News Release Details
Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Designations for RP-L102 Gene Therapy for Fanconi Anemia
Nov 27, 2018
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- Global Trial of RP-L102 Utilizing No Conditioning and “Process B” On Track for Early 2019 -
"The RMAT designation, which is the CBER equivalent of Breakthrough
Designation with the same benefits, and Fast Track designations
represent important milestones for the FA program and recognition of
RP-L102 as a potential treatment for this devastating disease,” said
The RMAT designation was established under the 21st Century
Cures Act to help expedite the development and approval of regenerative
medicine products, including cell and gene therapies. In order to be
considered for RMAT designation, products must be intended to treat,
modify, reverse, or cure a serious or life-threatening disease or
condition, as well as show preliminary clinical evidence indicating that
the product has the potential to address the unmet medical need. The
designation allows for companies to work closely with the
The
Rocket plans to initiate a clinical trial of RP-L102 for FA in early
2019 utilizing no conditioning and “Process B” which incorporates higher
cell doses, transduction enhancers, and commercial-grade vector
manufacturing and cell processing. The global trial 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 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, 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
www.rocketpharma.com
investors@rocketpharma.com