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Brainstorm 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.

Fennec Pharmaceuticals Inc., a biopharmaceutical company, develops product candidates for use in the treatment of cancer in the United States. Its lead product candidate is the Sodium Thiosulfate, which has completed the Phase III clinical trial for the prevention of cisplatin induced hearing loss or ototoxicity in children. The company was formerly known as Adherex Technologies Inc. and changed its name to Fennec Pharmaceuticals Inc. in September 2014. Fennec Pharmaceuticals Inc. was founded in 1996 and is based in Research Triangle Park, North Carolina.

Orchard 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, 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.

TScan Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops T cell receptor-engineered T cell (TCR-T) therapies for the treatment of patients with cancer in the United States. The company's lead product candidates include TSC-100 and TSC-101 that is in Phase I clinical trial for the treatment of patients with hematologic malignancies to eliminate residual disease and prevent relapse after allogeneic hematopoietic cell transplantation. It also develops TSC-200, TSC-201, TSC-203, and TSC-204, which are in Phase 1 clinical trial, for the treatment of solid tumors; and TSC-202 to treat solid tumors. In addition, the company develops vaccines for infectious diseases, such as SARS-CoV-2. It has collaborations with Novartis Institutes for BioMedical Research, Inc. To discover and develop novel TCR-T therapies; and Amgen Inc. to identify antigens recognized by T cells in patients with Crohn's disease using TargetScan, a proprietary target discovery platform. TScan Therapeutics, Inc. was incorporated in 2018 and is headquartered in Waltham, Massachusetts.