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
May 15, 2018
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NEW YORK--(BUSINESS WIRE)--May 15, 2018--
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20180515005165/en/
Source: 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
cp@rocketpharma.com