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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.

Cullinan Oncology, Inc., a biopharmaceutical company, focuses on developing oncology therapies for cancer patients in the United States. The company's lead program comprises CLN-619, a monoclonal antibody that is in Phase I clinical trial for the treatment of solid tumors. Its development portfolio also includes CLN-049, a humanized bispecific antibody that is in Phase I clinical trial for the treatment of acute myeloid leukemia or myelodysplastic syndrome; CLN-418, a human bispecific immune activator that is in Phase 1 clinical trial for the treatment of multiple solid tumors; and Zipalertinib, a bioavailable small-molecule for treating patients with non-small cell lung cancer. In addition, the company's development products comprise CLN-617, a fusion protein for the treatment of solid tumors; and CLN-978, a T cell engaging antibody for the treatment relapsed/refractory B-cell non-Hodgkin lymphoma. It has license and collaboration agreement with Adimab, LLC to discover and/or optimize antibodies; Harbour BioMed US Inc. for the development, manufacturing, and commercialization of CLN-418; and co-development agreement with Taiho Pharmaceutical Co., Ltd to develop Zipalertinib. Cullinan Oncology, Inc. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

4D Molecular Therapeutics, Inc., a clinical-stage biotherapeutics company, develops genetic medicines using its therapeutic vector evolution platform in the Netherland and the United States. The company develops a portfolio of genetic medicine product candidates focuses in three therapeutic areas for ophthalmology, cardiology, and pulmonology. Its product pipeline includes 4D-150, which is in phase 1/2 clinical trial for the treatment of wet age-related macular degeneration; and is in phase 1/2 clinical trial for the treatment of diabetic macular edema. The company develops 4D-125, which is in phase 1/2 clinical trial for the treatment of x-linked retinitis pigmentosa; 4D-110, which is in phase 1/2 clinical trial for the treatment of choroideremia; 4D-710, which is in phase 1/2 clinical trial for the treatment cystic fibrosis lung disease; and 4D-310 to treat fabry disease cardiomyopathy and is in phase 1/2 clinical trial. In addition, its product candidates comprises 4D-175, which is in pre-clinical development for the treatment for geographic trophy; and 4D-725 to treat alpha-1 antitrypsin deficiency lung disease and is in pre-clinical development. The company has collaboration and licensing agreements with Arbor Biotechnologies, Inc.; Astellas Gene Therapies, Inc; uniQure biopharma B.V.; and Cystic Fibrosis Foundation. 4D Molecular Therapeutics, Inc. was founded in 2013 and is headquartered in Emeryville, California.

Omega Therapeutics, Inc. operates as a clinical-stage biotechnology company. The company's OMEGA platform enables control of fundamental epigenetic processes to correct the root cause of disease by restoring aberrant gene expression to a range without altering native nucleic acid sequences. It also develops OTX-2002 for hepatocellular carcinoma; OTX-2101 for non-small cell lung cancer; omega epigenomic controllers (OEC) for inflammatory lung diseases, such as neutrophilic asthma, acute respiratory distress syndrome, dermatological, oncology, and rheumatological indications; OEC candidates for idiopathic pulmonary fibrosis; liver regeneration medicines; and OEC candidates for patients with diabetes and other conditions to treat corneal epithelial injury. In addition, the company develops OEC candidates for the treatment of alopecia, a disorder characterized by patches of non-scarring hair loss affecting the scalp and body. Omega Therapeutics, Inc. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.