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Autolus Therapeutics plc, a clinical-stage biopharmaceutical company, develops T cell therapies for the treatment of cancer and autoimmune diseases. The company's clinical-stage programs include obecabtagene autoleucel (AUTO1), a CD19-targeting programmed T cell investigational therapy that is in Phase 1b/2 clinical trial for the treatment of adult ALL; AUTO1/22, which is in a Phase 1 clinical trial in pediatric patients with relapsed or refractory ALL; AUTO4, a programmed T cell investigational therapy for the treatment of peripheral T-cell lymphoma targeting TRBC1 and TRBC2; AUTO6NG, a programmed T cell investigational therapy targeting GD2 in development for the treatment of neuroblastoma; and AUTO8, a product candidate to treat multiple myeloma. It also focuses on developing AUTO5, a preclinical TRBC2 programmed T cell product candidate for the treatment of peripheral T-cell lymphoma. The company was incorporated in 2014 and is headquartered in London, the United Kingdom.

Genfit S.A., a biopharmaceutical company, discovers and develops drug candidates and diagnostic solutions for metabolic and liver-related diseases. The company develops Elafibranor, which is in Phase III clinical trial to treat patients with primary biliary cholangitis. It also engages in the development of NIS4 technology for the diagnosis of nonalcoholic steatohepatitis (NASH) and fibrosis; VS-01 for the treatment of Urea Cycle Disorder (UCD) and Organic Acidemia Disorder (OAD); GNS561, which is in Phase 1b/2 trial to treat patients with cholangiocarcinoma (CCA); and VS-01-ACLF and Nitazoxanide (NTZ), which is in Phase 1 trial to treat acute-on-chronic liver failure, as well as VS-02-HE, which is in preclinical trial for the treatment of Reduction of Hyperammonemia and the Stabilization of Blood Ammonia. The company has a licensing agreement with Labcorp for the commercialization of NASHnext, a blood-based molecular diagnostic test; and Genoscience Pharma to develop and commercialize the investigational treatment GNS561 for CCA. The company was incorporated in 1999 and is headquartered in Loos, France.

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.

Solid Biosciences Inc., a life science company, develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a gene transfer candidate for the treatment of Duchenne muscular dystrophy; and SGT-501 to treat Catecholaminergic polymorphic ventricular tachycardia. It is also developing AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy; SGT-601 to treat a rare cardiac disease characterized by mutations in the gene that codes for cardiac troponin T protein, which helps coordinate contraction of the heart muscle; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, a cardiac splicing factor that regulates alternative splicing, and codes for RNA binding motif protein 20; and AVB-202-TT to treat Friedreich's ataxia, as well as other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne; and a license agreement with the University of Washington, University of Missouri, and University of Florida. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.

Voyager 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.