News Release Details
Rocket Pharmaceuticals Announces Buildout of R&D and Manufacturing Facility to Support Development of Innovative Gene Therapy Pipeline
Jan 11, 2021
—New 103,720 ft2 Facility to Serve as Headquarters and House 150 Employees in
—State of the Art R&D Facility to Support Manufacturing Including AAV Drug Product—
—cGMP Production to be Initiated in 2021 for Planned Phase 2 Study of First AAV-based Gene Therapy, RP-A501 for the Treatment of Danon Disease—
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Rocket Pharmaceuticals Headquarters (Photo: Business Wire)
“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
“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
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.
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the preclinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors’ ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended
SVP, Strategy & Corporate Development