MeiraGTx Holdings plc Ordinary Shares
MeiraGTx Holdings plc, a clinical stage genetics medicines company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases; neurodegenerative diseases; and xerostomia. It is also developing AAV-AQP1 in Phase 2 trial for radiation-induced grade 2/3 radiation-induced xerostomia, as well as in Phase ½ trial for Sjogren's syndrome; AAV-GAD in Phase 2 trial for Parkinson's disease; AAV-RPE65 in Phase 2 trial for RPE65-associated retinal dystrophy; and AAV-CNGB3 and AAV-CNGA3, which are both in Phase 2 trial for achromatopsia, as well as offers AAV-AIPL1 for Leber congenital amaurosis (LCA) 4. In addition, the company's products under preclinical development include AAV-RDH12 for retinol dehydrogenase 12 mutation-associated retinal dystrophy; AAV-BBS10 for Bardet-Biedl syndrome (BBS) due to BBS10 mutations; AAV-RetGC for LCA type 1; and AAV-UPF1 for amyotrophic lateral sclerosis, as well as programs for Alzheimer's disease, and wet and dry neovascular age related macular degeneration. Further, it provides riboswitch platform for the delivery of metabolic peptides; and cell therapies for oncology, autoimmune disease, and long term intractable pain. The company has collaborations with Johnson & Johnson Innovative Medicine for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease; and Hologen Limited for the research, development, manufacture, and commercialization of AAV-GAD investigational gene therapy for the treatment of Parkinson's disease, AAV-BDNF investigational gene therapy for genetic obesity disorders, and other genetic medicines to the central nervous system. MeiraGTx Holdings plc was founded in 2015 and is based in New York, New York.
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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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