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.
Open MGTX dashboard ↗Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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