Blueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally. The company is developing AYVAKIT for the treatment of systemic mastocytosis (SM) and gastrointestinal stromal tumors; BLU-263, an orally available, potent, and KIT inhibitor for the treatment of indolent SM, and other mast cell disorders. It is also developing GAVRETO for the treatment of RET fusion-positive non-small cell lung cancer, altered thyroid carcinoma, and medullary thyroid carcinoma; BLU-945 for the treatment of epidermal growth factor receptor driven non-small-cell lung carcinoma (NSCLC); and BLU-451 to treat NSCLC in patients with epidermal growth factor receptor gene (EGFR) exon 20 insertion mutations. In addition, the company is developing BLU-782, for the treatment of fibrodysplasia ossificans progressive; BLU- 222 to treat patients with cyclin E aberrant cancers; and BLU-852 for the treatment of advanced cancers. It has collaboration and license agreements with Clementia Pharmaceuticals, Inc.; Proteovant Therapeutics; CStone Pharmaceuticals; Genentech, Inc.; Hoffmann-La Roche Inc.; and Zai Lab (Shanghai) Co., Ltd. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was incorporated in 2008 and is headquartered in Cambridge, Massachusetts.

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.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, acute leukemias, and solid tumor; and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD) and idiopathic pulmonary fibrosis (IPF). The company is also developing Entinostat. It has an agreement with Eddingpharm International Company Limited for licensing, development, and commercialization of Entinostat. Syndax Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Waltham, 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.

Cullinan Therapeutics, Inc., a biopharmaceutical company, focuses on developing oncology therapies for cancer patients in the United States. The company's lead program comprises CLN-619, a monoclonal antibody that is in Phase I clinical trial for the treatment of solid tumors. Its development portfolio also includes CLN-049, a humanized bispecific antibody that is in Phase I clinical trial for the treatment of acute myeloid leukemia or myelodysplastic syndrome; CLN-418, a human bispecific immune activator that is in Phase 1 clinical trial for the treatment of multiple solid tumors; and Zipalertinib, a bioavailable small-molecule for treating patients with non-small cell lung cancer. In addition, the company's development products comprise CLN-617, a fusion protein for the treatment of solid tumors; and CLN-978, a T cell engaging antibody for the treatment relapsed/refractory B-cell non-Hodgkin lymphoma. It has license and collaboration agreement with Adimab, LLC to discover and/or optimize antibodies; Harbour BioMed US Inc. for the development, manufacturing, and commercialization of CLN-418; and co-development agreement with Taiho Pharmaceutical Co., Ltd to develop Zipalertinib. Cullinan Therapeutics, Inc. was formerly known as Cullinan Oncology, Inc. and changed its name to Cullinan Therapeutics, Inc. in April 2024. Cullinan Therapeutics, Inc. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Rigel Pharmaceuticals, Inc., a biotechnology company, engages in discovering, developing, and providing therapies that enhance the lives of patients with hematologic disorders and cancer. The company's commercialized products include Tavalisse, an oral spleen tyrosine kinase inhibitor for the treatment of adult patients with chronic immune thrombocytopenia; Rezlidhia, a non-intensive monotherapy for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test; and GAVRETO, a once daily, small molecule, oral, kinase inhibitor for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer, as well as for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer. It also develops R289, an oral IRAK1/4 Inhibitor, which is in Phase 1b clinical trials for the treatment of hematology-oncology, autoimmune, and inflammatory diseases; and a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor program in clinical development with partner Eli Lilly and Company. In addition, the company has product candidates in clinical development with partners BerGenBio ASA and Daiichi Sankyo. The company has strategic development collaboration with The University of Texas MD Anderson Cancer Center for the development of REZLIDHIA (Olutasidenib) in acute myeloid leukemia (AML) and other hematologic cancers. The company was incorporated in 1996 and is headquartered in South San Francisco, California.

Aptose Biosciences Inc., a clinical-stage biotechnology company, discovers and develops personalized therapies addressing unmet medical needs in oncology primarily in the United States. The company's clinical-stage oral kinase inhibitors include tuspetinib (HM43239), a once-daily oral kinase inhibitor that has completed Phase 1/2 clinical trial targeting a select group of kinases operative in myeloid malignancies, such as acute myeloid leukemia and the higher risk myelodysplastic syndromes; and luxeptinib (CG-806), a potent oral small molecule, which is in Phase 1 a/b clinical trial in patients with relapsed or refractory B cell malignancies, including chronic lymphocytic leukemia, small lymphocytic lymphoma, and certain non-Hodgkin's lymphomas, as well as relapsed or refractory acute myeloid leukemia. It has license agreements with CrystalGenomics Invites Co. Ltd. to research, develop, and commercialize luxeptinib; with National Cancer Institute for the clinical development of tuspetinib; and Hanmi Pharmaceutical Co Ltd. to research, develop, and commercialize tuspetinib. The company was formerly known as Lorus Therapeutics Inc. and changed its name to Aptose Biosciences Inc. in August 2014. Aptose Biosciences Inc. was incorporated in 1986 and is headquartered in Toronto, Canada.

Vor Biopharma Inc. operates as a clinical-stage cell and genome engineering company. Its lead product is tremtelectogene empogeditemcel (trem-cel), an engineered hematopoietic stem cell (eHSC) product candidate that is in phase 1/2 trial to treat acute myeloid leukemia (AML) and other hematological malignancies. The company is also developing Mylotarg, a CD33-directed antibody drug conjugate (ADC) therapy for patients with leukemia; VCAR33, a CAR-T therapy designed to target CD33, a clinically validated target for AML, which are under preclinical and Phase 1/2 clinical studies; trem-cel + VCAR33 treatment system that is under preclinical studies for the treatment of AML; and CD33-CLL1 treatment system made with CD33-CLL1 multiplex-engineered eHSC and CD33-CLL1 multi-specific CAR-T, which are under preclinical studies for the treatment of AML. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.