Rocket Pharmaceuticals Announces Presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Title: Immunotoxin-Based Conditioning Facilitates Autologous Hematopoietic Stem Cell Engraftment and Multi-Lineage Development in a Fanconi Anemia Mouse Model
Session: 115 Hematopoietic Cell Therapies
Presenter: Meera Srikanthan, M.D., Seattle Children's Hospital; Fred Hutchinson Cancer Research Center
Date: Wednesday, May 16, 2018
Session Time: 10:30 a.m. – 12:00 p.m. Central Time
Presentation Time: 10:45 a.m. – 11:00 a.m. Central 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: Paula Rio, Ph.D., Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Date: Friday, May 18, 2018
Session Time: 8:00 a.m. -10:00 a.m. Central Time
Presentation Time: 9:00 a.m. – 9:30 a.m. Central 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: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency I: A Closer Step to a Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central 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: Thursday, May 17, 2018
Time: 5:15 p.m. – 7:15 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency-I: A Comprehensive Review of Published Cases
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

Title: Towards the Gene Therapy Clinical Trial for Pyrivate Kinase Deficiency
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

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. Preclinical studies of additional bone marrow-derived disorders are ongoing and target 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 an undisclosed rare pediatric disease. For more information about Rocket, please visit www.rocketpharma.com.

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 December 31, 2017. 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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