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Homology is based on groundbreaking science that harnesses the naturally occurring process of homologous recombination. This non-nuclease-based approach offers clear advantages in its precision, efficiency and on-target in vivo editing of genetic mutations. Homology obtained an exclusive worldwide license to this technology platform, which is based on the pioneering research of Saswati Chatterjee, Ph.D., Professor of Virology at the Beckman Research Institute at the City of Hope in California, member of the Recombinant DNA Advisory Committee (RAC) to the Office of the Director, National Institutes of Health (NIH) and former charter member of the Therapeutic Approaches to Genetic Diseases Study Section of the NIH. Dr. Chatterjee and her team led the first adeno-associated virus (AAV) vector-mediated gene transfer studies into human hematopoietic stem cells and subsequently identified and isolated a series of naturally-occurring AAVs from human CD34+ cells.
Arkuda Therapeutics is a biotechnology company applying insights into lysosomal biology to drive the development of medicines to change the trajectory of neurodegenerative disease. Arkuda`s lead program ARKD-104 aims to correct progranulin deficiency and lysosomal dysfunction in patients with GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. The company is further exploring the therapeutic potential of its molecules in other neurodegenerative diseases where genetic links to dysfunction in progranulin biology have been established, including Alzheimer`s Disease and Parkinson`s Disease. Arkuda is backed by leading investors including Atlas Venture, Cormorant Asset Management, Pfizer Ventures, funds managed by Tekla Capital Management LLC, Mission BioCapital, and Eli Lilly and Company.
BIA Separations is a Wilmington, DE-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
PureTech is a science-driven healthcare company, seeking to solve some of today`s toughest health challenges through disruptive approaches. PureTech has a pipeline of 12 operating companies, seven of which are “growth stage” with external validation including strategic partnerships, outside funding, proof-of-concept or peer review in prestigious scientific journals. PureTech is problem-focused and solution-agnostic, looking beyond traditional disciplines and approaching healthcare problems from different perspectives. Focusing on areas of significant unmet medical need, PureTech evaluates more than 650 ideas per year, prioritizing, selecting and testing what it believes to be the most scientifically and commercially promising concepts to advance. PureTech’s leading team and board, along with an advisory network of more than 50 experts across multiple disciplines, gives PureTech access to potentially groundbreaking science and technological innovations.
NeuroPace is a Mountain View, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.