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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.

aTyr Pharma, Inc., a biotherapeutics company, engages in the discovery and development of medicines based on novel immunological pathways in the United States. Its lead therapeutic candidate is efzofitimod, a selective modulator of NRP2 that is in Phase III clinical trial for pulmonary sarcoidosis; and in Phase 1b/2a clinical trial for treatment of other interstitial lung diseases (ILDs), such as chronic hypersensitivity pneumonitis and connective tissue disease related ILD. The company is developing ATYR0101, a fusion protein derived from a domain of aspartyl-tRNA synthetase that is in preclinical development for the treatment of fibrosis; and ATYR0750, a domain of alanyl-tRNA synthetase for the treatment of liver disorders. It has collaboration and license agreement with Kyorin Pharmaceutical Co., Ltd. for the development and commercialization of efzofitimod for ILDs in Japan. aTyr Pharma, Inc. was incorporated in 2005 and is headquartered in San Diego, California.

Outlook Therapeutics, Inc., operates as a clinical-stage biopharmaceutical company, focuses on developing and commercializing monoclonal antibodies for various ophthalmic indications. Its lead product candidate is ONS-5010, an ophthalmic formulation of bevacizumab product candidate that is in Phase-III clinical trial for the treatment of wet age-related macular degeneration and other retina diseases. Outlook Therapeutics, Inc. has collaboration and license agreements with BioLexis Pte. Ltd. and Zhejiang Huahai Pharmaceutical Co., Ltd. The company was formerly known as Oncobiologics, Inc. and changed its name to Outlook Therapeutics, Inc. in November 2018. Outlook Therapeutics, Inc. was incorporated in 2010 and is based in Iselin, New Jersey.

Vigil Neuroscience, Inc., a clinical-stage biotechnology company, focuses on developing treatments for rare and common neurodegenerative diseases by restoring the vigilance of microglia, the sentinel immune cells of the brain. Its lead candidate is VGL101(Iluzanebart), a human monoclonal antibody agonist targeting human triggering receptor expressed on myeloid cells 2 and is in a Phase 2 trial in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare and fatal neurodegenerative disease. The company is also developing VG-3927, an orally-available small molecule TREM2 agonist to treat common neurodegenerative diseases associated with microglial dysfunction, with an initial focus on Alzheimer's disease in genetically defined subpopulations, which is being evaluated in a Phase 1 clinical trial. It has license agreement with Amgen Inc. to commercially develop, manufacture, use, distribute, and sell therapeutic products containing compounds that bind to TREM2. The company was incorporated in 2020 and is headquartered in Watertown, Massachusetts.