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CAPR vs PTCT

Capricor Therapeutics Inc and PTC Therapeutics, Inc.

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Capricor Therapeutics Inc

Capricor Therapeutics, Inc., a clinical-stage biotechnology company, engages in the development of transformative cell and exosome-based therapeutics for treating duchenne muscular dystrophy (DMD) and other diseases with unmet medical needs in the United States. Its lead product candidate is the Deramiocel, an allogeneic cardiosphere-derived cells, which is in phase 3 clinical trial for the treatment of DMD. The company also develops Exosome protein-based vaccine, which is in preclinical trial to treat SARS-CoV-2; StealthX Exosome Platform, and exosome platform program consists of engineered exosomes for vaccine and therapeutic development and is in preclinical trial. In addition, it engages in developing StealthX, an engineered exosome-based vaccine candidate, under phase 1 clinical study for a range of therapeutic applications, including targeted RNA, protein, and small molecule therapeutics to treat or prevent a variety of diseases; and CAP-2003, and allogeneic cardiosphere-derived cells-exosomes, which is in preclinical trial to treat DMD. It has license agreement with Johns Hopkins University, University of Rome License, and Cedars-Sinai Medical Center to develop and commercialize licensed products and licensed services under the licensed patent rights in all fields and a nonexclusive right to the know-how; Cell Line License Agreement with Life Technologies for the development of exosomes platform. The company was founded in 2005 and is headquartered in San Diego, California.

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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.

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