Market Overview

Rocket Pharmaceuticals Receives Funding from the California Institute for Regenerative Medicine (CIRM)


- $6.5mm Grant to Fund U.S. Phase I/II Leukocyte Adhesion
Deficiency-I Registration-Enabling Study -

Pharmaceuticals, Inc.
(NASDAQ:RCKT) ("Rocket"), a leading
U.S.-based multi-platform clinical-stage gene therapy company, today
announces that the California Institute for Regenerative Medicine (CIRM)
has awarded Rocket a $6.5mm CLIN2 grant award to support the clinical
development of gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I).
The CIRM was founded in 2004 following the passing of Proposition 71 or
the California Stem Cell Research and Cures Initiative, which allocated
$3 billion in state funding for stem cell research conducted in

Rocket's Investigational New Drug Application (IND) for RP-L201 was
accepted by the U.S. Food and Drug Administration in November 2018.
Proceeds from the grant will help fund clinical trial costs as well as
manufactured drug product for Phase I/II patients enrolled in the U.S.
clinical site, University of California, Los Angeles Mattel Children's
Hospital, led by PI Donald Kohn, M.D., UCLA 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 UCLA.
The trial will evaluate the safety and efficacy of the infusion of
autologous hematopoietic stem cells transduced with a lentiviral vector
encoding the ITGB2 gene.

"RP-L201 has the ability to address a significant unmet need in infants
and children who suffer from recurrent and life-threatening infections.
Pre-clinical data suggests RP-L201 can meaningfully increase CD18
expression and neutrophil migration and markedly increase potential
survival," said Gaurav Shah, M.D., Chief Executive Officer and President
of Rocket. "We are honored that CIRM has recognized the potential value
of RP-L201 and we look forward to dosing our first patient in the months

About Leukocyte Adhesion Deficiency-I

Severe leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal
recessive pediatric disease caused by a mutation of the ITGB2
gene that encodes 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 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 exceedingly rare. The unmet medical need for patients with
severe LAD-I is therefore significant.

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

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), 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.

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