News Release Details
Rocket Pharmaceuticals Announces FDA Clearance of IND Application for RP-L102 Gene Therapy for Fanconi Anemia
Nov 7, 2018
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- Patients to Be Treated with RP-L102 under “Process B” Incorporating Higher Cell Doses, Transduction Enhancers, and Commercial-grade Vector Manufacturing and Cell Processing –
- U.S. Trial to Commence Early 2019; No Conditioning Required -
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“Today marks Rocket’s first IND clearance by the
The planned clinical trial of “Process B” RP-L102 is expected to enroll
approximately 12 FA 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
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,
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
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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
cp@rocketpharma.com
www.rocketpharma.com
investors@rocketpharma.com