The AXL Platform: Precision Delivery for RNA Medicines
AXL is a next-generation lipid nanoparticle (LNP) platform engineered to deliver RNA medicines to the right place in the body with improved tissue selectivity and tolerability versus conventional LNP technologies.
The AXL library comprises four distinct ionizable lipid series, all of which outperformed LP-01 — the current Phase 3 LNP clinical benchmark — delivering up to 4x higher protein expression in head-to-head non-human primate studies and no signs of liver toxicity. Some formulations are liver-detargeted and spleen-selective, while others achieved best-in-class liver targeting, giving Axelyf a tunable platform across indications.
The platform is further sharpened by ANNA™, Axelyf's proprietary AI model for lipid design, which identifies high-performing LNP candidates from complex, real-world datasets.
In Vivo CAR-T for Solid Tumors
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
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.
Built for Multiple RNA Formats and Partners
The AXL platform has demonstrated superiority across four distinct RNA modalities, validated in collaboration with pharma and biotech partners. In addition to growing its own pipeline, Axelyf is partnering with companies seeking best-in-class LNP delivery for their own RNA programs.
The Intelligence Behind the Platform: ANNA™
Predicting LNP performance with AI is harder than it sounds. The available data in the field is fragmented and inconsistent, making it difficult for most AI models to learn from unless it's manually normalized.
Axelyf built ANNA™ (Artificial Network for Nanoparticle Assessment), which works directly with data in its original form and across multiple studies at once. When benchmarked against AGILE and LiON — two recently published models — ANNA outperformed both in identifying high-performing lipid candidates, particularly when data was fragmented across smaller studies, which is the typical real-world condition.
The model enables Axelyf to screen virtual lipid libraries and prioritize candidates for synthesis and testing with greater confidence. ANNA is part of how the AXL platform gets sharper over time.
ANNA™
Optimization through artificial intelligence
Predictive Lipid Design
Outperforms AGILE & LiON in fragmented data environments
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.