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Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting
–Oral Presentation to Provide Update on Phase 1/2 Clinical Trial Data of RP-L201 for Leukocyte Adhesion Deficiency-I– –Poster Presentation to Highlight
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n–Oral Presentation to Provide Update on Phase 1/2 Clinical Trial Data of RP-L201 for Leukocyte Adhesion Deficiency-I–\n\n–Poster Presentation to Highlight Preclinical Data on RP-L401 for Infantile Malignant Osteopetrosis–\n\n NEW YORK--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).\n\nAdditional presentation details can be found below:\n\nOral Presentation\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): Results from Phase 1\nSession Title: Treatment\nPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles\nSession Date: Friday, October 16, 2020\nSession Time: 10:45 a.m. – 12:01 p.m. CEST\nLecture Time: 11:45 a.m. CEST\nLocation: Hall D\n\nThis session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST\n\nE-Poster\n\nTitle: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial Initiation\nPresenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden\n\nAbout Leukocyte Adhesion Deficiency-I\n\nSevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recur...