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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.
Aquatic Biotechnologies Inc is a Camarillo, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The companys lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, renal, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
Abeona Therapeutics (Nasdaq: ABEO) is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. The Company`s multi-platform expertise across the manufacture, delivery, development, and discovery of novel gene and cell therapies has it uniquely positioned for success. Underpinning the Company`s robust pipeline is its fully-operational manufacturing facility producing therapies and vectors for preclinical and clinical studies. Abeona is also developing the AIM™ Vector Platform: 100+ next-generation AAV capsids for delivering gene therapies targeting a wide range of organs and multiple routes of delivery. A robust and diverse pipeline is led by a novel gene-corrected cell therapy poised to enter Phase 3 in mid-2019 and complemented by one-time gene therapy candidates across four lysosomal storage diseases. Several preclinical discoveries are led by an emerging program in cystic fibrosis that uses the AIM vector platform and a capsid that has shown potential across inherited and acquired retinal diseases.