Rocket Pharmaceuticals Announces Strategic Research Collaboration

- Agreement to Support the Advancement of Fanconi Anemia and Pyruvate Kinase Deficiency Gene Therapy Research -

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, and the Stanford University School of Medicine today announced a strategic collaboration to support the advancement of Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD) gene therapy research. Rocket's lentiviral vector (LVV)-based gene therapy program for FA is currently in clinical trials with academic partners in the U.S. and Europe. The LVV-based gene therapy program for PKD is currently in preclinical development in Europe.

Under the terms of the agreement, Stanford will serve as a lead clinical trial research center in the United States for a planned upcoming registrational trial for FA, and would also be the lead site for PKD clinical trials. Maria Grazia Roncarolo, M.D., director of the Stanford Center for Definitive and Curative Medicine and co-director of the school's Institute for Stem Cell Biology and Regenerative Medicine, will lead the school's effort. The center is a joint initiative of the School of Medicine, Stanford Health Care and Stanford Children's Health and is focused on bench to bedside development of innovative cell- and gene-based therapies.

Gaurav Shah, M.D., Chief Executive Officer and President of Rocket, commented, "Rocket is delighted to expand the reach of our gene therapy program in the U.S. and prepare for our registrational trial. We are committed to developing FA and PKD programs in collaboration with outstanding gene therapy centers and pioneers in the field. This collaboration with the Stanford Center for Definitive and Curative Medicine is a critical step within our overall strategy of building relationships with gene therapy experts, with investigators who have dedicated their careers to improving the care of patients afflicted with these disorders, and within the broader FA and PKD communities."

"This project will also evaluate the introduction of conditioning regimens for both FA and PKD, where we hope to develop best-in-class gene therapy approaches for both clinical indications. The regulatory design and preparation for our registrational trial for FA is ongoing and we remain on track to advance this program to a registration study in 2019. Our PKD program continues to advance in preclinical studies with an Investigational Medicinal Product Dossier (IMPD) expected to be filed in early 2019," continued Dr. Shah.

For more information about this news on the Stanford website, please visit

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket") is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket's multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Preclinical studies of additional bone marrow-derived disorders are ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for an undisclosed rare pediatric disease. For more information about Rocket, please visit

Rocket Pharmaceuticals, Inc. 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 products that Rocket may develop, including in collaboration with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile

Malignant Osteopetrosis (IMO), 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 Annual Report on Form 10-K for the year ended December 31, 2017. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


Rocket Pharmaceuticals, Inc.:
Claudine Prowse, Ph.D.
SVP Corporate Development and IRO
Rocket Pharmaceuticals, Inc.
The Alexandria Center for Life Science
430 East 29 Street, Suite 1040
New York, NY 10016


Source: Rocket Pharmaceuticals, Inc.

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