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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.

C4 Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutic candidates to degrade disease-causing proteins for the treatment of cancer, neurodegenerative conditions, and other diseases. Its lead product candidate is CFT7455, an orally bioavailable MonoDAC degrader of protein that is in Phase 1/2 trial targeting IKZF1 and IKZF3 for multiple myeloma and non-Hodgkin lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma, currently under Phase 1/2 clinical trials. The company is also developing CFT1946, an orally bioavailable BiDAC degrader targeting V600X mutant BRAF to treat melanoma, non-small cell lung cancer (NSCLC), colorectal cancer, and other solid malignancies, currently under Phase 1/2 clinical trials; and CFT8919, an orally bioavailable, allosteric, and mutant-selective BiDAC degrader of epidermal growth factor receptor, or EGFR, with an L858R mutation in NSCLC. It has strategic collaborations with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; Biogen MA, Inc.; Betta Pharmaceuticals, Co., Ltd.; and Merck Sharp & Dohme, LLC, as well as Calico Life Sciences LLC. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.

Kodiak Sciences Inc., a clinical stage biopharmaceutical company, researches, develops, and commercializes therapeutics to treat retinal diseases. Its lead product candidate is tarcocimab tedromer (KSI-301), an anti-vascular endothelial growth factor antibody biopolymer that is in Phase IIb/III clinical study to treat wet age-related macular degeneration (AMD), as well as Phase III clinical study for the treatment of diabetic macular edema, naïve macular edema due to retinal vein occlusion, and non-proliferative diabetic retinopathy. The company's preclinical stage product candidate includes KSI-501, a bispecific conjugate to treat retinal diseases with an inflammatory component; and KSI-601, a triplet biopolymer conjugate for the treatment of dry AMD. The company was formerly known as Oligasis, LLC and changed its name to Kodiak Sciences Inc. in September 2015. Kodiak Sciences Inc. was incorporated in 2009 and is based in Palo Alto, California.

Ocugen, Inc., a clinical-stage biopharmaceutical company, focuses on the developing gene therapies to cure blindness diseases. The company's pipeline product includes OCU400, a novel gene therapy product candidate restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases, such as retinitis pigmentosa and leber congenital amaurosis; OCU410 and OCU410ST for the treatment of dry age-related macular degeneration (AMD); and OCU200, a novel fusion protein that is in preclinical development stage for the treatment of diabetic macular edema, diabetic retinopathy, and wet AMD. It has a strategic partnership with CanSino Biologics Inc. for gene therapy co-development and manufacturing; and Bharat Biotech for the commercialization of COVAXIN in the United States market. The company is headquartered in Malvern, Pennsylvania.