Fulcrum Therapeutics, Inc. Common Stock
Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops small molecules to improve the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its clinical-stage product candidate is pociredir, a fetal hemoglobin inducer, which is in phase 1b clinical trial for the treatment of sickle cell disease and beta-thalassemia. The company also discovers drug targets for potential treatment inherited aplastic anemias, such as of diamond-blackfan anemia, shwachman-diamond syndrome, and fanconi anemia, as well as novel fetal hemoglobin inducers, fibrotic disorders, and cardiomyopathies which is in preclinical trial. It has collaboration and license agreement with MyoKardia, Inc. to identify biological targets that are capable of modulating genes of interest with relevance to cardiomyopathies; and licensce agreement with CAMP4 which includes certain small molecule compounds, composition of matter and method of use patent rights, and know-how to research, develop, manufacture, use, and commercialize; 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.
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Ionis Pharmaceuticals, Inc., a commercial-stage biotechnology company, provides RNA-targeted medicines in the United States. The company offers TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS) and acute pancreatitis; WAINUA for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults; and SPINRAZA for pediatric and adult patients with spinal muscular atrophy (SMA). It also provides QALSODY for the treatment of Amyotrophic Lateral Sclerosis (ALS); TEGSEDI for the treatment of ATTRv-PN in adults; and WAYLIVRA for treatment for FCS and familial partial lipodystrophy. It also develops products under Phase 3 clinical trials, such as Olezarsen for patients with hypertriglyceridemia (SHTG) and cardiovascular disease (CVD); Donidalorsen for patients with hereditary angioedema; and Zilganerse, a potential treatment for people with genetically confirmed Alexander disease, as well as ION582 that has completed Phase 2 clinical trial for the potential treatment of AS, a rare genetic neurological disease. In addition, the company develops Eplontersen to degrade mutant and wild-type TTR mRNA through binding to the TTR mRNA; Pelacarsen to inhibit the production of apolipoprotein(a) in the liver to offer a direct approach for reducing lipoprotein(a); Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus; Sefaxersen to reduce the production of complement factor B and lower activation of the alternative complement pathway; and Ulefnersen to reduce the production of the fused in sarcoma, as well as other mid-stage pipeline investigational medicines. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with GSK, AstraZeneca, Novartis, and Roche, as well as with Metagenomi. The company was incorporated in 1989 and is headquartered in Carlsbad, California.
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