What are the key takeaways from this Beyond Biotech episode?

Key insights include: **CNS Delivery Route:** For Huntington's, Evox uses convection-enhanced delivery — a one-time neurosurgical procedure that infuses exosome-packaged gene editors directly into the striatum. This bypasses blood-brain barrier challenges entirely and maximizes drug exposure to the precise brain region affected. UniQure is pursuing the same delivery device for Huntington's approval, de-risking the approach clinically.; **MSH3 as Huntington's Target:** Rather than targeting the HTT gene directly, Evox knocks out MSH3, a DNA damage response gene that accelerates somatic CAG repeat expansion throughout a patient's lifetime. Eliminating MSH3 protein via CRISPR-Cas9 aims to halt repeat expansion before it reaches the neurotoxic threshold in medium spiny neurons of the striatum.; **Why Gene Editing Over RNA:** CRISPR-Cas9 can completely ablate a target protein in a single dose — a depth of knockdown that RNA therapeutics cannot reliably achieve. For targets like MSH3 where near-total protein reduction is required to meaningfully slow somatic expansion, gene editing provides a mechanistic advantage that other modalities cannot replicate.

What did Per London discuss on Beyond Biotech?

Evox Therapeutics CEO Per London explains how engineered exosomes deliver gene editors directly into the brain, bypassing the blood-brain barrier to target Huntington's disease via MSH3 knockout and ALS via intracisternal administration, with a clinical trial planned for the second half of 2027. Key topics include: **CNS Delivery Route:** For Huntington's, Evox uses convection-enhanced delivery — a one-time neurosurgical procedure that infuses exosome-packaged gene editors directly into the striatum. This bypasses blood-brain barrier challenges entirely and maximizes drug exposure to the precise brain region affected. UniQure is pursuing the same delivery device for Huntington's approval, de-risking the approach clinically.; **MSH3 as Huntington's Target:** Rather than targeting the HTT gene directly, Evox knocks out MSH3, a DNA damage response gene that accelerates somatic CAG repeat expansion throughout a patient's lifetime. Eliminating MSH3 protein via CRISPR-Cas9 aims to halt repeat expansion before it reaches the neurotoxic threshold in medium spiny neurons of the striatum..

How long is this episode of Beyond Biotech?

This episode is 31 minutes long. SignalCast provides an AI-generated summary so you can get the key insights in about 3 minutes.

Beyond Biotech

How Evox Therapeutics is targeting CNS diseases with exosomes

September 4, 2026

31 min episode · 2 min read

Per London

Episode

31 min

Read time

2 min

Topics

Investing, Fundraising & VC, Leadership

AI-Generated Summary

Published Sep 4, 2026

Key Takeaways

✓CNS Delivery Route: For Huntington's, Evox uses convection-enhanced delivery — a one-time neurosurgical procedure that infuses exosome-packaged gene editors directly into the striatum. This bypasses blood-brain barrier challenges entirely and maximizes drug exposure to the precise brain region affected. UniQure is pursuing the same delivery device for Huntington's approval, de-risking the approach clinically.
✓MSH3 as Huntington's Target: Rather than targeting the HTT gene directly, Evox knocks out MSH3, a DNA damage response gene that accelerates somatic CAG repeat expansion throughout a patient's lifetime. Eliminating MSH3 protein via CRISPR-Cas9 aims to halt repeat expansion before it reaches the neurotoxic threshold in medium spiny neurons of the striatum.
✓Why Gene Editing Over RNA: CRISPR-Cas9 can completely ablate a target protein in a single dose — a depth of knockdown that RNA therapeutics cannot reliably achieve. For targets like MSH3 where near-total protein reduction is required to meaningfully slow somatic expansion, gene editing provides a mechanistic advantage that other modalities cannot replicate.
✓Exosome IP as Competitive Moat: Evox built broad foundational IP from Oxford and Karolinska Institute patents starting in 2016, covering RNA delivery, protein delivery, targeted tissue delivery, and manufacturing processes. This portfolio has directly enabled business development transactions. Companies building platform delivery technologies should prioritize filing manufacturing and cargo-loading patents early, not just product-specific claims.
✓Indication Selection Framework: Evox selects CNS indications by identifying three converging factors: a clear genetic target with strong preclinical validation, a patient population large enough to support commercial viability, and a disease where gene editing achieves mechanistic superiority over existing modalities. Huntington's, ALS, and Rett syndrome each meet this criteria through distinct genetic drivers — MSH3, Ataxin-2, and MECP2 respectively.

What It Covers

Evox Therapeutics CEO Per London explains how engineered exosomes deliver gene editors directly into the brain, bypassing the blood-brain barrier to target Huntington's disease via MSH3 knockout and ALS via intracisternal administration, with a clinical trial planned for the second half of 2027.

Key Questions Answered

•CNS Delivery Route: For Huntington's, Evox uses convection-enhanced delivery — a one-time neurosurgical procedure that infuses exosome-packaged gene editors directly into the striatum. This bypasses blood-brain barrier challenges entirely and maximizes drug exposure to the precise brain region affected. UniQure is pursuing the same delivery device for Huntington's approval, de-risking the approach clinically.
•MSH3 as Huntington's Target: Rather than targeting the HTT gene directly, Evox knocks out MSH3, a DNA damage response gene that accelerates somatic CAG repeat expansion throughout a patient's lifetime. Eliminating MSH3 protein via CRISPR-Cas9 aims to halt repeat expansion before it reaches the neurotoxic threshold in medium spiny neurons of the striatum.
•Why Gene Editing Over RNA: CRISPR-Cas9 can completely ablate a target protein in a single dose — a depth of knockdown that RNA therapeutics cannot reliably achieve. For targets like MSH3 where near-total protein reduction is required to meaningfully slow somatic expansion, gene editing provides a mechanistic advantage that other modalities cannot replicate.
•Exosome IP as Competitive Moat: Evox built broad foundational IP from Oxford and Karolinska Institute patents starting in 2016, covering RNA delivery, protein delivery, targeted tissue delivery, and manufacturing processes. This portfolio has directly enabled business development transactions. Companies building platform delivery technologies should prioritize filing manufacturing and cargo-loading patents early, not just product-specific claims.
•Indication Selection Framework: Evox selects CNS indications by identifying three converging factors: a clear genetic target with strong preclinical validation, a patient population large enough to support commercial viability, and a disease where gene editing achieves mechanistic superiority over existing modalities. Huntington's, ALS, and Rett syndrome each meet this criteria through distinct genetic drivers — MSH3, Ataxin-2, and MECP2 respectively.

Notable Moment

London reveals that Evox's Huntington's strategy deliberately targets a gene upstream of HTT itself — not the mutation everyone has focused on for thirty years. The rationale is that stopping repeat expansion at its accelerant, MSH3, may be more transformative than reducing HTT protein directly.

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Episode Transcript

Hello, and welcome to Beyond Biotech, the weekly podcast from LeBiotech. I'm Dylan Kussain, and this is episode two thirteen for the podcast. Gene therapy has largely stayed out of the brain, and the blood brain barrier has been the reason why. It protects the brain from harm, but it also blocks most advanced medicines from ever reaching it. That's now beginning to change. The recent FDA approval of a brain penetrant enzyme therapy for Hunter syndrome showed that biologics can cross the barrier and work. The next question is whether gene editing can do the same. My guest today is Doctor. Per London, co founder and CEO of Evox Therapeutics, a UK biotech using engineered exosomes to deliver genetic medicines directly into the brain. EVOX is now extending their platform from RNA and biologics into gene editing, targeting the genetic drivers of diseases like Huntington's and ALS, where treatment options remain extremely limited. Per also brings a background as a European patent attorney and repeat biotech founder. We'll talk delivery, pipeline, and what a commercially scalable genetic medicine for the brain might actually look like. But first, a word from our friends at Inside Biotech. Hi, everyone. My name is Karish Machigani, and I'm the cohost of Inside Biotech, the official podcast of Biotech Connection Los Angeles or BCLA. For those who may not know, BCLA is a nonprofit organization dedicated to inspiring, educating, and connecting emerging scientists, students, and entrepreneurs to help grow and diversify the biotech ecosystem in Los Angeles. On Inside Biotech, we invite you to get a behind the scenes look at one of the fastest growing biotech hubs in the country. Each month, we sit down with scientists, founders, investors, and industry leaders to talk about the cutting edge science inside their companies and their personal journeys that brought them there. From breakthrough therapeutics and AI in health care to career transitions beyond academia, we explore how science, business, and storytelling intersect to shape the future of biotech and SoCal. If you're a student exploring careers in STEM, a young professional navigating the industry, or simply curious about the people driving innovation, this podcast is for you. Search Inside Biotech wherever you get your podcasts and give us a listen. We'd love to have you join us. Per, welcome to Beyond Biotech. Thanks, Dylan. Good to be here. Peyr, you've been involved in founding five biotech companies. What keeps pulling you back to starting something new rather than just staying inside and growing something over ten, twenty years? Well, yeah, it's a good question actually, Dylan. And to some extent with this company, I have stayed for ten years, but I do like new things, right? I think it's ultimately one thing that I think brings people to biotech more broadly, and I think that's, you know, hopefully making a difference for patients, right? And so I've been mostly engaged in companies working on advanced modalities, but it tends to be about unmet medical …

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