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Rocket Pharmaceuticals Announces Upcoming Presentations at the European Society of Gene and Cell Therapy Annual Congress


—New Long-Term "Process A" Data from the Phase 1/2 EUROFANCOLEN Trial of RP-L102 to be Featured in Oral Session—

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, today announces data presentations at the upcoming European Society of Cell and Gene Therapy (ESGCT) 27th Annual Congress taking place October 22–25, 2019, in Barcelona, Spain. Presentations at this year's meeting include four oral presentations and one poster presentation related to Rocket's lentiviral pipeline programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD), Pyruvate Kinase Deficiency (PKD), and Infantile Malignant Osteopetrosis (IMO).

Details for Rocket's oral and poster presentations are as follows:

Title: Towards Haematopoietic Stem Cell-Targeted Gene Therapy of Infantile Malignant Osteopetrosis

Session Title: Skeletal Muscle & Bone Gene Therapy

Session Date: Wednesday, October 23, 2019

Session Time: 5:30 PM – 7:30 PM CEST

Room: 116-117

Title: Gene Therapy for Patients with Fanconi Anaemia

Session Title: Gene Therapy Clinical Trials II

Session Date: Thursday, October 24, 2019

Session Time: 8:30 AM - 10:30 AM CEST

Room: 113-117

Title: First Steps of a Lentiviral Gene Therapy Clinical Trial for Pyruvate Kinase Deficiency

Session Title: Blood Diseases

Session Date: Thursday, October 24, 2019

Session Time: 2:45 PM - 4:45 PM CEST

Room: 211

Title: Broad Applicability of NHEJ-Mediated Gene Editing to Correct Mutations in a Variety of Fanconi Anaemia Genes

Session Title: New Approaches in Gene Editing

Session Date: Friday, October 25, 2019

Session Time: 9:00 AM - 11:00 AM CEST

Room: 113-115

Title: Stable Transduction of Long-Term HSCs Under Optimized GMP-Conditions for the Gene Therapy of LAD-I Patients

Session Title: Poster Session II

Session Date: Thursday, October 24, 2019

Session Time: 1:15 PM - 2:45 PM CEST

Poster Number: P228

Full results from the ESGCT presentations will be available online at the conclusion of the presentation:

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 first two clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, and Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal. Rocket's first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rocket's pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO). 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, 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 pre-clinical 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.

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