BioMarin Pharmaceutical Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. It has license and collaboration agreements with Sarepta Therapeutics; Ares Trading S.A.; Catalyst Pharmaceutical Partners, Inc.; and CAMP4 Therapeutics Corporation. The company was incorporated in 1996 and is headquartered in San Rafael, California.

Disc Medicine, Inc., together with its subsidiaries, a clinical-stage biopharmaceutical company, engages in the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases in the United States. The company has assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, primarily heme biosynthesis and iron homeostasis. Its pipeline includes bitopertin for the treatment of erythropoietic porphyrias, including erythropoietic protoporphyria, X-linked protoporphyria, and diamond-blackfan anemia; DISC-0974 for the treatment of anemia of myelofibrosis, and anemia of chronic kidney disease; and DISC-3405 for the treatment of polycythemia vera, and other hematologic disorders. The company's preclinical programs include DISC-0998, for the treatment of anemia associated with inflammatory diseases. Disc Medicine, Inc. was founded in 2017 and is headquartered in Watertown, Massachusetts.

Keros Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops and commercializes novel therapeutics for patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta family of proteins in the United States. The company's lead product candidate is KER-050, which is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia in patients with myelodysplastic syndromes, as well as in patients with myelofibrosis. It also develops KER-012, which is in Phase II clinical trial to treat pulmonary arterial hypertension and cardiovascular disorders; and KER-065 that is in Phase I clinical trial for the treatment of obesity and neuromuscular diseases. In addition, the company has collaboration and license agreement with Hansoh (Shanghai) Healthtech Co., Ltd. to develop, manufacture, and commercialize KER-050 and licensed products containing KER-050. Keros Therapeutics, Inc. was incorporated in 2015 and is headquartered in Lexington, 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.

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.

Syros Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of treatment for hematologic malignancies. The company's lead product candidates are Tamibarotene, a selective retinoic acid receptor alpha agonist, which is in Phase III clinical trial for genomically defined subset of patients with myelodysplastic syndrome and Phase II clinical trial for patients with acute myeloid leukemia; SY-2101, a novel oral form of arsenic trioxide for treating patients with acute promyelocytic leukemia; and SY-5609, a cyclin-dependent kinase 7 inhibitor, which is in a Phase I clinical trial in patients with select advanced solid tumors. The company was formerly known as LS22, Inc. and changed its name to Syros Pharmaceuticals, Inc. in August 2012. Syros Pharmaceuticals, Inc. was incorporated in 2011 and is headquartered in Cambridge, 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.