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bluebird bio, Inc., a biotechnology company, researches, develops, and commercializes gene therapies for severe genetic diseases. Its product candidates for severe genetic diseases include ZYNTEGLO (betibeglogene autotemcel) for the treatment of transfusion-dependent ß-thalassemia; lovotibeglogene autotemcel for the treatment of sickle cell disease (SCD); and SKYSONA (elivaldogene autotemcel) to treat cerebral adrenoleukodystrophy. The company's clinical development programs include HGB-205, HGB-206, and HGB-210 to evaluate the safety and efficacy of lovo-cel in the treatment of patients with SCD; and HGB-204, HGB-205, HGB-207, and HGB-212 to evaluate the safety and efficacy of beti-cel in the treatment of patients with ß-thalassemia. It has license agreement with Orchard Therapeutics Limited. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was incorporated in 1992 and is headquartered in Somerville, Massachusetts.

Audentes Therapeutics, Inc., a clinical stage biotechnology company, focuses on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. The company is developing AT132, which is in Phase I/II clinical studies for the treatment of X-linked myotubular myopathy (XLMTM); AT342 that is in Phase I/II clinical studies to treat crigler-najjar syndrome; AT845, which is in preclinical studies for the treatment of pompe disease; and AT307 to treat CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. It is also developing vectorized antisense treatments for the treatment of duchenne muscular dystrophy and myotonic dystrophy type 1. The company has a collaborative development agreement with Genethon to research, develop, manufacture, and commercialize products for the treatment of XLMTM; and a license and collaboration agreement with the University of Pennsylvania to research, develop, sell, and import licensed products for the treatment of crigler-najjar. Audentes Therapeutics, Inc. was founded in 2012 and is headquartered in San Francisco, California.

Denali Therapeutics Inc., a biopharmaceutical company, develops a portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage diseases in the United States. The company's transport vehicle (TV)-enabled programs include DNL310 ETV, an IDS enzyme replacement therapy program for MPS II; TAK-594/DNL593 which is in Phase 1/II for frontotemporal dementia-granulin; DNL126 program for MPS IIIA; and DNL622 for MPS I, as well as other preclinical programs that target amyloid beta and HER2. Its brain-penetrant small molecule programs comprise BIIB122/DNL151 LRRK2 inhibitor program for Parkinson's disease; SAR443820/DNL788 RIPK1 inhibitor program for CNS disease; DNL343 eIF2B Activator program for amyotrophic lateral sclerosis; and SAR443122/DNL758 RIPK1 inhibitor program for peripheral inflammatory diseases. It also provides early stage program include TAK-594/DNL593 program for FTD-GRN; DNL126 program for MPS IIIA, a Sanfilippo Syndrome A; DNL622 for MPS I which is Hurler syndrome; Antibody Transport Vehicle Amyloid beta program; Oligonucleotide Transport Vehicle platform, a novel class of biotherapeutics to address the root cause of diseases through modulation of gene expression; and other TV-enabled discovery programs. The company has collaboration agreements with Biogen MA Inc. and Biogen International GmbH; Genzyme Corporation; Takeda Pharmaceutical Company Limited; F-star Gamma Limited, F-star Biotechnologische Forschungs-und Entwicklungsges m.b.H, and F-star Biotechnology Limited; and Genentech, Inc. The company was formerly known as SPR Pharma Inc. and changed its name to Denali Therapeutics Inc. in March 2015. Denali Therapeutics Inc. was incorporated in 2013 and is headquartered in South San Francisco, California.

Fate Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops programmed cellular immunotherapies for cancer and immune disorders worldwide. The company's chimeric antigen receptor (CAR)-targeted NK and T-cell product candidates include FT576 to treat multiple myeloma, and FT522, to treat lymphoma and autoimmune disorders. Its CAR T-cell programs include FT819 to treat hematologic malignancies and solid tumors, and FT825 to treat solid tumors. The company has a collaboration and option agreement with Ono Pharmaceutical Co. Ltd. for the development and commercialization of off-the-shelf, iPSC-derived CAR T-cell product candidates for the treatment of cancer. Fate Therapeutics, Inc. was incorporated in 2007 and is headquartered in San Diego, California.

Immunovant, Inc., a clinical-stage biopharmaceutical company, develops monoclonal antibodies for the treatment of autoimmune diseases. It develops batoclimab, a novel fully human monoclonal antibody that target the neonatal fragment crystallizable receptor for the treatment of myasthenia gravis, thyroid eye disease, chronic inflammatory demyelinating polyneuropathy, and Graves diseases, as well as warm autoimmune hemolytic anemia. The company is headquartered in New York, New York. Immunovant, Inc. operates as a subsidiary of Roivant Sciences Ltd.