Rocket Pharmaceuticals Appoints Gayatri R. Rao, M.D., J.D., Former U.S. Food and Drug Administration
Director of the Office of Orphan Products Development, as Vice President, Regulatory Policy and Patient Advocacy
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy
company, today announced the appointment of Gayatri R. Rao, M.D., J.D., as Vice President, Regulatory Policy and Patient Advocacy.
In this new role, Dr. Rao will provide critical direction to global regulatory policies and strategies for Rocket programs to bring
multiple therapies to patients with rare and devastating diseases. This will include oversight of regulatory strategy, patient
advocacy initiatives, and rare disease natural history studies supporting Rocket’s programs.
Dr. Rao most recently served as Director of the Office of Orphan Products Development (OOPD) within the U.S. Food and Drug
Administration (FDA) where she was responsible for implementing statutory programs focused on promoting the development of medical
products for rare diseases. As Director, Dr. Rao served as the FDA’s rare disease lead for interactions with Congress,
international regulatory counterparts, other federal agencies, press, sponsors, and patient advocacy groups. Dr. Rao previously was
an Associate Chief Counsel in FDA’s Office of Chief Counsel where she advised on a variety of issues related to clinical trials,
medical devices, and combination products. She began her career at an international law firm in Washington, D.C., where she focused
on healthcare and food and drug law related matters. She holds a J.D. from the University of Pennsylvania Law School, a Master of
Bioethics from the University of Pennsylvania School of Medicine, and an M.D. from Rutgers New Jersey Medical School.
“Rocket is pleased to welcome Dr. Rao during a particularly exciting time as we look forward to presenting updated data from our
Fanconi Anemia (FA) program later this week at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting,” said Gaurav
Shah, M.D., Chief Executive Officer and President of Rocket. “Dr. Rao’s accomplished track record of working at the FDA within the
Office of Orphan Products Development will be very valuable for Rocket in shaping the strategies for the multiple regulatory
filings that we have planned, including the global registration trial for FA.”
Kinnari Patel, Pharm.D., MBA, Chief Operating Officer & Head of Development of Rocket commented, “Dr. Rao’s commitment to
rare disease product development aligns strongly with Rocket’s mission of finding curative therapies for patients with devastating
diseases. In addition, Dr. Rao’s extensive experience in patient advocacy will be particularly important for building the natural
history studies, which constitute an essential scientific foundation for effective clinical development programs. This is
especially the case in drug development for rare diseases, for which comprehensive understanding of clinical pathogenesis is often
lacking.”
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 www.rocketpharma.com.
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
cp@rocketpharma.com
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