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ALNY vs RGNX

Alnylam Pharmaceuticals, Inc. and REGENXBIO 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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REGENXBIO Inc.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase III clinical trial for the treatment of Duchenne muscular dystrophy, as well as RGX-121 for the treatment of mucopolysaccharidosis type II. It also develops RGX-111 for the treatment of mucopolysaccharidosis type I, RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for the treatment of the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.

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