Business
Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)
—Incremental updates in LAD-I and PKD and previously disclosed data in FA to be presented— —Clinical updates across all programs anticipated later in Q4—
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Incremental updates in LAD-I and PKD and previously disclosed data in FA to be presented—\n\n—Clinical updates across all programs anticipated later in Q4—\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces data presentations at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) taking place virtually October 19-22, 2021. Oral presentations will include clinical data from Rocket’s lentiviral vector (LVV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Fanconi Anemia (FA). Elena Almarza, PhD, senior scientist at Rocket, will also give an Invited Talk on the multi-step development path for gene therapy in LAD-I, including vector design and comprehensive pre-clinical evaluation leading to robust clinical proof-of-concept.\n\nThese oral presentations will include interim data updates from additional patients in the RP-L201 trial for LAD-I, additional vector copy number data from the RP-L301 trial in PKD and previously disclosed data from RP-L102 in FA. Comprehensive updates for all the company's programs, which include Danon, FA, LAD-I, PKD and IMO, remain on track and are anticipated for later in Q4.\n\nDetails for Rocket’s oral presentations are as follows:\n\nTitle: A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results\nSession: Primary Immunodeficiencies (Session 2a)\nPresenter: Claire Booth, MBBS, PhD, FRCPCH, Infection, Immunity, & Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, United Kingdom\nDate: Wednesday, October 20, 2021\nSession Time: 9:00 a.m. – 11:00 a.m. CEST / 3:00 a.m. – 5:00 a.m. ET\nPresentation Time: 10:30 a.m. CEST / 4:30 a.m. ET\nPresentation Number: OR09\n\nRocket’s LAD-I trial will also be featured in an Invited Talk:\n\nTitle: Gene Therapy for Leukocyte Adhesion Deficiency Type I (LAD-I): A 12-year Journey\nSession: Primary Immunodeficiencies (Session 2a)\nPresenter: Elena Almarza, PhD, Senior Scientist, Rocket Pharmaceuticals\nDate: Wednesday, October 20, 2021\nSession Time: 9:00 a.m. – 11:00 a.m. CEST / 3:00...