Solving the Wnt nuclear puzzle – Nature.com

Authors and Affiliations

Molecular Biology and Biochemistry, Simon Fraser University, Vancouver, British Columbia, Canada

Esther M. Verheyen

Centre for Cell Biology, Development, and Disease, Simon Fraser University, Vancouver, British Columbia, Canada

Esther M. Verheyen

Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

Cara J. Gottardi

Department of Cell and Developmental Biology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

Cara J. Gottardi

Correspondence to Esther M. Verheyen or Cara J. Gottardi.

Excerpt from:
Solving the Wnt nuclear puzzle - Nature.com

One essential step for a germ cell, one giant leap for the future of reproductive medicine – EurekAlert

image:

Image inspired by NASA's Apollo Program, representing the successfulin vitrogerm cell differentiation from TFAP2C-EGFP +ve human primordial germ cell-like cells (hPGCLCs; labeled in green) to DAZL-tdTomato +ve human mitotic pro-spermatogonia (labeled in red).

Credit: WPI-ASHBi/Kyoto University

KYOTO, Japan May 20, 2024

Infertility affects approximately 1 in 6 people in their lifetime worldwide according to the World Health Organization (WHO). Infertility as defined by the American Society for Reproductive Medicine (ASRM) is a disease, condition, or status characterized by the inability to achieve a successful pregnancy based on a patients medical, sexual, and reproductive history, age, physical findings, diagnostic testing, or any combination of those factors or requiring medical intervention such as the use of mature donor gametes to achieve a successful pregnancy either as an individual or with a partner. Although assisted reproductive technologies (ARTs), such as in vitro fertilization (IVF), have had a tremendous impact in treating certain forms of infertility not all forms of infertility (as defined by the ASRM) can be targeted with existing strategies.

Recently, one powerful technology has emerged known as human in vitro gametogenesis (IVG) using pluripotent stem cells (PSCs) such as induced pluripotent stem cells (iPSCs) from patients, to generate human germ cells with the capacity to potentially give rise to mature gametes in culture, offering a gateway to treating all form of infertility independent of gender. Nevertheless, human IVG research still remains in its infancy, with the current goal being to reconstitute the complete process of human gametogenesis. To date, one major challenge has been to recapitulate in the founder population of germ cells, or the human primordial germ cells (hPGCs), a hallmark event known as epigenetic reprogramming in which the inherited parental memory of cells, present on its DNA, is reset/erased that is required for proper germ cell differentiation.

Now, in a study published in Nature, researchers at the Institute for the Advanced Study of Human Biology (WPI-ASHBi) in Kyoto University, led by Dr. Mitinori Saitou, identify robust culture conditions necessary to drive epigenetic reprogramming and germ cell differentiation into precursors of mature gametes, the mitotic pro-spermatogonia and pro-oogonia with the capacity for extensive amplification, achieving a new milestone for human IVG research.

Previous work from Saitous team and other groups were successful in generating so-called human primordial germ cell-like cells (hPGCLCs) from PSCs in vitro, which recapitulated several fundamental features of hPGC, including the capacity to propagate. However, these hPGCLCs were unable to undergo epigenetic reprogramming and differentiation. Although such limitations could be bypassed by aggregating hPGCLCs with mouse embryonic (non-germinal) gonadal cells to mimic the microenvironment of the testis/ovary, thereby effectively reconstitute the tissue(s). However, this process is highly inefficient (with approximately only 1/10th of cells differentiating). Furthermore, the introduction of non-human cells is neither ideal nor practical from a clinical application perspective. Therefore, in order to achieve the ultimate goal of human IVG research, it is essential to identify the minimal culture conditions necessary to generate mature human gametes.

In their new study, Saitou and colleagues conducted a cell culture-based screen to identify potential signaling molecules required to drive epigenetic reprogramming and differentiation of hPGCLCs into mitotic pro-spermatogonia and oogonia. Surprisingly, the authors found that the well-established developmental signaling molecule, bone morphogenetic protein (BMP), played a crucial role in this reprogramming and differentiation process of hPGCLCs.

Indeed, considering that BMP signaling already has an established role in germ cell specification, it was highly unexpected that it also drives hPGCLC epigenetic reprogramming comments Saitou.

Remarkably, these hPGCLC-derived mitotic pro-spermatogonia/oogonia not only displayed similarities in gene expression and epigenetic profiles to that of actual hPGC differentiation in our bodies, but also underwent extensive amplification (over 10 billion-fold). Our approach enables near-indefinite amplification of mitotic pro-spermatogonia and oogonia in culture and we now also have the ability to store and re-expand these cells as needed says Saitou.

The authors also revealed the potential mechanisms of how BMP signaling may be leading to epigenetic reprogramming and hPGCLC differentiation. BMP (signaling) appears to be attenuating the MAPK/ERK (mitogen-activated protein kinase/extracellular-regulated kinase) signaling pathway and both the de novo and maintenance activities of DNMT (DNA methyltransferase), but further investigation will be necessary to determine the precise mechanism and whether this is direct or indirect, explains Saitou.

Our study represents not only a fundamental advance in our understanding of human biology and the principles behind epigenetic reprogramming in humans but also a true milestone in human IVG research says Saitou.

Saitou comments, although many challenges remain and the path will certainly be long, especially when considering the ethical, legal, and social implications associated with the clinical application of human IVG, nevertheless, we have now made one significant leap forward towards the potential translation of IVG into reproductive medicine.

These findings were published in Nature on May 20th 2024.

###

By

Spyros Goulas, PhD

Scientific Advisor

Institute for the Advanced Study of Human Biology (ASHBi)/Kyoto University

Email: goulas.spyros.3n@kyoto-u.ac.jp

Lead Principal Investigator

Mitinori Saitou, MD PhD

Institute for the Advanced Study of Human Biology (ASHBi)/Kyoto University

Email: saitou@anat2.med.kyoto-u.ac.jp

###

About Institute for the Advanced Study of Human Biology (ASHBi), Kyoto University

What key biological traits make us human, and how can knowing these lead us to better cures for disease?ASHBi investigates the core concepts of human biology with a particular focus on genome regulation and disease modeling, creating a foundation of knowledge for developing innovative and unique human-centric therapies.

About the World Premier International Research Center Initiative (WPI)

The WPI programwas launched in 2007 by Japan's Ministry of Education, Culture, Sports, Science and Technology (MEXT) to foster globally visible research centers boasting the highest standards and outstanding research environments. Numbering more than a dozen and operating at institutions throughout the country, these centers are given a high degree of autonomy, allowing them to engage in innovative modes of management and research. The program is administered by the Japan Society for the Promotion of Science (JSPS).

Experimental study

Cells

In Vitro Reconstitution of Epigenetic Reprogramming in the Human Germ Line.

20-May-2024

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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One essential step for a germ cell, one giant leap for the future of reproductive medicine - EurekAlert

Prof. Jay Shendure Joins Somite Therapeutics as Scientific Co-founder – BioSpace

[[To comply with academic institution guidelines, the founders' academic affiliations and roles are listed only at the end of the statement.]]

BOSTON, May 21, 2024 /PRNewswire/ -- Somite Therapeutics, a tech-bio company harnessing big data and AI to pioneer novel cell replacement therapies, is thrilled to announce the addition of Prof. Jay Shendure as its newest Scientific Co-Founder.

Prof. Shendure, an HHMI Investigator and world leader in single-cell and functional genomic assays, has pushed the envelope on the scale of analyses that are possible today. He has developed massively parallel measurement approaches that solve open problems in biology and has increased the throughput of digital twin embryos by several orders of magnitude.

His addition to the team will help Somite advance its AI platform, AlphaStem, to develop cell replacement therapies for diseases such as diabetes, obesity, and muscular dystrophies.

"Our plan is to generate massive amounts of data to lay the foundation of our AI/ML platform, Alphastem," commented Dr. Micha Breakstone, Co-founder and CEO of Somite. "Prof. Shendure's addition marks a pivotal moment for our company as we continue to innovate and push the boundaries of what is possible in cell therapy."

About Prof. Jay Shendure

Jay Shendure, M.D., Ph.D. is an Investigator of the Howard Hughes Medical Institute, a Professor of Genome Sciences at the University of Washington, and Scientific Director of the Seattle Hub for Synthetic Biology (Allen-CZI-UW), the Allen Discovery Center for Cell Lineage Tracing, and the Brotman Baty Institute for Precision Medicine. His lab is known for the development and application of genomic technologies to outstanding challenges in genetics, molecular biology and developmental biology. Dr. Shendure is the recipient of the Curt Stern Award from the American Society of Human Genetics, the Richard Lounsbery Award from the National Academy of Sciences and the Mendel Award from the European Society of Human Genetics. He is also an elected member of the American Association for the Advancement of Science and the National Academy of Sciences. He received his MD and PhD degrees from Harvard Medical School.

About Somite

Somite.ai is a venture-backed company aiming to become the OpenAI of stem cell biology, developing AI foundation models to produce human tissue for cell therapies at scale for diseases such as diabetes, obesity, and muscular dystrophies. Somite's AI platform, AlphaStem, fuels a virtuous cycle: It enables new cell therapies, generating massive data that further improve the platform, empowering even faster therapy creation with broader applications.

Incorporated in Oct. 2023, Somite.ai has raised $5.3m to date.

Somite Management Team:

Scientific Co-founders:

Media Contact: media-relations@somite.ai Website: http://www.somite.ai

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May: academy-medical-sciences | News and features – University of Bristol

Two Bristol academics, Professors Eugenia Piddini and Gene Feder OBE, have been elected to the Academy of Medical Sciences respected and influential Fellowship. They join 58 exceptional biomedical and health scientists selected for their exceptional contributions to the advancement of medical science.

The new Fellows, announced on Tuesday 21 May, have been recognised for their remarkable contributions to advancing biomedical and health sciences, groundbreaking research discoveries and translating developments into benefits for patients and wider society.

Awardees join an esteemed Fellowship of over 1,400 researchers who are at the heart of the Academy's work, which includes nurturing the next generation of researchers and shaping research and health policy in the UK and worldwide. The expertise of Fellows elected this year spans a wide range of clinical and non-clinical disciplines, from midwifery to cancer stem cell biology.

Eugenia Piddini, Professor of Cell Biology in the School of Cellular and Molecular Medicine, is conducting innovative work to identify cell competition-based strategies to gain control over tissue colonisation, its impact in tissue colonisation in regenerative medicine and to prevent tumour expansion in cancer.

A cell and developmental biologist,Eugenia is known for her seminal work in the field of cell competition the mechanism of tissue quality control that removes damaged cells from tissues. Eugenias discoveries have helped widen the scope of cell competition in terms of physiological relevance and potential therapeutic impact. Recently, Eugenias group demonstrated that cell competition acts in adult tissues. There it can potentially slow down the onset of disease/ageing by eliminating damaged cells.

Eugenias team has also shown that tumour cells kill surrounding normal cells via cell competition to free space for their own growth. Their work has identified many mechanisms and signals that cells use to compete. By explaining the mechanisms that cells use to compete the Piddini group aims to identify cell competition-based strategies to gain control over tissue colonisation.

In recognition of her work Eugenia, who is also School Research Director, was awarded the British Society for Cell Biology Hooke Medal in 2019 and in 2023, was elected as a Member of the European Molecular Biology Organisation.

Gene Feder, is a GP and Professor of Primary Care at Bristols Centre for Academic Primary Care, Bristol Medical School and Director of VISION, a UK Prevention Research Partnership (UKPRP) consortium.

Professor Feder leads ground-breaking national and international research on domestic violence and abuse (DVA) from epidemiology to health care response. He is the architect of IRIS, a national DVA programme for general practice, and co-founded IRISi, a social enterprise implementing IRIS nationally. He has extended his research globally through EU and Medical Research Council grants, and co-leadership of HERA, a National Institute for Health and Care Research (NIHR) Global Health Group in collaboration with researchers in Brazil, Nepal Sri Lanka, and the occupied Palestinian territories (oPT).

Committed to developing and evaluating effective and compassionate health care, Professor Feder has championed the use of randomised controlled trials to test improvements in general practice care of patients with heart and respiratory conditions, and robust methods to develop and implement clinical guidelines that make a difference to patients. He extended epidemiological, trial and meta-analytic methods to research on gender-based violence, combining quantitative and qualitive data to evaluate interventions, collaborating with statisticians, epidemiologists, economists, and social scientists. He has chaired four NICE guidelines and the World Health Organisation (WHO) intimate partner and sexual violence guideline development group.

In 2012, he co-founded the Foundation for Family Medicine in Palestine, which aims to support universal health coverage throughout the occupied Palestinian Territories based on effective, efficient and high-quality primary care. In 2016, Professor Feder was awarded an OBE for services to health care and survivors of domestic violence. In 2022, Gene was appointed Director of VISION, a five-year UKPRP inter-disciplinary consortium researching the intersection of violence and health to reduce and mitigate the effects of violence through better measurement and analysis of health care, police, criminal justice, and voluntary sector data. He is an expert advisor to UK Government and WHO.

Professor Andrew Morris PMedSci, President of the Academy of Medical Sciences, said: It is an honour to welcome these brilliant minds to our Fellowship. Our new Fellows lead pioneering work in biomedical research and are driving remarkable improvements in healthcare. We look forward to working with them, and learning from them, in our quest to foster an open and progressive research environment that improves the health of people everywhere through excellence in medical science.

This year's cohort marks a significant milestone in the Academy's efforts to promote equality, diversity and inclusion (EDI) within its Fellowship election. Among the new Fellows, 41 per cent are women, the highest percentage ever elected. Additionally, Black, Asian and minority ethnic representation is 29 per cent, an 11 per cent increase from the previous year. The new Fellows hold positions at institutions across the UK, including in Edinburgh, Birmingham, Liverpool, Manchester, Sheffield, Nottingham and York.

Professor Morris added: It is also welcoming to note that this year's cohort is our most diverse yet, in terms of gender, ethnicity and geography. While this progress is encouraging, we recognise that there is still much work to be done to truly diversify our Fellowship. We remain committed to our EDI goals and will continue to take meaningful steps to ensure our Fellowship reflects the rich diversity of the society we serve."

The new Fellows will be formally admitted to the Academy at a ceremony on Wednesday 18 September 2024.

The Academy of Medical Sciences is the independent, expert body representing the diversity of medical science in the UK. Its mission is to advance biomedical and health research and its translation into benefits for society. The Academy's elected Fellows are the most influential scientists in the UK and worldwide, drawn from the NHS, academia, industry and the public service.

About the Academy of Medical SciencesThe Academy of Medical Sciences is the independent, expert voice of biomedical and health research in the UK. Our Fellowship comprises the most influential scientists in the UK and worldwide, drawn from the NHS, academia, industry, and the public service. Our mission is to improve the health of people everywhere by creating an open and progressive research sector. We do this by working with patients and the public to influence policy and biomedical practice, strengthening UK biomedical and health research, supporting the next generation of researchers through funding and career development opportunities, and working with partners globally.

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May: academy-medical-sciences | News and features - University of Bristol

Universal tool for tracking cell-to-cell interactions – ASBMB Today

One of the fundamental goals of basic biology is understanding how diverse cell types work in concert to form tissues, organs, and organ systems. Recent efforts to catalog the different cell types in every tissue in our bodies are a step in the right direction, but only one piece of the puzzle. The great mystery of how those cells communicate with one another remains unsolved.

The LIPSTIC technology can track the physical interactions between cells, such as a dendritic cell activating T cells.

Now, a new paper in Nature describes uLIPSTIC, a tool capable of laying the groundwork for a dynamic map tracking the physical interactions between different cellsthe elusive cellular interactome. The authors have been perfecting the technology since 2018 and the latest iteration can in principle allow researchers to directly observe any cell-to-cell interaction in vivo.

With uLIPSTIC we can ask how cells work together, how they communicate, and what messages they transfer, says Rockefellers Gabriel D. Victora. Thats where biology resides.

Ever since single-cell mRNA sequencing came into its own, researchers have been scrambling to connect the dots and explain how diverse cells unite to form tissue. Several methods of cataloging cell-to-cell interactions have already emerged, but all have considerable shortcomings. Early efforts that involved direct observation under a microscope failed to retrieve interacting cells for further analysis; subsequent attempts leaned on advanced imaging techniques that intuit how cells might interact based on their structure and proximity to other cells. No approach captured true physical interactions and signal exchange between cell membranes.

Enter LIPSTIC, an innovative approach from the Victora lab that involved labeling cellular structures that touch when two cells make fleeting, kiss-and-run contact before parting ways. The labels ensured that, if one cell kissed another, it would leave a mark akin to a lipstick, enabling easy identification and quantification of physical interactions between cells.

Originally, the platform had narrow applications. Victora and colleagues designed LIPSTIC to record a very specific kind of cell-to-cell interaction between T cells and B cells, a major focus of their lab. Other researchers, however, began clamoring for a version of LIPSTIC that would work on other cellular interactions too. We could have tailored a LIPSTIC for every type of interaction, Victora says. But why not try to make a universal version, instead?

In the original version of LIPSTIC, a donor cell uses an enzyme borrowed from bacteria to place a labeled peptide tag onto the surface of an acceptor cell upon contactthe biochemical equivalent of applying lipstick to one cell and looking for a kiss print on another. That method required knowing exactly how the kiss would occur, identifying molecules the donor cell uses to interact with recipient cells and painstakingly forcing the tags onto those molecules. But over time the team discovered that dousing the cells with a high volume of enzyme and its target would ensure that any interaction that one cell had with another cell would be tracked just as efficiently.

If you cram partner cells with enough enzyme and target, you can make any any cell pair capable of LISPTIC labeling without needing to know in advance what molecules these cells will use for their interaction, Victora says.

The result was uLIPSTIC, a universal platform not bound by foreknowledge of molecules, ligands, or receptors. Scientists can now theoretically smear uLIPSTIC on any cell, without preconceived notions of how it would interact with its environment, and observe physical cell-to-cell interactions. To demonstrate the power of the platform, the team showed that uLIPSTIC could expand beyond LIPSTICs narrow repertoire of B cells and T cells to track how dendritic cells kickstart the bodys immune response against tumors and food allergens.

The reception to uLIPSTIC has been great, says Sandra Nakandakari-Higa, a PhD student in the Victora lab and lead author on the paper. Were already getting a lot of inquiries from other labs about how they can adapt our system to their models.

The team hopes to eventually use uLIPSTIC to discover the receptor-ligand pairs key to cellular interactions, in an effort to better understand how cells unite into tissue at the molecular level. Eventually, the team envisions uLIPSTIC as a key tool in the effort to generate comprehensive atlases describing how cells interact to form tissuea key to the long-awaited interactome.

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Universal tool for tracking cell-to-cell interactions - ASBMB Today

Biogen joins immunology wave with $1.15 billion acquisition of HI-Bio – STAT

Biogen is joining the industrys fervor over immune and inflammatory disease drug development with a new acquisition.

The Cambridge, Mass., drugmaker announced Wednesday that it will acquire Human Immunology Biosciences, or HI-Bio, for $1.15 billion and up to $650 million in additional payments if certain milestones are met.

HI-Bio, which is based in San Francisco, is developing therapies for immune-mediated diseases like primary membranous nephropathy and IgA nephropathy, both of which impact kidney function. The startups lead drug, felzartamab, is a monoclonal antibody that selectively depletes CD38+ and natural killer cells in the hopes of alleviating the diseases effects. It has already completed Phase 2 studies.

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Biogen joins immunology wave with $1.15 billion acquisition of HI-Bio - STAT

Biogen Boosts Immunology Portfolio with $1.8 Billion Acquisition of HI-Bio – BioPharm International

Biogens acquisition of HI-Bio includes lead investigational mAb, felzartamab, under development for treating a range of immune-mediated diseases.

Biogen announced on May 22, 2024 that it has entered into a definitive agreement to acquire Human Immunology Biosciences (HI-Bio), a privately-held US-based clinical-stage biotechnology company focused on targeted therapies for severe immune-mediated diseases (IMDs). Under the agreement, Biogen will pay $1.15 billion upfront and up to $650 million in potential milestone payments. The transaction is expected to close in the third quarter of 2024, pending necessary regulatory approvals and customary closing conditions.

With the acquisition, Biogen gains felzartamab, HI-Bios lead asset. Felzartamab is a fully human anti-CD38 monoclonal antibody (mAb) that has been shown to selectively deplete CD38+ cells in clinical studies. This selective depletion includes plasma cells and natural killer (NK) cells. This action may allow for additional applications that improve clinical outcomes in a broad range of immune-mediated diseases, Biogen stated in a company press release.

We believe this late-stage asset, which has demonstrated impact on key biomarkers and clinical endpoints in three renal diseases with serious unmet needs, is a strategic addition to the Biogen portfolio as we continue to augment our pipeline and build on our expertise in immunology, said Priya Singhal, MD, head of Development at Biogen, in the press release. We look forward to welcoming HI-Bio employees into Biogen and, together, working to advance potential therapies for patients with rare immune diseases with high unmet need.

FDA has granted felzartamab breakthrough therapy designation and orphan drug designation for development in treating primary membranous nephropathy (PMN). The mAb has also received orphan drug designation for treating antibody-mediated rejection (AMR) in kidney transplant recipients. Felzartamab has completed Phase II studies in PMN and AMR and remains in ongoing Phase II studies in immunoglobulin A nephropathy (IgAN). HI-Bio plans to advance the mAb to Phase III studies in each indication; the company is presenting two abstracts at the European Renal Association Congress in Stockholm, which is occurring May 2326, 2024. HI-Bios presentation includes complete Phase II data from the AMR study in kidney transplant patients and interim data from the Phase II IgAN study, according to the press release. Studies have also generated clinical data for felzartamab in the AMR, PMN, and IgAN indications.

With its deep development and commercialization capabilities, Biogen is in a position to accelerate the development of new medicines, including felzartamab, for patients with severe immune-mediated diseases, said Travis Murdoch, MD, chief executive officer of HI-Bio, in the release. We are excited to combine the HI-Bio teams expertise with Biogens global footprint.

In addition to the felzartamab lead program, HI-Bios pipeline consists of izastobart/HIB210, an anti-C5aR1 antibody currently in a Phase I trial. This candidate has the potential for continued development in a range of complement-mediated diseases. HI-Bio also has discovery-stage mast cell programs with potential application in a range of immune-mediated diseases.

The treatment of IMDs has largely benefitted from the evolution of targeted biologic therapies. Targeted biologics have demonstrated efficacy, speed of onset, and tolerability. Whats more, research efforts have shown that clinically unrelated immune-mediated inflammatory conditions can share similar immune dysregulation. This discovery has since led to a shift in way IMDs and other inflammatory disorders are managed (1).

1. Kuek, A.; Hazleman, B. L.; Ostr A. J. Immune-Mediated Inflammatory Diseases (IMIDs) and Biologic Therapy: A Medical Revolution. Postgrad Med J. 2007, 83 (978), 251260. DOI: 10.1136/pgmj.2006.052688

Source: Biogen

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Biogen Boosts Immunology Portfolio with $1.8 Billion Acquisition of HI-Bio - BioPharm International

Biogen Buys Desired Growth In Immunology With $1.15bn Hi-Bio Deal – Scrip

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Biogen Buys Desired Growth In Immunology With $1.15bn Hi-Bio Deal - Scrip

Biogen to expand immunology and rare disease portfolio with $1.8bn HI-Bio acquisition – PMLiVE

Biogen has announced it has entered into a definitive agreement to acquire Human Immunology Biosciences (HI-Bio) in a deal worth more than $1.8bn, gaining access to its lead asset to improve a range of immune-mediated and rare diseases. Anticipated to close in the third quarter of 2024, the acquisition will expand Biogens immunology portfolio while also addressing the serious unmet needs of three renal diseases. Immune-mediated diseases result from an abnormal immune system response, which causes the immune system to mistakenly target the body and create an inflammatory response that causes damage. Under the terms of the agreement, HI-Bio will receive an upfront payment of 1.15bn and will be eligible for additional payments of up to $650m contingent on certain development milestones. As part of the deal, Biogen will gain access to HI-Bios lead asset, felzartamab, an investigational fully human anti-CD38 monoclonal antibody that has been shown to selectively deplete CD38+ cells in a broad range of immune-mediated diseases, as well as its izastobart/HIB210, an anti-C5aR1 antibody in development to treat a range of complement-mediated diseases. Furthermore, Biogen aims to establish a San Francisco Bay Area team focused on expanding efforts in immune-mediated diseases. Felzartamab has already received Breakthrough Therapy Designation and Orphan Drug Designation (ODD) from the US Food and Drug Administration to treat primary membranous nephropathy and has received ODD to treat antibody-mediated rejection in kidney transplant recipients. In addition, phase 2 studies have been completed in both indications and remain ongoing in IgA nephropathy, a chronic kidney disease, with plans to advance each indication to phase 3. Priya Singhal, head of development, Biogen, commented: We believe this late-stage asset is a strategic addition to the Biogen portfolio as we continue to augment our pipeline and build on our expertise in immunology. We look forward to advancing potential therapies for patients with rare immune diseases with high unmet need. In July 2023, Biogen entered into a definitive agreement to acquire rare disease specialist Reata Pharmaceuticals in a deal worth approximately $7.3bn to gain access to the biotechs Skyclarys (omaveloxolone), the only US-approved treatment for Friedreichs ataxia, a rare and inherited neurologic disorder.

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Biogen to expand immunology and rare disease portfolio with $1.8bn HI-Bio acquisition - PMLiVE