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ALNY vs RCKT

Alnylam Pharmaceuticals, Inc. and Rocket Pharmaceuticals, Inc. Common Stock

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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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Rocket Pharmaceuticals, Inc. Common Stock

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

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