Motixafortide helped 11 of 15 sickle cell and thalassemia patients fail-safe to gene therapy, overcoming a key treatment barrier.

Ayrmid, Ltd. reported new real-world data from a 2025 study showing motixafortide, sold as APHEXDA®, successfully mobilized stem cells in 11 of 15 patients with sickle cell disease or beta-thalassemia who had failed prior plerixafor treatment, enabling access to gene therapies. Five patients received gene therapy and engrafted, with more in manufacturing. The drug worked alone in sickle cell disease and with G-CSF in beta-thalassemia, highlighting its potential to overcome a major barrier to curative treatments like Casgevy and Lyfgenia.