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Rocket Pharmaceuticals Announces Upcoming Presentations at the American Society of Gene and Cell Therapy 2019 Annual Meeting

– 4 oral presentations and 3 poster presentations focus on Rocket’s
clinical and preclinical programs –

NEW YORK–(BUSINESS WIRE)–Rocket
Pharmaceuticals, Inc.
(Nasdaq:RCKT) (“Rocket”), a leading U.S.-based
multi-platform gene therapy company, today announces data presentations
at the upcoming American Society of Gene and Cell Therapy (ASGCT) 2019
Annual Meeting taking place April 29 – May 2, 2019, in Washington, D.C.
Presentations at this year’s meeting include 4 oral presentations and 3
poster presentations related to Rocket’s four leading pipeline programs:
lentiviral vector (LVV) based gene therapy programs for Fanconi Anemia
(FA), Pyruvate Kinase Deficiency (PKD), and Leukocyte Adhesion
Deficiency-I (LAD-I) and adeno-associated viral vector (AAV) based gene
therapy program for Danon disease.

“Rocket is pleased to share additional emerging preclinical and clinical
data from several gene therapy pipeline programs at ASGCT,” said Gaurav
Shah, M.D. Chief Executive Officer and President of Rocket. “This
includes updated long-term data from the ongoing Phase 1/2 trial of
RP-L102 for FA that utilizes ‘Process A’ without the use of
myeloablative conditioning. Results for the four patients who have been
followed for at least one year and up to three years show increasing and
durable engraftment in peripheral blood and bone marrow, and restoration
of bone marrow hematopoietic stem cell function, which we believe
suggests patients are approaching an FA mosaic phenotype. These
long-term data further support the clinical development of RP-L102 as a
potential therapeutic option for FA patients as a means of averting bone
marrow failure and avoiding the need for a more toxic bone marrow
transplantation.”

Dr. Shah continued, “Data from our preclinical toxicology studies of
RP-A501 for Danon disease in mice and non-human primates showed no
treatment-related adverse events or safety issues up to the highest
dose. There was also robust vector copy number detection in the organs
most affected in Danon disease, including high concentrations in heart
tissue (~10x higher on average than in skeletal muscle and CNS). We are
encouraged by the safety profile of RP-A501 and look forward to
initiating our Phase 1 clinical trial of the program in the second
quarter of 2019.”

Details on all abstracts accepted for presentation can be found online: https://www.tripbuilder.net/html5/asgct2019/home.php

Information for Rocket’s oral presentations:

Title: Preliminary Conclusions Obtained in Fanconi Anemia
Patients Treated by Lentiviral-mediated Gene Therapy after 2 years of
Follow-up
Session Title: Clinical Gene Therapies for Blood
Diseases
Session Date/Time: Monday April 29, 2019 10:30 AM –
12:00 PM
Presentation Time: 11:00 AM – 11:15 AM

Title: Genome-Wide Assessment of Lentiviral Integration Sites of
Gene-Corrected Llympho-Hematopoietic Cells in FA-A Patients
Session
Title:
 Clinical Gene Therapies for Blood Diseases
Session
Date/Time:
Monday April 29, 2019 10:30 AM – 12:00 PM
Presentation
Time:
 11:15 AM – 11:30 AM
Room: Jefferson Room

Title: AAV9.LAMP2B Reverses Metabolic and Physiologic Multiorgan
Dysfunction in Murine Model of Danon Disease
Session Title:
Cardiovascular Gene Therapy
Session Date/Time: Monday April
29, 2019 1:30 PM – 3:00 PM
Presentation Time: 1:30 PM – 1:45
PM
Room: Heights Courtyard 1

Title: Systemic Delivery of AAV9.LAMP2B for the Treatment of
Danon Disease: Toxicology Studies in Mice and Cynomolgus Monkeys
Session
Title:
AAV Vectors and Disease Targets II
Session Date/Time:
Thursday May 2, 2019 10:15 AM – 12:15 PM
Presentation Time:
11:00 AM – 11:15 AM
Room: Monroe Room

Information for Rocket’s poster presentations:

Title: Efficient Gene Correction of AGXT Mutations Causing
Primary Hyperoxaluria Type 1 in Patient-Derived Fibroblasts
Session
Title:
Metabolic, Storage, Endocrine, Liver and Gastrointestinal
Diseases
Session Date/Time: Tuesday April 30, 2019 5:00 PM –
6:00 PM
Room: Columbia Hall

Title: Finding the Minimum Vector Copies Per Cell Needed to Reach
Phenotypic Correction in a Mouse Model of Erythrocyte Pyruvate Kinase
Deficiency Using a Clinically Applicable Lentiviral Vector
Session
Title:
Hematologic & Immunologic Diseases II
Session
Date/Time:
Wednesday May 1, 2019 5:00 PM – 6:00 PM
Room:
Columbia Hall

Title: Gene Therapy of Patients with Leukocyte Adhesion
Deficiency Type I (LAD-I): Preclinical Studies and Clinical Trial Design
Session
Title:
Hematologic & Immunologic Diseases II
Session
Date/Time:
Wednesday May 1, 2019 5:00 PM – 6:00 PM
Room:
Columbia Hall

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT) (“Rocket”) is an emerging,
clinical-stage biotechnology company focused on developing
first-in-class gene therapy treatment options for rare, devastating
diseases. Rocket’s multi-platform development approach applies the
well-established lentiviral vector (LVV) and adeno-associated viral
vector (AAV) gene therapy platforms. Rocket’s lead clinical program is a
LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a
difficult to treat genetic disease that leads to bone marrow failure and
potentially cancer. Rocket’s additional pipeline programs for bone
marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD),
Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant
Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy
program for a devastating, pediatric heart failure indication, Danon
disease. 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 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 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 December 31, 2018. 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.

Contacts

Claudine Prowse, Ph.D.
SVP, Strategy, Corporate Development and IRO
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
www.rocketpharma.com
[email protected]

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