Rocket Pharmaceuticals, a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, announces the Company’s plans for the buildout of its new Research and Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operation which will also serve as the Company’s new headquarters in Cranbury, New Jersey.

This new 103,720 ft2 facility will support clinical development of Rocket’s growing pipeline of lentivirus (LV) and adeno-associated virus (AAV) gene therapies from discovery through pivotal trials, with space for potential future expansion and commercialization.

This buildout comes on the heels of the Company’s recent successful capital raise of approximately $300 million that provides a cash runway into the second half of 2023.

“Investing in R&D and manufacturing innovation, talent, and capacity through this new world-class facility will allow us to deliver on our mission of bringing five curative gene therapies to rare disease patients by 2025,” said Gaurav Shah, M.D., President and Chief Executive Officer of Rocket. “With data on five clinical programs expected this year, including two that are in registration-enabling trials, these new capabilities will enable us to work with continued urgency and purpose towards bringing transformational therapies to patients.”

“We are collaborating with some of the best scientists and innovators worldwide. This new facility is instrumental in bolstering Rocket’s gene therapy capabilities to rapidly advance multiple platforms and programs efficiently and effectively. Producing clinical drug product will enable greater control of supply, cost, quality, and timing to pave a smoother path toward commercialization,” added Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development.

Approximately one-half of the facility is being scaled for AAV Current Good Manufacturing Practice (cGMP) production. The other half features state-of-the-art R&D labs to support the expanding pipeline and Quality Control (QC) laboratories to support CMC development for process and analytics.

Rocket recently reported positive interim Phase 1 results for its first AAV-based gene therapy, RP-A501 for the treatment of Danon Disease. The first cGMP production at this facility will be initiated in 2021 and will be used in a planned Phase 2 registrational study evaluating RP-A501, following the completion of the current Phase 1 trial.

On December 14, 2020, Rocket closed an upsized underwritten public offering of 6,035,714 shares of its common stock, inclusive of greenshoe, at the public offering price of $56.00 per share. The offering was ~7.3x oversubscribed based on the initial deal size of $175 million. Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the U.S. and Europe, accelerate the buildout of in-house manufacturing capabilities, and for general corporate purposes. This capital raise extends Rocket’s cash runway to the second half of 2023.