Rocket Pharmaceuticals, Inc.(NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, today announces that the first patient has been dosed in the open-label, Phase 1 clinical trial of RP-L102, the Company's lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA). The patient was dosed at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, the lead U.S. clinical site. The clinical trial will evaluate "Process B" RP-L102 which incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing.

"The dosing of our first FA patient in our U.S. clinical trial of RP-L102 represents a significant milestone for Rocket," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We believe RP-L102 can alter the current treatment landscape for FA patients by providing a one-time, potentially curative therapeutic option as compared to more toxic bone marrow transplants. We look forward to sharing initial data from this trial and 'Process B' at the end of 2019."

The Phase 1 clinical trial of "Process B" RP-L102 is expected to enroll 2 FA pediatric patients at the Center for Definitive and Curative Medicine at Stanford. The study is designed to assess the safety and tolerability of a single infusion of PR-L102, as well as efficacy endpoints. The Phase 2 study is expected to begin in the second half of 2019 following final alignment with the U.S. Food and Drug Administration (FDA) on clinical endpoints. Patients will not undergo conditioning in this trial. Further information is available here.

RP-L102 is Rocket's lentiviral vector (LVV)-based gene therapy in development for patients with FA with Rocket's collaboration partners at Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain, CIBER-Rare Diseases and IIS-Fundación Jiménez Díaz. The International Fanconi Anemia Gene Therapy Working Group helped the development of new generation of FA gene therapy programs, which began with a HIV-1-derived, self-inactivating lentiviral vector. RP-L102's lentiviral vector carries the FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which includes a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The ex vivo administration process begins with the removal and isolation of hematopoietic stem cells using a CD34+ selection process. Autologous genetically modified CD34+ enriched hematopoietic cells (fresh or cryopreserved) are infused back into patients to restore function. RP-L102 is currently being studied in a Phase 1/2 clinical trial in the European Union with an Investigational Medicinal Product Dossier (IMPD) in place with the Spanish Agency for Medicines and Health Products. RP-L102 is also being studied in a Phase 1 clinical trial in the U.S. under the Company's Investigational New Drug (IND) application for RP-L102 that utilizes "Process B" which incorporates higher cell doses, transduction enhancers, and commercial-grade vector and cell processing. RP-L102 has been granted Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations for the treatment of Fanconi Anemia type A in the United States and Advanced Therapy Medicinal Product (ATMP) classification and Orphan Drug designation in Europe.

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