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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.

Precision BioSciences, Inc., an advanced gene editing company, develops in vivo gene editing therapies for gene edits, including gene elimination, insertion, and excision in the United States. The company offers ARCUS, a genome editing platform to DNA genome insertion, deletion, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; PBGENE-PMM for the treatment of m.3243 associated primary mitochondrial myopathy (PMM) which is expected to submit an IND and/or CTA. In addition, it develops PBGENE-NVS for sickle cell disease/beta thalassemia for insertion; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LL2 (insertion), a liver directed target; PBGENE-LL3, a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. The company has license and collaboration agreement with Caribou Biosciences, Inc.; license agreement with TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; development and license agreement with Eli Lilly and Company for the research and development of potential in vivo therapies for genetic disorders; Cellectis S.A.; iECURE, Inc. to develop ARCUS-based gene-insertion therapies; Duke University; and Novartis Pharma AG to discover and develop in vivo gene editing products. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.

Harpoon Therapeutics, Inc., a clinical-stage immunotherapy company, engages in the development of a novel class of T cell engagers that harness the power of the body's immune system to treat patients suffering from cancer and other diseases in the United States. The company develops tri-specific T cell activating construct (TriTAC) product candidate, including HPN328, which is in Phase I/II clinical trials for the treatment of small cell lung cancer and other Delta-like canonical Notch ligand 3-expressing tumors; and HPN217 that is in Phase I clinical trials for the treatment of multiple myeloma. Its preclinical stage product is HPN601 for the treatment of multiple solid tumor indications. It has a discovery collaboration and license agreement with AbbVie Biotechnology Ltd. to develop and commercialize products that incorporate its proprietary TriTAC platform technology together with soluble T cell receptors. The company was incorporated in 2015 and is headquartered in South San Francisco, California. As of March 11, 2024, Harpoon Therapeutics, Inc. operates as a subsidiary of Merck Sharp & Dohme LLC.

MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and xerostomia. Its programs in clinical development include Phase I/II clinical stage programs in achromatopsia, X-linked retinitis pigmentosa, and RPE65-deficiency; Phase I clinical trials for radiation-induced xerostomia; and Parkinson's program that has completed a Phase II trial. It has a research collaboration agreement with Janssen Pharmaceuticals, Inc. for the research, development, and commercialization of gene therapies for the treatment of inherited retinal disease. The company was incorporated in 2015 and is based in New York, New York.

TCR2 Therapeutics, Inc. is a clinical-stage cell therapy company, which engages in the development of biological drugs and engineering T-cells for cancer therapy. It is also involved in the research and collaboration with academic laboratories and industry partners in the field of T-cell immunology, cell therapy, gene editing, and process development. The company was founded by Patrick A. Baeuerle on May 29, 2015 and is headquartered in Cambridge, MA.