Company: BLRX
Filing Date: 2025-03-31
Form Type: 20-F
Source: 0001178913-25-001123
Chunk: 66

Company: BioLineRx Ltd.
Filing Date: 2025-03-31
Form: 20-F
Item: Item 3
Chunk 66
---
 affected by SCD. SCD arises from mutations in the hemoglobin gene, ultimately leading to the production of abnormally
shaped (sickle) red blood cells that tend to stick within blood vessels causing their occlusion. The clinical manifestations of SCD include
anemia and blood vessel occlusion which can lead to both acute and chronic pain, as well as tissue ischemia across multiple organ systems
(e. g., stroke, heart attack, respiratory failure), ultimately compromising end organ function. The cumulative impact of these complications
significantly impacts morbidity and mortality for patients with SCD.

Effective HSC-based gene therapies depend upon the collection of
significant quantities of stem cells to engineer the treatments that enable the potential genetic treatment of SCD. Currently available
mobilization regimens can carry serious risk and side effects for patients with SCD or may not reliably yield optimal numbers of HSCs
for gene therapy. Peripheral blood mobilization of stem cells using the mobilization agent plerixafor is the current strategy to
collect HSCs for SCD gene therapies.

37

In March 2023, we entered into a clinical collaboration with Washington
University School of Medicine in St. Louis to advance a Phase 1 clinical trial in which motixafortide will be evaluated as a monotherapy
and in combination with natalizumab (VLA-4 inhibitor), as novel regimens to mobilize CD34+ hematopoietic stem cells (HSC) for gene therapies
in Sickle Cell Disease. The proof-of-concept investigator-initiated study plans to enroll five adults with a diagnosis of SCD who are
receiving automated red blood cell exchanges via apheresis. The trial’s primary objective is to assess the safety and tolerability
of motixafortide alone and in combination with natalizumab in SCD patients, defined by dose-limiting toxicities. Secondary objectives
include determining the number of CD34+ hematopoietic stem and progenitor cells (HSPCs) mobilized via leukapheresis; and determining the
pharmacokinetics of CD34+ HSPCs mobilization to peripheral blood in response to motixafortide alone and motixafortide plus natalizumab
in SCD patients. As anticipated, the study began enrolling in 2023, with first patient dosed in December 2023, and is ongoing. Initial
data from this study was presented at an oral presentation at the 66th