Rocket Pharmaceuticals Sponsors 3rd Annual PKD Patients’ Forum

NEW YORK–(BUSINESS WIRE)–Rocket
Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading
U.S.-based multi-platform clinical-stage gene therapy company, today
sponsors the 3^rd Annual Pyruvate Kinase Deficiency (PKD) Day
in Madrid, Spain, a day devoted to patients, families, clinicians and
global scientific experts. This event is co-sponsored by Centro de
Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT),
Centro de Investigación Biomédica en Red de Enfermedades Raras
(CIBERER), Instituto de Investigación Sanitaria Fundación Jiménez Díaz
(IIS-FJD) and Hospital Universitario Infantil Niño Jesús. Rocket has
in-licensed RP-L301, a lentiviral vector (LVV)-based gene therapy for
the treatment of PKD, developed by CIEMAT-CIBERER/IIS-FJD and Dr.
José-Carlos Segovia, leader of the project and PKD Scientific Advisor.
Rocket anticipates RP-L301 will enter the clinic during the second half
of this year.

“At Rocket, we are making significant progress towards developing
transformative gene therapies for patients living with devastating, rare
diseases, including PKD,” said Kinnari Patel, Pharm.D., MBA, Chief
Operating Officer and Executive Vice President of Development of Rocket.
“We are proud to work with our partners at CIEMAT-CIBERER/IIS-FJD and
other institutions worldwide to shine a spotlight on PKD’s impact on
patients’ lives as we advance towards a potential cure.”

“We are pleased to sponsor the 3^rd Annual PKD Patients’ Forum
to discuss PKD diagnosis and present and future treatments, including
the forthcoming gene therapy clinical trial for PKD,” said Dr. Segovia.
“Internationally recognized PKD experts, as well as clinicians in close
contact with the patients are attending today’s Forum. Our objective is
to educate families and patients and discuss their physical,
psychological and social needs.”

Dr. Segovia continued, “Patients with severe PKD suffer greatly as the
only treatment options currently available to them are splenectomy and
lifelong red blood cell transfusions. Unfortunately, these treatment
options are far from optimal and are associated with considerable side
effects. The experts at this event have devoted countless years in
research and are an instrumental resource to patients and their
families.”

Sponsoring PKD Day is part of Rocket’s commitment to promoting rare
disease education and awareness. Additional recent initiatives include a
Rocket-sponsored Rare Disease Day educational event on February 28 to
show support for those living with rare diseases, like PKD. To celebrate
this significant milestone, for the first time ever, The Empire State
Building, Rocket’s headquarters, shined its world-famous lights in Rare
Disease Day colors. For more information about Rare Disease Day hosted
by Rocket, go to https://www.rocketpharma.com/patients-and-families/rare-disease-day/

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.

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, 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
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
www.rocketpharma.com
[email protected]