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INSM vs PTCT

Insmed, Inc. and PTC Therapeutics, Inc.

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Insmed, Inc.

Insmed Incorporated develops and commercializes therapies for patients with serious and rare diseases in the United States, Europe, Japan, and internationally. The company offers ARIKAYCE for the treatment of refractory nontuberculous mycobacterial lung infections, as well as is in phase 3 clinical trial for the treatment of mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients. It also devlops brensocatib, an oral reversible inhibitor of dipeptidyl peptidase 1(DPP1) that is in phase 3 clinical trial for the treatment of bronchiectasis; and in phase 2 clinical trial for the treatment of chronic rhinosinusitis without nasal polyps and hidradenitis suppurativa. In addition, the company is developing treprostinil palmitil inhalation powder, an inhaled formulation of a treprostinil prodrug treprostinil palmitil, which is in phase 3 clinical trial for the treatment of pulmonary hypertension associated with interstitial lung disease; and phase 2 clinical trial for the treatment of pulmonary arterial hypertension. Further, it develops gene therapy, a microdystrophin adeno-associated virus gene replacement therapy which is in phase 1 clinical trial for the treatment of Duchenne muscular dystrophy, as well as is in pre-clinical development for gene therapy, next generation DPP1 inhibitor, deimmunized therapeutic protein, and synthetic rescue. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.

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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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