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Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The company`s non-viral platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses its proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bio`s manufacturing scale to hundreds of millions of doses to support their mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.
Bayer and CRISPR Therapeutics have entered into an agreement to create Casebia to discover, develop and commercialize new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart disease. CRISPR Therapeutics will contribute its proprietary CRISPR-Cas9 gene-editing technology and intellectual property, while Bayer will make available its protein engineering expertise and relevant disease know-how. It is the first long-term strategic partnership of its kind to make a substantial investment in the development of target delivery systems in an effort to bring systemic in vivo CRISPR-Cas9 gene editing technology applications to patients.
RootPath, Inc. is a Cambridge, MA-based early-stage biotechnology company focusing on next-generation immuno- and cell-therapy.
Navitor Pharmaceuticals, Inc., is a biopharmaceutical company developing novel medicines by targeting cellular nutrient signaling pathways. The companys proprietary drug discovery platform targets mTORC1, which responds to and integrates the cells response to nutrient availability and plays a key role in protein synthesis and cellular growth. Navitors therapeutics are designed to selectively modulate the cellular signals that are aberrant in disease processes caused by the dysregulation of mTORC1 activity to address a wide range of diseases, including metabolic, neurodegenerative, autoimmune and musculoskeletal diseases, as well as several rare disorders. The companys founding intellectual property is based on the groundbreaking discoveries related to the mTORC1 pathway and nutrient signaling mechanisms by Dr. David Sabatini at The Whitehead Institute for Biomedical Research. The company is backed by leading financial and corporate investors, including Polaris Partners, Atlas Venture, Johnson and Johnson Development Corporation, SR One and The Longevity Fund.
BioMedica, Inc. is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.