Alector
NASDAQ:ALECAlector, Inc., a clinical stage biopharmaceutical company, develops therapies for the treatment of neurodegeneration diseases. Its products include AL001, an immune activity in the brain with genetic links to multiple neurodegenerative disorders, which is in Phase III clinical trial for the treatment of frontotemporal dementia, Alzheimer's, Parkinson's, and amyotrophic lateral sclerosis diseases; and AL101 that is in Phase I clinical trial for the treatment of neurodegenerative diseases, including Alzheimer's and Parkinson's diseases. The company also offers AL002, a product candidate that is in Phase II clinical trial for the treatment of Alzheimer's disease. Alector, Inc. has a collaboration agreement with Adimab, LLC for the research and development of antibodies; and a strategic collaboration agreement with GlaxoSmithKline plc for the development and commercialization of monoclonal antibodies, such as AL001 and AL101 to treat neurodegenerative diseases. The company was founded in 2013 and is headquartered in South San Francisco, California.
Audentes Therapeutics
NASDAQ:BOLDAudentes Therapeutics, Inc., a clinical stage biotechnology company, focuses on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. The company is developing AT132, which is in Phase I/II clinical studies for the treatment of X-linked myotubular myopathy (XLMTM); AT342 that is in Phase I/II clinical studies to treat crigler-najjar syndrome; AT845, which is in preclinical studies for the treatment of pompe disease; and AT307 to treat CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. It is also developing vectorized antisense treatments for the treatment of duchenne muscular dystrophy and myotonic dystrophy type 1. The company has a collaborative development agreement with Genethon to research, develop, manufacture, and commercialize products for the treatment of XLMTM; and a license and collaboration agreement with the University of Pennsylvania to research, develop, sell, and import licensed products for the treatment of crigler-najjar. Audentes Therapeutics, Inc. was founded in 2012 and is headquartered in San Francisco, California.
Editas Medicine
NASDAQ:EDITEditas Medicine, Inc., a clinical stage genome editing company, focuses on developing transformative genomic medicines to treat a range of serious diseases. It develops a proprietary gene editing platform based on CRISPR technology. The company develops EDIT-101, which is in Phase 1/2 BRILLIANCE trial for Leber Congenital Amaurosis; and reni-cel, a clinical development gene-edited medicine to treat sickle cell disease and transfusion-dependent beta-thalassemia. In addition, the company is developing alpha-beta T cells for solid and liquid tumors; and gamma delta T cell therapies to treat cancer. It has a research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; strategic alliance and option agreement with Allergan Pharmaceuticals International Limited. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was incorporated in 2013 and is based in Cambridge, Massachusetts.
Taysha Gene Therapies
NASDAQ:TSHATaysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN7 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 for the treatment of for SLC13A5 deficiency; TSHA-113 for the treatment of tauopathies; TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center. Taysha Gene Therapies, Inc. was incorporated in 2019 and is headquartered in Dallas, Texas.