— Includes Updates from LAD-I, PKD and FA
Clinical Trials —
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage
company advancing an integrated and sustainable pipeline of genetic
therapies for rare childhood disorders, today announces clinical
data presentations at the upcoming 24thAmerican Society of Gene and
Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021.
Investigators will review new data from Rocket’s Leukocyte Adhesion
Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi
Anemia (FA) gene therapy programs in oral and poster
presentations.
Details for oral presentations are as follows:
Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo
Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion
Deficiency-I (LAD-I): Interim Results Session: Genetic Blood
and Immune Disorders Presenter: Donald Kohn, M.D., Professor
of Microbiology, Immunology and Molecular Genetics, Pediatrics
(Hematology/Oncology), Molecular and Medical Pharmacology, and
member of the Eli and Edythe Broad Center of Regenerative Medicine
and Stem Cell Research at the University of California, Los Angeles
Date: Tuesday May 11, 2021 Time: 6:15-6:30 p.m. EDT
Location: Room 7 Abstract number: 39
Title: Lentiviral Mediated Gene Therapy for Pyruvate
Kinase Deficiency: Updated Results of a Global Phase 1 Study for
Adult and Pediatric Patients Session: Gene Therapies for
Hemoglobinopathies Presenter: José Luis López Lorenzo, M.D.,
Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
Date: Wednesday May 12, 2021 Time: 6:45-7:00 p.m. EDT
Location: Room 7 Abstract number: 83
Title: Gene Therapy in Fanconi Anemia: Current Strategies
to Enable the Correction of HSCs Session: International
Focus on Stem Cell Gene Therapy Presenter: Juan A. Bueren,
Ph.D., Head of the Hematopoietic Innovative Therapies Division at
the Centro de Investigaciones Energéticas, Medioambientales y
Tecnológicas (CIEMAT) in Spain / CIBER-Rare Diseases /
IIS-Fundación Jiménez Díaz Date: Thursday, May 13, 2021
Time: 10:00-10:45 a.m. EDT Location: Room 7
Abstract number: 36
Select results from Dr. Bueren’s presentation will also be
highlighted by Paula Rio, Ph.D. Details for this Invited
Presentation are as follows:
Title: Gene Therapy in Fanconi Anemia: Current Strategies
to Enable the Correction of HSCs Session: International
Focus on Stem Cell Gene Therapy Presenter: Paula Río, Ph.D.,
Senior Researcher, Hematopoietic Innovative Therapies Division at
CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz
Date: Thursday May 13, 2021 Time: 10:00-11:45 a.m.
EDT
Details for poster presentation are as follows:
Title: Gene Therapy for Fanconi Anemia [Group A]:
Preliminary Results of Ongoing RP-L102 Clinical Trials
Session: Hematologic and Immunologic Diseases
Presenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant
Professor of Pediatrics, Division of Stem Cell Transplantation,
Stanford University School of Medicine Date: Tuesday, May
11, 2021 Time: 8:00-10:00 a.m. EDT Location: Digital
Gallery Abstract number: 697
Abstracts for the presentations can be found online at:
https://annualmeeting.asgct.org/
About Leukocyte Adhesion Deficiency-I
Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare,
autosomal recessive pediatric disease caused by mutations in the
ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is
a key protein that facilitates leukocyte adhesion and extravasation
from blood vessels to combat infections. As a result, children with
severe LAD-I are often affected immediately after birth. During
infancy, they suffer from recurrent life-threatening bacterial and
fungal infections that respond poorly to antibiotics and require
frequent hospitalizations. Children who survive infancy experience
recurrent severe infections including pneumonia, gingival ulcers,
necrotic skin ulcers, and septicemia. Without a successful bone
marrow transplant, mortality in patients with severe LAD-I is
60-75% prior to the age of 2 and survival beyond the age of 5 is
uncommon. There is a high unmet medical need for patients with
severe LAD-I.
Rocket’s LAD-I research is made possible by a grant from the
California Institute for Regenerative Medicine (Grant Number
CLIN2-11480). The contents of this press release are solely the
responsibility of Rocket and do not necessarily represent the
official views of CIRM or any other agency of the State of
California.
About Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood
cell disorder resulting from a mutation in the PKLR gene encoding
for the pyruvate kinase enzyme, a key component of the red blood
cell glycolytic pathway. Mutations in the PKLR gene result in
increased red cell destruction and the disorder ranges from mild to
life-threatening anemia. PKD has an estimated prevalence of 3,000
to 8,000 patients in the United States and the European Union.
Children are the most commonly and severely affected subgroup of
patients. Currently available treatments include splenectomy and
red blood cell transfusions, which are associated with immune
defects and chronic iron overload.
RP-L301 was in-licensed from the Centro de Investigaciones
Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de
Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and
Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
(IIS-FJD).
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. Graft-versus-host disease, a known
complication of allogeneic HSCT, is associated with an increased
risk of solid tumors, mainly squamous cell carcinomas of the head
and neck region. Approximately 60-70% of patients with FA have a
Fanconi Anemia complementation group A (FANCA) gene mutation, which
encodes for a protein essential for DNA repair. Mutation in the
FANCA gene leads to chromosomal breakage and increased sensitivity
to oxidative and environmental stress. Increased sensitivity to
DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane
(DEB) is a ‘gold standard’ test for FA diagnosis. Somatic mosaicism
occurs when there is a spontaneous correction of the mutated gene
that can lead to stabilization or correction of a FA patient’s
blood counts in the absence of any administered therapy. Somatic
mosaicism, often referred to as ‘natural gene therapy’ provides a
strong rationale for the development of FA gene therapy because of
the selective growth advantage of gene-corrected hematopoietic stem
cells over FA cells.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an
integrated and sustainable pipeline of genetic therapies that
correct the root cause of complex and rare childhood disorders. The
Company’s platform-agnostic approach enables it to design the best
therapy for each indication, creating potentially transformative
options for patients afflicted with rare genetic diseases. Rocket's
clinical programs using lentiviral vector (LVV)-based gene therapy
are for the treatment of Fanconi Anemia (FA), a difficult to treat
genetic disease that leads to bone marrow failure and potentially
cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric
genetic disorder that causes recurrent and life-threatening
infections which are frequently fatal, Pyruvate Kinase Deficiency
(PKD), a rare, monogenic red blood cell disorder resulting in
increased red cell destruction and mild to life-threatening anemia,
and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived
disorder. Rocket’s first clinical program using adeno-associated
virus (AAV)-based gene therapy is for Danon disease, a devastating,
pediatric heart failure condition. For more information about
Rocket, please visit www.rocketpharma.com.
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 its guidance for 2021 in light of
COVID-19, the safety, effectiveness and timing of product
candidates that Rocket may develop, 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 monitor the impact of COVID-19 on its business
operations and take steps to ensure the safety of patients,
families and employees, the interest from patients and families for
participation in each of Rocket’s ongoing trials, our expectations
regarding the delays and impact of COVID-19 on clinical sites,
patient enrollment, trial timelines and data readouts, our
expectations regarding our drug supply for our ongoing and
anticipated trials, 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 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 December 31, 2020, filed
March 1, 2021 with the SEC. Accordingly, you should not place undue
reliance on these forward-looking statements. All such statements
speak only as of the date made, and Rocket undertakes no obligation
to update or revise publicly any forward-looking statements,
whether as a result of new information, future events or
otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20210427006049/en/
Claudine Prowse, Ph.D. SVP, Strategy & Corporate Development
investors@rocketpharma.com
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