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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.

Krystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, and commercializes genetic medicines for patients with rare diseases in the United States. It commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). The company also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.

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.

Vericel Corporation, a commercial-stage biopharmaceutical company, engages in the research, development, manufacture, and distribution of cellular therapies for sports medicine and severe burn care markets in North America. The company markets autologous cell therapy products comprising MACI, an autologous cultured chondrocytes on porcine collagen membrane for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; Epicel, a permanent skin replacement humanitarian use device for the treatment of adult and pediatric patients with deep-dermal or full-thickness burns; and NexoBrid, a biological orphan product for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was incorporated in 1989 and is headquartered in Cambridge, 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.