Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company, develops novel cell therapies for neurological and ophthalmic conditions in the United States and internationally. The company develops OpRegen, an allogeneic retinal pigment epithelium cell replacement therapy, which is in Phase 2a clinical trial for the treatment of the age-related macular degeneration; OPC1, an allogeneic oligodendrocyte progenitor cell therapy that is in Phase 1/2a multicenter clinical trial for the treatment of cervical spinal cord injuries; and VAC2, which is in Phase I clinical trial to treat non-small cell lung cancer. It also offers ReSonance (ANP1), an allogeneic auditory neuron progenitor cell transplant, which is in preclinical development for the treatment of sensorineural hearing loss; and PNC1, an allogeneic photoreceptor cell transplant, which is in preclinical development for the treatment of vision loss due to photoreceptor dysfunction or damage. In addition, the company is developing RND1, a cell transplant program for an undisclosed indication through a gene editing collaboration, engages in the research and development of therapeutic products for retinal diseases, neurological diseases, and disorders and oncology. Lineage Cell Therapeutics, Inc. has a collaboration with Immunomic Therapeutics, Inc., for the treatment of glioblastoma multiforme. The company was formerly known as BioTime, Inc. and changed its name to Lineage Cell Therapeutics, Inc. in August 2019. Lineage Cell Therapeutics, Inc. was incorporated in 1990 and is headquartered in Carlsbad, California.
Open LCTX 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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