Axelyf advances potent RNA delivery platform
“We’re not chasing science fiction, we’re contributing real solutions for tackling complex diseases”Örn Almarsson
The company’s lead candidate, AXL-003, is advancing towards preclinical evaluation in an autoimmune indication with liver-targeted delivery. This programme builds directly on results showing that AXL formulations exceed the potency of LP-01, a benchmark LNP currently in late-stage clinical development by firms such as Verve and Intellia.
In mouse studies, AXL LNPs encapsulating CRISPR components achieved approximately four- to five-fold higher gene editing efficiency in the liver at moderate doses compared to LP-01, without evidence of liver toxicity (see Figure 1).

The delivery system proved capable of supporting not only gene disruption but also gene insertion, with reduced immune stimulation, suggesting versatility across different gene therapy approaches.
»LNP delivery tends to be inflammatory and needs updates to drive potency with lower doses and improved immune system tolerability,« explains Örn Almarsson, PhD, CEO and co-founder of Axelyf and former head of drug delivery at Moderna.
The AXL lipid library comprises four distinct ionizable lipid series designed through targeted medicinal chemistry, with intellectual property consolidated in 2024 (see Figure 2).

These lipids exhibit enhanced potency, biodegradability and potential for extrahepatic delivery, addressing a recognised limitation of current LNP systems, which primarily accumulate in the liver. Axelyf is also developing surface chemistries for tissue-specific targeting, drawing on precedents such as ligand-decorated nanoparticles for directing delivery to T cells or neural tissue.
»We’re not chasing science fiction, we’re contributing real solutions for tackling complex diseases,« Örn Almarsson says. »With our AXL technology showing exceptional performance, our AI-informed capabilities and our team’s expertise, we can help unlock the next chapter in RNA medicine. This gives us a meaningful opportunity to generate transformative products, both for Axelyf and our partners.«
“The company is a rare combination of cutting-edge science, proven leadership and IP strength. This team can deliver on RNA medicine’s promise, and we are excited to be a part of the journey”Árni Blöndal
Complementing this chemistry-driven approach is ANNA, Axelyf’s artificial intelligence model trained to predict lipid performance. According to benchmarking against a 2024 Nature Biotechnology dataset, ANNA outperformed existing machine learning models for LNP optimisation, an area considered more challenging than nucleic acid or protein prediction due to the multi-component nature of formulations. The company has published a technical white paper describing ANNA’s architecture and applications.
Árni Blöndal, Founding Partner of Brunnur Ventures, which led the round, noted: »The company is a rare combination of cutting-edge science, proven leadership and IP strength. This team can deliver on RNA medicine’s promise, and we are excited to be a part of the journey.«
The seed financing round was led by Brunnur Ventures GP with participation from Icelandic firms Omega ehf and Silfurberg ehf. Axelyf is headquartered in Brookline, Massachusetts, with an R&D subsidiary in Iceland. The company was founded by Almarsson and John Lucas, with Yan Xia leading lipid–RNA nanotechnology development. Read more in Axelyf's recent press release.
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CLINICAL TRIALS
Sponsors:
Guangzhou Women and Children's Medical Center
Sponsors:
AMERICAN ORGAN TRANSPLANT AND CANCER RESEARCH INSTITUTE LLC