News Release Details
Rocket Pharmaceuticals Announces Clearance of IND for RP-A501 Gene Therapy for Danon Disease
Jan 22, 2019
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- Phase 1 Clinical Trial to Commence in the Second Quarter of 2019 -
“This acceptance marks the first Rocket-sponsored IND cleared for our
AAV platform and the third for our pipeline in only three months,” said
“Danon disease is a devastating heart failure syndrome that develops during childhood and adolescence, and is followed by a rapid progression toward end-stage heart failure and death,” said Dr. Adler. “We look forward to advancing this potentially curative treatment option for the patients and families affected by this life-threatening disease.”
In parallel with the Phase 1 clinical trial, the Company plans to publish a comprehensive literature review of Danon cases and conduct a retrospective chart review. In addition, the Company will initiate a prospective natural history study with enrollment planned to commence in the first quarter of 2019.
About Danon Disease
Danon disease is a rare neuromuscular and cardiovascular disease
characterized by profound cardiomyopathy, skeletal myopathies, and mild
cognitive impairment. It is estimated to have a prevalence of 15,000 to
30,000 patients in the U.S. and the
About
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,
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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
View source version on businesswire.com: https://www.businesswire.com/news/home/20190122005265/en/
Source:
Claudine Prowse, Ph.D.
SVP, Corporate Strategy and IRO
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
www.rocketpharma.com
investors@rocketpharma.com