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

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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 years meeting include 4 oral presentations and 3 poster presentations related to Rockets 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:

Information for Rockets 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 Rockets 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. Rockets 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. Rockets 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, 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.

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