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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.

Cytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases. The company develops small molecule drug candidates primarily engineered to impact muscle function and contractility. Its drug candidates include omecamtiv mecarbil, a novel cardiac myosin activator that is in Phase III clinical trial in patients with heart failure. The company also develops CK-136, a novel cardiac troponin activator that is in Phase I clinical trial; CK-586, a small molecule cardiac myosin inhibitor, that is in Phase I clinical trial; and aficamten, a novel cardiac myosin inhibitor, which is in Phase III clinical trial for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy. Cytokinetics, Incorporated has a strategic alliance with Ji Xing Pharmaceuticals Limited. The company was incorporated in 1997 and is headquartered in South San Francisco, California.

Mirati Therapeutics, Inc., a commercial-stage oncology company, develops novel therapeutics to address the genetic and immunological promoters of cancer in the United States. The company provides KRAZATI, an oral targeted treatment option for adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung (NSCLC), as well as in clinical development as a monotherapy and in combination with other agents. It also develops Sitravatinib, an investigational spectrum-selective kinase inhibitor in Phase 3 clinical development that inhibits receptor tyrosine kinases (RTKs), including TAM family receptors, split family receptors, and RET, which overcomes resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment that enhances antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation. In addition, the company pipeline comprises the MRTX1719, an investigational synthetic lethal PRMT5 inhibitor designed to target the PRMT5/methylthioadensoine (MTA) complex and is in clinical development; MRTX0902, a selective SOS1 inhibitor that improves anti-tumor efficacy in combination with targeted mitogen-activated protein kinase (MAPK)-pathway inhibitors, and is in clinical development; and MRTX1133, an investigational lead KRAS G12D compound. It has a collaboration and license agreement with BeiGene, Ltd. to develop, manufacture, and commercialize sitravatinib; and Zai Lab Ltd. to research, develop, manufacture, and commercialize adagrasib in various indications. Mirati Therapeutics, Inc. was founded in 1995 and is headquartered in San Diego, California.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.

Zai Lab Limited develops and commercializes therapies to treat oncology, autoimmune disorders, infectious diseases, and neuroscience. Its commercial products include Zejula, an orally administered poly polymerase 1/2 inhibitor; Optune, a cancer therapy that uses electric fields tuned to specific frequencies to kill tumor cells; NUZYRA for acute bacterial skin and skin structure infections, and community acquired bacterial pneumonia; Qinlock to treat gastrointestinal stromal tumors, and VYVGART, a human IgG1 antibody fragment for myesthenia gravis. The company also develops Tumor Treating Fields, a portable device for delivery of electric fields; Repotrectinib, a tyrosine kinase inhibitor (TKI) to target ROS1 and TRK A/B/C in TKI-naïve- or -pretreated cancer patients; Tisotumab vedotin, an antibody drug conjugate; Adagrasib for treating KRAS-G12C-mutated NSCLC, colorectal cancer, and pancreatic cancer; and Bemarituzumab to treat gastric and gastroesophageal junction cancer patients. In addition, it develops Sulbactam/durlobactam, a combination of a beta-lactam antibiotic and a beta-lactamase inhibitor for the treatment of serious infections caused by Acinetobacter; KarXT for the treatment of psychiatric and neurological conditions. It has license and collaboration agreement with Tesaro, Inc. to develop, manufacture, and commercialize niraparib; NovoCure to develop and commercialize Tumor Treating Fields; Deciphera to develop and commercialize ripretinib; Paratek Bermuda Ltd. to develop, manufacture, and commercialize omadacycline; argenx, to develop and commercialize efgartigimod; BMS to develop and commercialize tisotumab vedotin and repotrectinib; Mirati to research, develop, manufacture, and commercialize adagrasib; Amgen to develop and commercialize bemarituzumab; and Innoviva to develop and commercialize Sulbactam-Durlobactam; Karuna to develop and commercialize KarXT. The company was incorporated in 2013 and is headquartered in Shanghai, China.