Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

Rocket 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 28^th 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.

These 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.

Details for Rocket’s oral presentations are as follows:

Title: 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
Session: Primary Immunodeficiencies (Session 2a)
Presenter: Claire Booth, MBBS, PhD, FRCPCH, Infection, Immunity, & Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, United Kingdom
Date: Wednesday, October 20, 2021
Session Time: 9:00 a.m. – 11:00 a.m. CEST / 3:00 a.m. – 5:00 a.m. ET
Presentation Time: 10:30 a.m. CEST / 4:30 a.m. ET
Presentation Number: OR09

Rocket’s LAD-I trial will also be featured in an Invited Talk:

Title: Gene Therapy for Leukocyte Adhesion Deficiency Type I (LAD-I): A 12-year Journey
Session: Primary Immunodeficiencies (Session 2a)
Presenter: Elena Almarza, PhD, Senior Scientist, Rocket Pharmaceuticals
Date: Wednesday, October 20, 2021
Session Time: 9:00 a.m. – 11:00 a.m. CEST / 3:00 a.m. – 5:00 a.m. ET
Presentation Time: 9:00 a.m. CEST / 3:00 a.m. ET
Presentation Number: INV14

Title: Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials
Session: Hematopoietic & Bleeding Disorders I (Session 4c)
Presenter: Julián Sevilla, MD, PhD, Hematología y Hemoterapia, Fundación para la Investigación Biomédica HIUNJ, Hospital Infantil Universitario Niño Jesús, Madrid, Spain