News Release Details
Rocket Pharmaceuticals Announces First Patient Dosed in Phase 1 Clinical Trial of RP-L102 in the U.S.
Mar 12, 2019
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- Trial to Use “Process B” RP-L102, Which Incorporates Higher Cell Doses, Transduction Enhancers, and Commercial-grade Vector Manufacturing and Cell Processing -
- Preliminary Data Expected by the End Of 2019 -
“The dosing of our first FA patient in our U.S. clinical trial of
RP-L102 represents a significant milestone for Rocket,” said
The Phase 1 clinical trial of “Process B” RP-L102 is expected to enroll
2 FA pediatric patients at the
About RP-L102 (LVV-based gene therapy for Fanconi Anemia)
RP-L102 is Rocket’s lentiviral vector (LVV)-based gene therapy in
development for patients with FA with Rocket’s collaboration partners at
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
(CIEMAT) in
About Fanconi Anemia
Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of solid tumors, mainly squamous cell carcinomas. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. The DEB assay can further differentiate FA patients from somatic mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1.
1Soulier, J., et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336
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
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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
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Source:
Claudine Prowse, Ph.D.
SVP, Corporate Strategy and IRO
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
investors@rocketpharma.com
www.rocketpharma.com