PTC Therapeutics, Inc.
PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.
Open PTCT dashboard ↗Rigel Pharmaceuticals Inc. (New)
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 to treat 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 (NSCLC), as well as to treat adult and pediatric patients twelve years of age and older with advanced or metastatic RET fusion-positive thyroid cancer. It also develops R289, an oral interleukin receptor-associated kinases 1 and 4 (IRAK1/4) inhibitor for the treatment of hematology-oncology, autoimmune, and inflammatory diseases, as well as to treat lower-risk myelodysplastic syndrome. In addition, the company has product candidates in clinical development with partners BerGenBio ASA, Eli Lilly and Company, and Daiichi Sankyo. It has strategic development collaboration with The University of Texas MD Anderson Cancer Center for the development of Olutasidenib in AML and other hematologic cancers with IDH1mutations, as well as with collaborative network for neuro-oncology clinical trials to evaluate Olutasidenib in combination with temozolomide in patients with high-grade glioma harboring an IDH1 mutation. Rigel Pharmaceuticals, Inc. was incorporated in 1996 and is headquartered in South San Francisco, California.
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