News Release Details
Rocket Pharmaceuticals Sponsors Clinical Trial at University of California, Los Angeles (UCLA)
Mar 11, 2019
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“Rocket has put the building blocks in place to execute upon our
strategy to transform our pipeline with precision and exigency. Dr. Kohn
was chosen to lead the LAD-I and IMO programs as he shared our passion
for gene and cellular therapy research for rare diseases,” said
Rocket’s Investigation New Drug (IND) application for RP-L201 was
cleared by the
About Leukocyte Adhesion Deficiency-I
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by a mutation of the ITGB2 gene that encodes for the Beta-2 Integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to combat infections. The degree of CD18 deficiency determines the severity of the disease, which can be categorized as moderate or severe based on functional CD18 expression. Most patients have the severe form of the disease, with less than 2% of normal neutrophil CD18 expression. Severe LAD-I causes recurrent and life-threatening infections which are frequently fatal despite antibiotic use. Approximately 75% of patients die before age 2 unless an allogenic hematopoietic stem cell transplantation is performed.
About Infantile Malignant Osteopetrosis
Infantile Malignant Osteopetrosis (IMO) is a severe form of osteopetrosis most commonly caused by a genetic mutation of the TCIRG1 gene which leads to ineffective osteoclast function. Osteoclasts play a vital role in maintaining bone growth health by breaking down bone tissue through the bone resorption process and maintaining equilibrium with bone generation. Impaired bone resorption causes increased bone mass and density, skeletal deformities, debilitating neurological abnormalities and bone marrow failure. Symptoms are typically present in the first year of life and the disorder is frequently fatal within the first decade of life unless treated with an allogenic hematopoietic stem cell transplantation.
About
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), Infantile
Malignant Osteopetrosis (IMO), and Danon disease 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
View source version on businesswire.com: https://www.businesswire.com/news/home/20190311005076/en/
Source:
Claudine Prowse, Ph.D.
SVP, Corporate Strategy and IRO
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
cp@rocketpharma.com
www.rocketpharma.com
investors@rocketpharma.com