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.

Editas Medicine, Inc., a clinical stage genome editing company, focuses on developing transformative genomic medicines to treat a range of serious diseases. It develops a proprietary gene editing platform based on CRISPR technology. The company develops EDIT-101, which is in Phase 1/2 BRILLIANCE trial for Leber Congenital Amaurosis; and reni-cel, a clinical development gene-edited medicine to treat sickle cell disease and transfusion-dependent beta-thalassemia. In addition, the company is developing alpha-beta T cells for solid and liquid tumors; and gamma delta T cell therapies to treat cancer. It has a research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; collaboration and nonexclusive license agreement with Genevant Sciences to develop Novel mRNA-LNP gene editing therapeuticsand strategic alliance and option agreement with Allergan Pharmaceuticals International Limited, as well as collaboration with Bristol Myers Squibb for research and development of alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was incorporated in 2013 and is based in Cambridge, Massachusetts.

Adverum Biotechnologies, Inc., a clinical-stage company, develops gene therapy product candidates to treat ocular diseases. Its lead product candidate is ixoberogene soroparvovec (ADVM-022), a single intravitreal injection gene therapy candidate used for the treatment of patients with wet age-related macular degeneration and diabetic macular edema which is in phase 2 clinical trials. The company is developing ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate for the treatment of blue cone monochromacy via a single IVT injection. Adverum Biotechnologies, Inc. has license and collaboration agreements with University of California; GenSight Biologics; Lexeo Therapeutics; and Virovek, Inc. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was incorporated in 2006 and is headquartered in Redwood City, California.

Cabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. The company's lead product candidate is CABA-201, a fully human anti-CD19 binder for the treatment of Phase 1/2 clinical trials in dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, lupus nephritis, non-renal systemic lupus erythematosus, systemic sclerosis, and generalized myasthenia gravis. It also develops DSG3-CAART, which is in Phase I/II clinical trial for the treatment of mucosal pemphigus vulgaris; and MuSK-CAART, an investigational cell therapy that is in Phase I/II clinical trial for treating patients with anti- muscle-specific kinase antibody positive myasthenia gravis. It has a collaboration with the University of Pennsylvania and the Children's Hospital of Philadelphia; Nanjing IASO Biotherapeutics Co., Ltd; Oxford Biomedica; and WuXi Advanced Therapies, Inc. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.

bluebird bio, Inc., a biotechnology company, researches, develops, and commercializes gene therapies for curative severe genetic diseases in the United States. The company's 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. Its 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. 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.

Abpro Holdings, Inc., a biotechnology company, engages in developing antibody therapeutics for patients with immuno-oncology, ophthalmology, and autoimmunity issues. Its platform DiversImmune addresses a bottleneck in the antibody therapeutics industry. Its lead product candidates include ABP-100 for the treatment of breast, gastric, and colorectal cancers; and ABP-201 for the treatment of vascular diseases of the eye. The company has a strategic partnership with Abpro Bio Co. Ltd and NJCTTQ. The company was formerly known as Abpro Corporation and changed its name to Abpro Holdings, Inc. in November 2024. Abpro Holdings, Inc. was incorporated in 2004 and is headquartered in Woburn, Massachusetts.

AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company, develops novel cell therapies for unmet medical needs in the United States and internationally. The company develops OpRegen, an allogeneic retinal pigment epithelium cell replacement therapy, which is in Phase 2a clinical trial for the treatment of the dry age-related macular degeneration; OPC1, an allogeneic oligodendrocyte progenitor cell therapy that is in Phase 1/2a multicenter clinical trial for the treatment of cervical spinal cord injuries; and VAC, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells, which is in Phase I clinical trial to treat non-small cell lung cancer. It also offers ANP1, an allogeneic auditory neuron progenitor cell transplant, which is in preclinical development for the treatment of debilitating hearing loss; and PNC1, an allogeneic photoreceptor cell transplant, which is in preclinical development for the treatment of vision loss due to photoreceptor dysfunction or damage. In addition, the company engages in the research and development of therapeutic products for retinal diseases, neurological diseases, and disorders and oncology. Lineage Cell Therapeutics, Inc. has a collaboration with Immunomic Therapeutics, Inc., for the treatment of glioblastoma multiforme. The company was formerly known as BioTime, Inc. and changed its name to Lineage Cell Therapeutics, Inc. in August 2019. Lineage Cell Therapeutics, Inc. was incorporated in 1990 and is headquartered in Carlsbad, California.