Our Programs
Two proprietary programs built on the AXL platform, targeting diseases with significant unmet need.
In Vivo CAR-T for Solid Tumors — AX-004
AX-004 is Axelyf's lead drug candidate: a non-viral, highly potent, redosable in vivo CAR-T therapy targeting solid tumors — an indication where no approved CAR-T treatment currently exists. Rather than removing a patient's T cells, engineering them in a lab, and reinfusing them, AX-004 uses an AXL lipid nanoparticle to deliver a proprietary antigen in mRNA-LNP, eliminating the manufacturing complexity of current CAR-T therapy and the one-and-done limitation that has kept it out of solid tumors entirely.
In CD5 humanized mouse models, AXL LNPs delivered RNA to circulating T cells at just 0.05 mg/kg, with full delivery effect retained on repeat dosing. In NHP studies, AXL achieved potent extrahepatic delivery with enhanced selectivity over the liver, confirming the platform can reach immune cells without off-target liver accumulation.
Precision mRNA Delivery for the Liver — AX-003
Where AX-004 targets immune cells beyond the liver, AX-003 uses the opposite capability: highly selective liver delivery. Designed for autoimmune hepatitis, the program delivers immune checkpoint-encoding mRNA directly to liver cells, dampening the autoimmune response at its source. Proof-of-concept was demonstrated in the ConA liver injury model, where a single dose reduced ALT/AST by five-fold vs. positive controls.
Partner with Us
Axelyf is exploring co-development and licensing opportunities for the AXL lipid library across therapeutic areas. If you're interested in best-in-class RNA-LNP delivery for your programs, we'd like to hear from you. Contact us at info@axelyf.com.