ROCKET PHARMACEUTICALS Business Summary

Rocket Pharmaceuticals, Inc. (RCKT) is a late-stage biotechnology company primarily focused on developing first-in-class gene therapies for rare and devastating diseases. The company operates through a research and development business model, currently generating revenue through strategic collaborations and licensing, with a transition toward commercial product sales following the late-2025 BLA acceptance for KRESLADI™ (marnetegragene autotemcel). Its primary business segments are categorized by therapeutic platform: Adeno-Associated Virus (AAV) for cardiovascular diseases—including Danon Disease, PKP2-arrhythmogenic cardiomyopathy, and BAG3-dilated cardiomyopathy—and Lentiviral (LV) for hematologic disorders like Leukocyte Adhesion Deficiency-I (LAD-I) and Fanconi Anemia. Rocket fits into the market as a specialized leader in monogenic cardiomyopathies, competing directly with gene therapy developers such as uniQure (QURE), Lexeo Therapeutics (LXEO), and Tenaya Therapeutics (TNA) by leveraging a prioritized, high-density pipeline of potentially curative one-time treatments. Leadership is spearheaded by Co-founder and CEO Dr. Gaurav Shah, an oncologist by training with a pedigree from Novartis and Memorial Sloan Kettering. The executive team includes Dr. Jonathan Schwartz (Chief Science and Gene Therapy Officer), who brings extensive experience in clinical development, and Aaron Ondrey (CFO), who joined in 2024 to oversee the company’s capital allocation and strategic reorganization. Other key officers include COO Chris Stevens and CMO Dr. Syed Rizvi. The company is backed by prominent institutional investors, most notably RTW Investments, LP, whose Managing Partner, Dr. Roderick Wong, serves as Chairman of the Board. Other significant shareholders include Vanguard Group, BlackRock, and Maverick Capital, providing a stable institutional base as the company targets a cash runway extending into 2027.