Scholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery, development, and delivery of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of myostatin that is in Phase 3 clinical trial for the treatment of spinal muscular atrophy; and SRK-181, which has completed Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is developing a pipeline of product candidates to deliver novel therapies to treat a range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.

Akebia Therapeutics, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics for patients with kidney diseases. The company's lead product investigational product candidate is Vafseo (vadadustat), an oral hypoxia-inducible factor prolyl hydroxylase, which is in Phase III development for the treatment of anemia due to chronic kidney disease (CKD) in dialysis-dependent and non-dialysis dependent patients. It offers Auryxia, a ferric citrate that is used to control the serum phosphorus levels in adult patients with DD-CKD on dialysis; and the treatment of iron deficiency anemia in adult patients with CKD not on dialysis. The company's product pipeline includes AKB-9090, a drug targeting critical-care indications; and AKB-10108, a drug targeting conditions related to premature birth. It has collaboration agreements with Mitsubishi Tanabe Pharma Corporation for the development and commercialization of vadadustat in Japan and other Asian countries, as well as research and license agreement with Janssen Pharmaceutica NV for the development and commercialization of hypoxia-inducible factor prolyl hydroxylase targeted compounds worldwide. Akebia Therapeutics, Inc. was incorporated in 2007 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.

Inozyme Pharma, Inc., a clinical-stage rare disease biopharmaceutical company, focuses on developing novel therapeutics for the treatment of vasculature, soft tissue, and skeleton diseases. The company's lead product candidate is INZ-701, a soluble, recombinant, genetically engineered, and fusion protein for the treatment of ENPP1 and ABCC6 deficiencies, as well as patients with end-stage kidney disease receiving hemodialysis. It has a license agreement with Yale University for specified therapeutic and prophylactic products. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

Unicycive Therapeutics, Inc., a biotechnology company, develops treatments for various medical conditions in the United States. Its drug candidates include UNI 494, a pro-drug of Nicorandil that is being developed as a treatment for acute kidney injury; and Renazorb (Oxylanthanum Carbonate), which is being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease. Unicycive Therapeutics, Inc. has license agreements with Shilpa Medicare Ltd; Sphaera Pharma Pte. Ltd.; Syneos Health LLC; Inotiv; Lee's Pharmaceutical (HK) Limited; Celerion; and Lotus International Pte Ltd. The company was incorporated in 2016 and is based in Los Altos, California.

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.

Synlogic, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of synthetic biotics to treat metabolic diseases in the United States. Its pipeline include SYNB1618, an orally administered, non-systemically absorbed drug candidate to treat phenylketonuria; SYNB1934, an orally administered, non-systemically absorbed drug candidate, which is in Phase III clinical trial to treat phenylketonuria; SYNB1353, an orally administered, non-systemically absorbed drug candidate, which is in Phase I clinical to treat homocystinuria; SYNB8802, an orally administered, non-systemically absorbed drug candidate that is in Phase II clinical trial for the treatment of enteric hyperoxaluria; and SYNB2081 to lower uric acid for the potential treatment of gout. The company has a collaboration agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. for the research and pre-clinical development of a synthetic biotic medicine for the treatment of inflammatory bowel disease; and Ginkgo Bioworks, Inc. Synlogic, Inc. is based in Cambridge, Massachusetts.

Cyclacel Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer and other proliferative diseases in the United States, the United Kingdom, and internationally. The company's lead product includes fadraciclib, a cyclin dependent kinase Inhibitors (CDK) that is in Phase 1/2 clinical trial for the treatment of solid tumors and hematological malignancies, as well as in combination with venetoclax to treat relapsed or refractory chronic lymphocytic leukemia; and Plogosertib, a polo-like kinase inhibitor program, which is in Phase 1/2 clinical trial for the treatment of advanced solid tumors and hematological malignancies. It has a clinical collaboration agreement with the University of Texas MD Anderson Cancer Center to clinically evaluate the safety and efficacy of three cyclacel medicines in patients with hematological malignancies, including chronic lymphocytic leukemias, acute myeloid leukemias, myelodysplastic syndromes, and other advanced leukemias. The company was founded in 1996 and is headquartered in Berkeley Heights, New Jersey.