REGENXBIO Inc.
REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.
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Sana Biotechnology, Inc., a biotechnology company, focuses on utilizing engineered cells as medicines in the United States. It develops ex vivo and in vivo cell engineering platforms for various therapeutic areas with unmet treatment needs, including type 1 diabetes, B cell mediated autoimmune diseases, and oncology. The company's product pipeline consist of UP421, a HIP-modified allogeneic primary islet cell product, which is in phase 1 clinical trial to treat type 1 diabetes; SC451, a HIP-modified, stem cell derived pancreatic islet cell therapy, which is in preclinical trial for the treatment of type 1 diabetes; SC291, a HIP-modified allogeneic CD19-dirtected allogeneic CAR T cell product candidate which is in phase 1 clinical trial to treat systemic lupus erythematosus and ANCA-associated vasculitis, as well as in preclinical trial to treat other indications and b-cell mediated autoimmune disease; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. It also develops SC262, a hypoimmune-modified CD22-directed allogeneic CAR T program, which is in phase 1 to treat non-Hodgkin's lymphoma; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. The company has an option and license agreement with Beam Therapeutics Inc. for use of Beam's proprietary CRISPR Cas12b nuclease editing technology to research, develop, and commercialize engineered cell therapy products; and a license agreement with Harvard College to access certain intellectual property for the development of hypoimmune-modified cells. The company was formerly known as FD Therapeutics, Inc. and changed its name to Sana Biotechnology, Inc. in September 2018. Sana Biotechnology, Inc. was incorporated in 2018 and is headquartered in Seattle, Washington.
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