BioLineRx Ltd. announced a multi-center Phase 1 clinical trial sponsored  by St. Jude Children's Research Hospital, Inc. to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with sickle cell disease (SCD).  Investigators in the trial from St. Jude Children's Research Hospital, Inc. and two other clinical sites have extensive SCD gene therapy clinical development experience and are recognized leaders in the field.

Hematopoietic stem cell transplantation after genetic modification is potentially curative for patients with SCD.  Significant quantities of HSCs (minimum 16.5-20 million cells/kg) are required for genetic manipulation and transplant success, however, the most commonly used drug for collection of stem cells, granulocyte colony-stimulating factor (G-CSF), is contraindicated in patients with SCD.  Peripheral blood mobilization of stem cells using the mobilization agent plerixafor is the current strategy to collect HSCs for SCD gene therapies, however limitations exist including the need for multiple collection cycles to achieve the necessary HSC yields. For some, gene therapy may be prohibitive by the failure to obtain adequate numbers of HSCs. Enrollment in the St.

Jude Children's Research Hospital, Inc. study is expected to begin in the next few months.  Initial data from the Washington University School of Medicine in St. Louis sponsored clinical trial (ClinicalTrials.gov Identifier: NCT05618301) is anticipated in the second half of 2024.   Motixafortide, BioLineRx's lead therapeutic candidate, was approved by the U.S. Food & Drug Administration (FDA) in September 2023, in combination with filgrastim (G-CSF), to mobilize HSCs for collection and subsequent autologous transplantation in patients with multiple myeloma, under the brand name APHEXDA®.