Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Vericel Corporation, a commercial-stage biopharmaceutical company, engages in the research, development, manufacture, and distribution of cellular therapies for sports medicine and severe burn care markets in North America. The company markets autologous cell therapy products comprising MACI, an autologous cultured chondrocytes on porcine collagen membrane for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; Epicel, a permanent skin replacement humanitarian use device for the treatment of adult and pediatric patients with deep-dermal or full-thickness burns; and NexoBrid, a biological orphan product for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was incorporated in 1989 and is headquartered in Cambridge, Massachusetts.

Mersana Therapeutics, Inc., a clinical stage biopharmaceutical company, develops antibody drug conjugates (ADC) for cancer patients with unmet needs. The company develops XMT-1660, a B7-H4-targeted Dolasynthen ADC candidate; and XMT-2056, an immunosynthen ADC. It has research and development collaborations with Janssen Biotech, Inc., Ares Trading S.A., Merck KGaA, and Asana BioSciences, LLC for the development of ADC product candidates. The company was formerly known as Nanopharma Corp. and changed its name to Mersana Therapeutics, Inc. in November 2005. Mersana Therapeutics, Inc. was incorporated in 2001 and is headquartered in Cambridge, Massachusetts.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. It has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

2seventy bio, Inc., a cell and gene therapy company, focuses on the research, development, and commercialization of treatments for cancer in the United States. The company's products pipeline includes Abecma, a chimeric antigen receptor T-cell product candidates for the treatment of multiple myeloma. It has a collaboration arrangement with Bristol Myers Squibb Company. The company was incorporated in 2021 and is headquartered in Cambridge, Massachusetts.

Camp4 Therapeutics Corporation, a clinical-stage biopharmaceutical company, discovers and develops regulatory RNA-based therapeutics to treat a range of genetic diseases. Its lead product candidate is CMP-CPS-001 for the treatment of urea cycle disorders. The company also develops CMP-SYNGAP to treat synaptic Ras GTPase activating protein 1-related disorders. Camp4 Therapeutics Corporation was formerly known as Marauder Therapeutics, Inc. and changed its name to Camp4 Therapeutics Corporation in March 2018. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Pulmatrix, Inc., a clinical stage biotechnology company, focused on development of novel inhaled therapeutic products to prevent and treat respiratory and other diseases with unmet medical needs in the United States. The company offers iSPERSE, an engineered dry powder delivery platform, which enables delivery of small or large molecule drugs to the lungs by inhalation for local or systemic applications. It engages in developing PUR1800, a narrow spectrum kinase inhibitor completed Phase 1b clinical trials for the treatment of acute exacerbations in chronic obstructive pulmonary disease; PUR1900 for the treatment of allergic bronchopulmonary aspergillosis in patients with asthma and cystic fibrosis; and PUR3100, an iSPERSE formulation of dihydroergotamine which is in Phase 1 for the treatment of acute migraine. It has a license agreement with RespiVert Ltd. for access to a portfolio of kinase inhibitor drug candidates. The company was founded in 2003 and is headquartered in Bedford, Massachusetts.

Deciphera Pharmaceuticals, Inc., a biopharmaceutical company, develops drugs to enhance the lives of cancer patients by addressing key mechanisms of drug resistance that limit the rate and durability of response to existing cancer therapies in the United States and internationally. Its lead drug candidate is QINLOCK used for the treatment of gastrointestinal stromal tumors (GIST), as well as in INTRIGUE Phase 3 study to treat second-line GIST. The company is also developing vimseltinib, an orally administered, potent, and highly selective switch-control kinase inhibitor of colony stimulating factor 1 receptor (CSF1R) for the treatment of tenosynovial giant cell tumor (TGCT); and DCC-3116 to treat RAS/RAF mutant cancers that is in the preclinical-stage. Deciphera Pharmaceuticals, Inc. was founded in 2003 and is headquartered in Waltham, Massachusetts.