News Release Details
Rocket Pharmaceuticals Announces Presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
Apr 30, 2018
|
- Multiple presentations at ASGCT Highlight Potential of Rocket’s Multi-Platform Approach to Address Devastating Rare Diseases with Gene Therapy -
- Updated Data of Ongoing Phase 1/2 Trial in Fanconi Anemia (FA) Showcased at Presidential Symposium -
“Rocket has a robust pipeline of five innovative gene therapy programs
across lentiviral vector (LVV) and adeno-associated viral vector (AAV)
platforms, and we are pleased that our programs to treat Fanconi Anemia
(FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase
Deficiency (PKD) are featured at ASGCT,” said
Oral Presentations:
Title: Engraftment
and Phenotypic Correction of Hematopoietic Stem Cells in Non-Conditioned
Fanconi Anemia Patients Treated with Ex Vivo Gene Therapy
Session:
330 Presidential Symposium & Presentation of the Top Abstracts
Presenter:
Date:
Session Time:
Presentation Time:
Location: International Ballroom North & South
Title: Immunotoxin-Based
Conditioning Facilitates Autologous Hematopoietic Stem Cell Engraftment
and
Session:
115 Hematopoietic Cell Therapies
Presenter:
Date:
Session
Time:
Presentation
Time:
Location: Salon
A-5
Title: Gene
Editing in Fanconi Anemia Hematopoietic Stem and Progenitor Cells
Session:
302 Advances in Genome Editing in HSCs - Organized by the Hematologic
and Immunologic Gene and Cell Therapy Committee
Presenter:
Date:
Session Time:
Presentation
Time:
Location:
International Ballroom North
Poster Presentations:
Title: Improvements
in the Transduction Conditions of Human Hematopoietic Progenitors with
the CPcoRPKW-17 Therapeutic Lentiviral Vector to be Used in a Pyruvate
Kinase Deficiency Gene Therapy Clinical Trial
Session: Hematologic
& Immunologic Diseases I
Date:
Time:
Location:
Title: Leukocyte
Adhesion Deficiency I: A Closer Step to a Gene Therapy Clinical Trial
Session:
Hematologic & Immunologic Diseases I
Date:
Time:
Location: Stevens Salon C, D
Title: Pairs
of Guide RNAs Mediate Precise Deletions on the PKLR Gene via Non
Homologous End Joining Generating a Human Hematopoietic Progenitor Model
of Pyruvate Kinase Deficiency
Session: Hematologic &
Immunologic Diseases II
Date:
Time:
Location:
Title: Leukocyte
Adhesion Deficiency-I: A Comprehensive Review of Published Cases
Session:
Hematologic & Immunologic Diseases III
Date:
Time:
Location:
Stevens Salon C, D
Title: Towards
the Gene Therapy Clinical Trial for Pyrivate Kinase Deficiency
Session:
Hematologic & Immunologic Diseases III
Date:
Time:
Location:
Stevens Salon C, D
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
products 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, 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/20180430006197/en/
Source:
Claudine Prowse, Ph.D.
SVP Corporate Development and IRO
Rocket
Pharmaceuticals, Inc.
The Alexandria Center for Life Science
430
East 29 Street, Suite 1040
New York, NY 10016
cp@rocketpharma.com