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ALNY vs PTCT

Alnylam Pharmaceuticals, Inc. and PTC Therapeutics, Inc.

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Alnylam Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc. discovers, develops, and commercializes therapeutics based on ribonucleic acid interference. It offers ONPATTRO and AMVUTTRA for hereditary transthyretin-mediated amyloidosis; GIVLAARI for acute hepatic porphyria; OXLUMO for primary hyperoxaluria type 1; and Leqvio for hypercholesterolemia. It also develops vutrisiran, which is in phase III clinical trial for transthyretin amyloidosis (ATTR) with cardiomyopathy; Nucresiran that is in phase I clinical trial for ATTR amyloidosis; Fitusiran, which is in phase III clinical trial for hemophilia; cemdisiran that is in phase III clinical trial for Myasthenia Gravis and paroxysmal nocturnal hemoglobinuria, and geographic atrophy; and ALN-6400, which is in phase I clinical trial for bleeding disorders. In addition, the company is also developing Zilebesiran, which is in phase II clinical trial for hypertension; Rapirosiran that is in phase II clinical trial for Metabolic dysfunction-associated steatohepatitis; ALN-4324, which is in phase I clinical trial for type 2 diabetes mellitus; ALN-PNP that is in phase I clinical trial for non-alcoholic fatty liver disease; ALN-APOC3, which is in phase I clinical trial for dyslipidemia; Mivelsiran that is in phase II clinical trial for cerebral amyloid angiopathy, and phase I clinical trial for Alzheimer's disease; and ALN-HTT02 that is in phase I clinical trial for Huntington's disease. Further, it develops ALN-SOD, which is in phase II clinical trial for SOD1 Amyotrophic lateral sclerosis; Elebsiran that is in phase II clinical trial for Hepatitis B and D virus infections; ALN-BCAT that is in phase I clinical trial for hepatocellular carcinoma; and ALN-ANG3. It has collaborations with Regeneron Pharmaceuticals, Inc.; Roche Holding AG; Regeneron Pharmaceuticals, Inc.; Sanofi S.A.; Novartis AG; PeptiDream, Inc; Dicerna Pharmaceuticals, Inc.; and Ionis Pharmaceuticals, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

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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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