Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals is a late-stage biotechnology company focused on developing, manufacturing, and selling genetic therapies for rare and devastating diseases in the United States. Its pipeline includes both in vivo adeno-associated viral (AAV) programs and ex vivo lentiviral (LV) programs. Key AAV programs target Danon disease (RP-A501, Phase 2), Plakophilin-2 Arrhythmogenic Cardiomyopathy (RP-A601, Phase 1), and BAG3 Dilated Cardiomyopathy (preclinical). Key LV programs address Leukocyte Adhesion Deficiency-I (RP-L201), Fanconi Anemia (RP-L102), and Pyruvate Kinase Deficiency (RP-L301). The company also has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas.

Rocket Pharmaceuticals is listed on the Nasdaq Global Market (NasdaqGM) under the ticker symbol RCKT. Its financial reporting and trading are conducted in US Dollars (USD), as the company is based in the United States and its stock is traded on a US exchange.

Rocket Pharmaceuticals operates in the Healthcare sector, specifically within the Biotechnology industry. As a biotechnology company, it focuses on developing genetic therapies for rare diseases, leveraging advanced technologies such as adeno-associated viral (AAV) and lentiviral (LV) vectors to address serious genetic disorders.

Rocket Pharmaceuticals is headquartered in the United States and primarily serves the US market. The company's description indicates it focuses on developing and selling genetic therapies for rare and devastating diseases in the United States. Its research and development activities, as well as its license agreements, are centered in the US, with a notable license agreement with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas in Spain.

Rocket Pharmaceuticals' pipeline includes several key programs. In its in vivo AAV portfolio, RP-A501 targets Danon disease (Phase 2), RP-A601 targets Plakophilin-2 Arrhythmogenic Cardiomyopathy (Phase 1), and a preclinical program targets BAG3 Dilated Cardiomyopathy. In its ex vivo LV portfolio, RP-L201 addresses Leukocyte Adhesion Deficiency-I, RP-L102 targets Fanconi Anemia, and RP-L301 treats Pyruvate Kinase Deficiency. These programs aim to treat rare genetic disorders with significant unmet medical needs.