On November 6, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported presentations at the upcoming 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 7-10, 2019 in Orlando, Florida (Press release, Rocket Pharmaceuticals, NOV 6, 2019, View Source [SID1234550533]). The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing "Process B" for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

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Details for Rocket’s poster presentations are as follows:
Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells
Session Title: Gene Therapy and Transfer: Poster II
Presenter: Sandeep Soni, M.D.
Session Date: Sunday, December 8, 2019
Session Time: 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy
Session Title: Gene Therapy and Transfer: Poster III
Presenter: Paula Río, Ph.D.
Session Date: Monday, December 9, 2019
Session Time: 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Soni’s presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Río, Ph.D., Senior Scientist, División de Terapias Innovadoras en el Sistema Hematopoyético, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundación Jiménez Díaz will be participating in a Q&A panel. For further information, please contact [email protected].

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as ‘nature’s gene therapy’ provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336