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Rocket Pharmaceuticals Recognizes Rare Disease Day® 2019

February 28, 2019

NEW YORK--(BUSINESS WIRE)--Feb 28, 2019--Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today commemorates Rare Disease Day ®, a day devoted to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients and families. Over 7,000 rare diseases have been identified worldwide. Collectively, they affect 30 million – or 1 in 10 –Americans. In New York City alone, close to 1 million people may be affected.

“Our team at Rocket is motivated everyday by our goal of advancing potentially curative gene therapy treatment options on behalf of the pediatric patients and families suffering from devastating, monogenic rare diseases,” said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Executive Vice President, Development, of Rocket. “We are honored to stand with the global community on Rare Disease Day and do our part to raise awareness of the daily challenges patients and families with rare diseases face as we work to address these areas of significant unmet medical need.”

In recognition of Rare Disease Day, Rocket, in collaboration with the Alexandria Center for Life Science, will be hosting an inaugural Rare Disease Day NYC patient-centered educational event at the Alexandria Center. This closed-event will feature patients and families sharing their experiences living with a rare disease, including Fanconi Anemia, Danon disease, X-linked agammaglobulinemia (a primary immune deficiency), and spinal muscular atrophy. Rare disease patients, caregivers or healthcare providers are welcome to attend this event and registration is available on www.rocketpharma.com.

Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD) ®, the leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases. For more information about Rare Disease Day, go to www.rarediseaseday.org.

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. Rocket’s additional pipeline programs for bone marrow-derived disorders are for 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 a devastating, pediatric heart failure indication, Danon disease. For more information about Rocket, please visit www.rocketpharma.com.

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, 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, Rocket’s ability to commence a registrational study in FA within the projected time periods, 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.

View source version on businesswire.com:https://www.businesswire.com/news/home/20190228005478/en/

CONTACT: Claudine Prowse, Ph.D.

SVP, Strategy, Corporate Development and IRO

Rocket Pharma, Inc.

The Empire State Building, Suite 7530

New York, NY 10118




SOURCE: Rocket Pharmaceuticals, Inc.

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PUB: 02/28/2019 08:00 AM/DISC: 02/28/2019 08:01 AM


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