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.
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..
Renovacor
NYSE:RCORRenovacor, Inc., a biotechnology company, focuses on delivering various precision therapies to enhance the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. It primarily focuses on the treatment of BCL2-associated athanogene 3 (BAG3) mutation-associated dilated cardiomyopathy (DCM). The company's lead product candidate is REN-001, a recombinant adeno-associated virus 9-based gene therapy designed to deliver a functional BAG3 gene to augment BAG3 protein levels in cardiomyocytes, and slow or halt progression of BAG3 DCM. It is also developing a pipeline of BAG3-associated gene therapies for diseases with high unmet medical need associated with mutations in the BAG3 gene and mechanistically linked to BAG3's expression and function. The company was founded in 2013 and is based in Greenwich, Connecticut.
Talaris Therapeutics
NASDAQ:TALSTalaris Therapeutics, Inc. operates as a late-clinical stage cell therapy company in the United States. The company engages in developing a method of allogeneic hematopoietic stem cell transplantation to transform the standard of care in solid organ transplantation and severe autoimmune diseases, as well as severe blood, immune, and metabolic disorders. Its lead product candidate is FCR001, a novel allogeneic cell therapy that is in Phase II trial for living donor kidney transplant patients. The company is also developing FCR001 for the patients with a severe form of scleroderma. Talaris Therapeutics, Inc. was founded in 1988 and is headquartered in Wellesley, Massachusetts.