Rocket Pharmaceuticals, Inc.

Rocket is a fully integrated, late-stage biotechnology company focused on developing, manufacturing, and potentially commercializing genetic therapies for rare and often fatal diseases with high unmet medical need. The company uses an innovative multi-platform approach to create gene therapy product candidates designed to correct the underlying genetic causes of complex inherited cardiomyopathies and related disorders. Rocket's platform is supported by in-house research and development capabilities and current cGMP manufacturing facilities, which enable end-to-end control of clinical production, process development, and scale-up for potential commercialization. Rocket's strategy is to develop and commercialize potentially curative first-in-class gene therapies for devastating, undertreated, and rare diseases. In July 2025, the company announced a strategic corporate reorganization and pipeline prioritization to maximize near-term value, extend its operational runway, and position for sustained long-term growth. This initiative focuses resources on advancing its AAV cardiovascular gene therapy platform and supported the submission of responses to the FDA's Complete Response Letter (CRL) for KRESLADI™. The program for KRESLADI™ contemplates a scaled commercial effort tailored to the exceptionally small patient population affected by this ultra-rare indication. As part of this realignment, Rocket is de-prioritizing further development activities related to its FA and PKD programs. The restructuring included a workforce reduction of approximately 30%, which, along with other planned cost-saving initiatives, is expected to reduce Rocket's 12-month operating expenses by approximately 25%.