Alector, Inc. Common Stock
Alector, Inc., a late-stage clinical biotechnology company, develops therapies that is focused on counteracting the devastating progression of neurodegenerative diseases. The company's products under development include Latozinemab (AL001), an investigational human recombinant monoclonal antibody designed to elevate progranulin (PGRN), a protein encoded by the granulin (GRN) gene that regulates lysosomal function, neuronal survival, and inflammation in the brain. Its lead PGRN-elevating candidate is being developed for the potential treatment of frontotemporal dementia with a granulin gene mutation (FTD-GRN) which is caused by heterozygous loss-of-function (LOF) mutations in the GRN gene and results in PGRN haploinsufficiency and is in Phase 3 clinical trial. It also develops AL101/GSK4527226 that is in Phase 2 clinical trial for the treatment of neurodegenerative diseases, including Alzheimer's and Parkinson's diseases. In addition, the company's preclinical and research pipeline products include ADP037-ABC, a proprietary anti-amyloid beta (Aß) antibody paired with proprietary ABC for the treatment of AD; ADP050-ABC, a GCase replacement therapy paired with proprietary ABC for GBA gene mutation carriers with Parkinson's disease and Lewy body dementia; ADP056, a Reelin modulator designed to block tau pathology and promote synaptic function in AD; and ADP063-ABC and ADP064-ABC that are therapeutic candidates paired with proprietary ABC that target tau pathology in AD through distinct approaches. It has a strategic collaboration agreement with GlaxoSmithKline plc for the development and commercialization of progranulin-elevating monoclonal antibodies, including latozinemab and AL101 to treat neurodegenerative diseases. The company was founded in 2013 and is headquartered in South San Francisco, California.
Open ALEC 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.
Open RARE dashboard ↗