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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.
Brain diseases collectively represent one of the greatest challenges of our generation, affecting upwards of 1.5 billion people. However, treatments for many of the most prevalent brain disorders have been dominated by a single class of drugs, such as serotonin targeting antidepressants for MDD, leaving patients with a high degree of unmet medical need given the lack of diverse treatment options and mechanisms of action. We are taking a fundamentally different approach to the way treatments for brain diseases are developed. Our pipeline is comprised of programs pursuing therapeutically relevant targets implicated in Central Nervous System (CNS) diseases, each targeting a novel mechanism of action with best-in-class pharmacology. Our work is supported by our Precision Toolbox, which integrates a suite of translational, clinical, and computational tools to generate insights that can enable precision medicine approaches. Neumora is relentless in its commitment to discovering, developing and commercializing novel therapies for the 1.5 billion people living with brain diseases.
Progenika is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Federation Bio is a biotechnology company pioneering a novel approach to create potent, durable and safe cell therapies. The company`s first-in-class platform combines the power of genetic engineering and synthetic consortium design to control systemic immune responses and broad metabolic functions. Federation Bio`s pipeline addresses a range of serious illnesses from metabolic disorders to metastatic cancer. The company`s lead program is in enteric hyperoxaluria, a serious renal condition that affects more than 250,000 Americans and for which there are currently no approved therapies.
Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA™) platform, in combination with Cabaletta`s proprietary technology, utilizes CAAR T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company`s lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please visit our website ( DesCAARTes™ Phase 1 Trial ). The Company`s lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis.