unveils new way of crossing blood brain barrier as the big neuroscience bets enter its clinical years - Endpoints News

Neuroscience

First spine surgeries performed in the region – Waushara Argus

The surgical team of providers from ThedaCare and Neuroscience Group have successfully performed the first robotic-assisted spine surgeries in Northeast and Central Wisconsin using the Mazor X Platform this week.

It is estimated that nearly 80 percent of the population will suffer from lower back pain at some point in their lives, with one in 20 of them needing surgery. As spinal surgery has evolved, more focus has been placed on new techniques often providing benefits of less blood loss, less radiation exposure, less tissue damage, and less pain and resulting opioid use.

To enhance access to specialized care close to home, ThedaCare recently installed the new Mazor X StealthTM Edition Robotic Guidance Platform. ThedaCare, in partnership with Neuroscience Group, is one of the first in the area to offer this spine surgical technology to patients and is one of the first to adopt this technology in the United States.

The Mazor X is a guide for the surgeons hands that provides a huge advance in accurate pedicle screw placement, said Randall Johnson, MD PhD, neurosurgeon at Neuroscience Group and Spine Surgery Medical Director at ThedaCare. This technology will set the standard for the future of minimally invasive and scoliosis surgery.

The Mazor X Platform enhances surgeons limited view of patients anatomy with a 3D surgical plans and analytics. This provides comprehensive information and visualization before the surgery starts, ultimately supporting the commitment to operate with precision. According to Dr. Johnson, this means the angle, width and length of every screw, is planned from the preoperative CT scan and two x-rays during surgery. Its safer for the patient and the surgical team in terms of radiation exposure as it limits the number of necessary x-rays.

A patients health and safety are top priorities for ThedaCare, said Michael Hooker, DO, Vice President and Chief Medical Officer of Acute Care at ThedaCare. By bringing new technology to the area, ThedaCare is helping promote faster recovery times, reducing postoperative pain and improving results for our patients. The staff is committed to getting patients back to doing what they love most, as quickly as possible.

Due to the COVID-19 response, much like health care systems across the country, ThedaCare made necessary adjustments to operations and services. In early May, ThedaCare began reinstating services that had previously been deferred, including surgeries.

The mission is to improve the health of the communities ThedaCare serves, and that means connecting community members to the care they need, said Lynn Detterman, Senior Vice President of ThedaCare South Region. Because of the teams tireless work through the intentional phased reopening plan, multiple safeguards are in place and are in a position to begin offering essential and routine services for illness and ongoing medical care. ThedaCare is proud to welcome technology like this to enhance care options for patients.

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First spine surgeries performed in the region - Waushara Argus

Neuroscience

The Neuroscience of Thriving Through Crisis: How to Make Work Sustainable and Leverage the Moment – ATD

Dr. David Rock coined the term Neuroleadership and is the Director of the NeuroLeadership Institute, a global initiative bringing neuroscientists and leadership experts together to build a new science for leadership development. With operations in 24 countries, the Institute also helps large organizations operationalize brain research in order to develop better leaders and managers.

David co-edits the NeuroLeadership Journal and heads up an annual global summit. He has written many of the central academic and discussion papers that have defined the Neuroleadership field. He is the author of the business best-seller Your Brain at Work (Harper Business, 2009), as well as Quiet Leadership (Harper Collins, 2006) and the textbook Coaching with the Brain in Mind (Wiley & Sons, 2009). He blogs for the Harvard Business Review, Fortune Magazine, Psychology Today, and the Huffington Post, and is quoted widely in the media about leadership, organizational effectiveness, and the brain.

Academically, David is on the faculty and advisory board of CIMBA, an international business school based in Europe. He has been a guest lecturer at many universities including Oxford Universitys Said Business School. He is on the board of the BlueSchool, an initiative in New York City building a new approach to education. He received his professional doctorate in the Neuroscience of Leadership from Middlesex University in 2010.

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The Neuroscience of Thriving Through Crisis: How to Make Work Sustainable and Leverage the Moment - ATD

Neuroscience

Neuroscience-led AI leads to launch of universal hearing aid – Med-Tech Innovation

ChatableApps has launched a universal hearing aid, using neuroscience-led artificial intelligence to empower those suffering from hearing loss to hear one-to-one conversations clearly, using an everyday smartphone and a standard pair of earbuds.

ChatableApps AI was created by reverse engineering the brain to understand how a healthy brain should work, when listening to speech. The resulting technique is called end-to-end neural speech synthesis and involves the neuroscience-led AI listening to the sound mixture of a conversation, and repeating the voice only element in real-time.

While traditional approaches attempt to label and remove sounds we arent interested in, ChatableApps proprietary artificial intelligence called VOXimity works differently, identifying the voice we want to hear, and creating a new, identical voice which sounds the same as the original but without any other background sounds.

Mark Cuban, who appears on the US version of Dragons Den and was the first investor in ChatableApps, said: "Chatable is using AI to address growing health care inequality by providing an affordable hearing aid app that helps with conversation."

Giles Tongue, ChatableApps CEO, said: Our unique ground-up AI makes our universal hearing aid the first app capable of addressing the global hearing epidemic. Following successful preclinical trials, we have decided to launch immediately due to urgent demand from Audiologists to help people struggling because of Coronavirus: With many unable to lipread due to face masks or unable to visit a hearing clinic in an emergency, our app provides a lifeline that will help people communicate.

Globally there are 466 million people with disabling hearing loss. Hearing loss affects 11 million in the UK. The high costs of traditional hearing aids, and the fact that many people arent covered by their insurance or health service around the world, they are currently going untreated.

Dr Andrew Simpson, the founding scientist of ChatableApps, holds a PhD in auditory neural signal processing and has published widely on artificial intelligence and hearing topics. Working at the intersection of auditory neuroscience and artificial intelligence has led Dr Simpson to create VOXimity, which is real-time on-device speech processing.

Dr Simpson said: "Putting an auditory cortex on a smartphone has been a dream for a long time.As far as I know this is the most advanced real-time AI that has ever done so. Its been a long road, but our preclinical trials reveal something of a medical breakthrough: a hearing aid that doesnt need hearing tests and going straight into the hands of people who cant afford a hearing aid.

At pre-clinical trials where 86% reported that the ChatableApps universal hearing aid was better for conversation than their traditional hearing aid.

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Neuroscience-led AI leads to launch of universal hearing aid - Med-Tech Innovation

Neuroscience

Prothena to Present at the Jefferies Virtual Healthcare Conference on June 3 – Financialbuzz.com

DUBLIN, Ireland, May 27, 2020 (GLOBE NEWSWIRE) Prothena Corporation plc (NASDAQ:PRTA), a clinical-stage neuroscience company with expertise in protein misfolding, today announced that members of its senior management team will present and participate in the Jefferies Virtual Healthcare Conference on Wednesday June 3 at 3:30 PM ET.

A webcast of the company presentation can be accessed through the investor relations section of the Companys website at http://www.prothena.com. Following the presentation, a replay of the webcast will be available on the Companys website for at least 90 days following the presentation date.

About Prothena

Prothena Corporation plc is a clinical-stage neuroscience company with expertise in protein misfolding, focused on the discovery and development of novel therapies with the potential to fundamentally change the course of devastating diseases. Fueled by its deep scientific expertise built over decades of research, Prothena is advancing a pipeline of therapeutic candidates for a number of indications and novel targets for which its ability to integrate scientific insights around neurological dysfunction and the biology of misfolded proteins can be leveraged. Prothenas partnered programs include prasinezumab (PRX002/RG7935), in collaboration with Roche for the potential treatment of Parkinsons disease and other related synucleinopathies, and programs that target tau, TDP-43 and an undisclosed target in collaboration with Bristol-Myers Squibb for the potential treatment of Alzheimers disease, amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) or other neurodegenerative diseases. Prothenas proprietary programs include PRX004 for the potential treatment of ATTR amyloidosis, and programs that target A (Amyloid beta) for the potential treatment of Alzheimers disease. For more information, please visit the Companys website at http://www.prothena.com and follow the Company on Twitter @ProthenaCorp.

Media and Investor Contact:

Ellen Rose, Head of Communications650-922-2405, ellen.rose@prothena.com

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Prothena to Present at the Jefferies Virtual Healthcare Conference on June 3 - Financialbuzz.com