Core Insights - BioLineRx Ltd. announced promising results from a Phase 1 clinical trial of motixafortide for mobilizing CD34+ hematopoietic stem cells in sickle cell disease, indicating potential for improved gene therapy access [1][2][4] Group 1: Clinical Trial Findings - The trial demonstrated that motixafortide, both alone and in combination with natalizumab, was safe and well-tolerated, with common adverse events being transient [3][4] - A median of 198 CD34+ cells/μl was mobilized with motixafortide alone, projecting the collection of 13.9 million HSCs in a single apheresis session [4] - In combination with natalizumab, a median of 231 CD34+ cells/μl was mobilized, projecting the collection of 18.6 million HSCs in a single session [4] Group 2: Comparison with Current Standards - Current gene therapies for sickle cell disease require 16.5 million and 22 million total CD34+ HSCs, with plerixafor being the standard mobilization agent, which often requires multiple attempts [5] - Patients previously mobilized with plerixafor experienced 2.8-fold greater HSC mobilization with motixafortide alone and 3.2-fold greater with the combination therapy [6] Group 3: Future Presentation and Research - An oral presentation detailing these findings will occur at the ASH 2024 Annual Meeting on December 7, 2024, in San Diego, California [1][6] - The trial is part of ongoing research to enhance treatment options for patients with sickle cell disease, with continued collaboration with Washington University [2][8]
BioLineRx Announces Oral Presentation on Data from Phase 1 Clinical Trial Evaluating Motixafortide for CD34+ Hematopoietic Stem Cell Mobilization for Gene Therapies in Sickle Cell Disease at ASH 2024