BioLineRx Ltd. announced that the first patient has been dosed in the Phase 1 clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD). The proof-of-concept trial, which plans to enroll five patients diagnosed with SCD, is being conducted in collaboration with Washington University School of Medicine in St. Louis and will assess the safety and tolerability of the two regimens.

Approved gene therapies rely on the collection of significant quantities of CD34+ hematopoietic stem cells to enable therapeutic manufacturing and backup storage. However, available mobilization regimens may not reliably yield desired numbers of HSCs for gene therapy, and the common mobilization agent G-CSF is contraindicated in patients with SCD. Difficulties in obtaining target quantities of HSCs may extend patient treatment journeys and increase patient and caregiver burdens.

Initial data from this study is expected 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 hematopoietic stem cells for collection and subsequent autologous transplantation in patients with multiple myeloma, under the brand name APHEXDA.