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Atara Biotherapeutics, Inc. develops therapies for patients with solid tumors, hematologic cancers, and autoimmune diseases in the United States. Its lead product includes Tab-cel (tabelecleucel), a T-cell immunotherapy program that is in Phase 3 clinical trials for the treatment of epstein-barr virus (EBV) driven post-transplant lymphoproliferative disease, as well as nasopharyngeal carcinoma. Its CAR T immunotherapy pipeline products include ATA2271 and ATA3271 to treat mesothelin; and ATA3219 for the treatment of B-cell malignancies, as well as ATA188 that is in Phase 2 clinical trials to treat multiple sclerosis. Atara Biotherapeutics, Inc. has a license agreement with Memorial Sloan Kettering Cancer Center; license, and research and development collaboration agreement with QIMR Berghofer Medical Research Institute; and strategic collaboration with H. Lee Moffitt Cancer Center. The company was incorporated in 2012 and is headquartered in Thousand Oaks, California.

AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Genfit S.A., a late-stage 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/2a trial to treat patients with cholangiocarcinoma (CCA); 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; CML-022; SRT-015, an ASK1 inhibitor targets the inhibition of cellular apoptosis, inflammation, and fibrosis. 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.

Proteon Therapeutics, Inc., a late-stage biopharmaceutical company, focuses on the development of pharmaceuticals to address the needs of patients with renal and vascular disease. Its lead product candidate is vonapanitase, an investigational drug intended to enhance hemodialysis vascular access outcomes. The company develops vonapanitase, a recombinant human elastase, which has completed Phase II and Phase III clinical trials for patients with chronic kidney disease. It is also evaluating vonapanitase in a Phase I clinical trial in patients with peripheral artery disease. The company was founded in 2001 and is based in Waltham, Massachusetts.