Brainstorm Cell Therapeutics
NASDAQ:BCLIBrainstorm Cell Therapeutics Inc., a biotechnology company, engages in the development and commercialization of autologous cellular therapies for the treatment of neurodegenerative diseases. The company, through its NurOwn proprietary cell therapy platform, leverages cell culture methods to induce autologous bone marrow-derived mesenchymal stem cells to secrete high levels of neurotrophic factors, modulate neuroinflammatory and neurodegenerative disease processes, promote neuronal survival, and enhance neurological function. It is developing NurOwn, which has completed Phase III clinical trial for the treatment of amyotrophic lateral sclerosis; Phase II clinical trial for the treatment of progressive multiple sclerosis; and for the treatment of alzheimer's disease, as well as for other neurodegenerative diseases. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004. Brainstorm Cell Therapeutics Inc. was incorporated in 2000 and is headquartered in New York, New York.
BiondVax Pharmaceuticals
NASDAQ:BVXVBiondVax Pharmaceuticals Ltd., a development stage biopharmaceutical company, focuses on developing, manufacturing, and commercializing products for the prevention and treatment of infectious and autoimmune diseases, and other illnesses in Israel. The company has licensing and collaboration agreement with Max Planck Society and University Medical Center Göttingen for the development and commercialization of COVID-19 nanosized antibody (NanoAb); and development and commercialization of NanoAbs for various other disease indications. The company was incorporated in 2003 and is headquartered in Jerusalem, Israel.
Freeline Therapeutics
NASDAQ:FRLNFreeline Therapeutics Holdings plc, a clinical-stage biotechnology company, develops transformative adeno-associated virus (AAV) vector-mediated gene therapies. It develops FLT180a for the treatment of hemophilia B.; FLT201 for the treatment of Type 1 Gaucher disease; and FLT190 for the treatment of Fabry disease. in addition, it has research programs for systemic gene therapy. The company was founded in 2015 and is headquartered in Stevenage, the United Kingdom.
Orchard Therapeutics
NASDAQ:ORTXOrchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..
Voyager Therapeutics
NASDAQ:VYGRVoyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.