Exsilio debuts with $82M to develop redosable gene therapies

2024-06-25

基因疗法信使RNAsiRNA

A hallmark of the handful of gene therapies thus far approved has been their ‘one-and-done’ dosing. But Tal Zaks, interim CEO and chair of Exsilio Therapeutics, says that’s “more of a bug, not a feature.”

Zaks’ Boston-based newco emerged from stealth Tuesday with $82 million to create redosable gene therapies using mRNA and lipid nanoparticle (LNP) technologies.

A self-professed “R&D guy,” Zaks said the round was triggered because the company had generated robust data that justified transitioning Exsilio from mainly research-focused to begin development – that is, to start putting together a pipeline of medicines.

The financing was co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed – Exsilio’s seed funder – as well as Insight Partners, JP Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures and Deep Insight.

All-mRNA approach

Exsilio’s goal is to create genomic medicines for autoimmune and genetic diseases, as well as cancer, based on “naturally occurring, programmable genetic elements” that can be encoded in mRNA and delivered using LNPs.

“The challenge of current approaches in gene therapy is that while we all understand the potential for curative intent treatments, delivering them in a way that's both effective and safe has proven challenging,” Zaks explained.

Current offerings can’t be given more than once because they use viral vectors to deliver genetic material, which patients can then develop antibodies against – posing a problem if the therapy’s effect begins to wane. Plus, there’s no way to ensure the treatment ends up at the intended location in the genome. The exception is Krystal Biotech’s Vyjuvek (beremagene geperpavec), a redosable, topical gene therapy for the treatment of dystrophic epidermolysis bullosa, which uses a genetically modified herpes-simplex virus.

Exsilio is keeping much of its technology under wraps, but Zaks said it’s “the missing link” between viral vector-encapsulated DNA, and mRNA editing tools that are effective at eliciting small changes in the DNA, but struggle to incorporate an entire gene.

“We’re using an all-mRNA approach, which means that our elements are proteins, or they’re elements that encode an mRNA. And the trick is to bring in the right elements that would lead to the insertion of a gene transcribed into DNA,” he said.

The end goal, he added, is a therapy that can insert an entire gene in a safe locus in the genome, leads to a corrective or therapeutic function, and can be titrated to effect and redosed if needed.

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机构

Exsilio Therapeutics, Inc.