What are the key takeaways from this Beyond Biotech episode?

Key insights include: **Brain delivery strategy selection:** Three distinct approaches exist for CNS drug delivery, each with different trade-off profiles. EVOX uses convection-enhanced delivery directly into the striatum via neurosurgical procedure for a single-dose gene editing application in Huntington's disease. Arvanus achieves blood-brain barrier penetration through medicinal chemistry alone, demonstrated clinically with ARV-102 now in Phase 1 trials.; **Liver clearance as a platform-level problem:** Nanobiotics' nanoprimer approach addresses a systemic bottleneck affecting hundreds to thousands of therapies in development, including cell therapies, oncolytic viruses, and in vivo CAR-T. Injecting the nanoprimer IV before the therapeutic agent occupies the reticuloendothelial system long enough to allow the therapeutic to circulate, improving both efficacy and reducing liver toxicity.; **Human genetics as drug target validation:** Step Pharma's CTPS1 inhibitor program originated from a 2014 discovery of children with a loss-of-function CTPS1 mutation who could not mount viral immune responses. Because CTPS2 compensates in all other tissue types, selectively inhibiting CTPS1 blocks lymphocyte proliferation in lymphoma while sparing healthy tissue, resolving the toxicity barrier that blocked prior nucleotide synthesis inhibitors.

What did Per Lundin and Randy Thiel discuss on Beyond Biotech?

Beyond Biotech's summer recap episode features seven founders and executives from companies including EVOX, Arvanus, Nanobiotics, Step Pharma, Gain Therapeutics, Alterna, and Thirona, covering three core themes: novel drug delivery to the brain, platform science mechanisms, and the financing and partnership strategies keeping each program alive. Key topics include: **Brain delivery strategy selection:** Three distinct approaches exist for CNS drug delivery, each with different trade-off profiles. EVOX uses convection-enhanced delivery directly into the striatum via neurosurgical procedure for a single-dose gene editing application in Huntington's disease. Arvanus achieves blood-brain barrier penetration through medicinal chemistry alone, demonstrated clinically with ARV-102 now in Phase 1 trials.; **Liver clearance as a platform-level problem:** Nanobiotics' nanoprimer approach addresses a systemic bottleneck affecting hundreds to thousands of therapies in development, including cell therapies, oncolytic viruses, and in vivo CAR-T. Injecting the nanoprimer IV before the therapeutic agent occupies the reticuloendothelial system long enough to allow the therapeutic to circulate, improving both efficacy and reducing liver toxicity..

How long is this episode of Beyond Biotech?

This episode is 40 minutes long. SignalCast provides an AI-generated summary so you can get the key insights in about 3 minutes.

Beyond Biotech

The best biotech conversations you missed this summer

September 18, 2026

40 min episode · 2 min read

Per Lundin,Randy Thiel,Laurent Levy

Episode

40 min

Read time

2 min

Topics

Productivity, Health & Wellness, Relationships

AI-Generated Summary

Published Sep 18, 2026

Key Takeaways

✓Brain delivery strategy selection: Three distinct approaches exist for CNS drug delivery, each with different trade-off profiles. EVOX uses convection-enhanced delivery directly into the striatum via neurosurgical procedure for a single-dose gene editing application in Huntington's disease. Arvanus achieves blood-brain barrier penetration through medicinal chemistry alone, demonstrated clinically with ARV-102 now in Phase 1 trials.
✓Liver clearance as a platform-level problem: Nanobiotics' nanoprimer approach addresses a systemic bottleneck affecting hundreds to thousands of therapies in development, including cell therapies, oncolytic viruses, and in vivo CAR-T. Injecting the nanoprimer IV before the therapeutic agent occupies the reticuloendothelial system long enough to allow the therapeutic to circulate, improving both efficacy and reducing liver toxicity.
✓Human genetics as drug target validation: Step Pharma's CTPS1 inhibitor program originated from a 2014 discovery of children with a loss-of-function CTPS1 mutation who could not mount viral immune responses. Because CTPS2 compensates in all other tissue types, selectively inhibiting CTPS1 blocks lymphocyte proliferation in lymphoma while sparing healthy tissue, resolving the toxicity barrier that blocked prior nucleotide synthesis inhibitors.
✓Circular RNA manufacturing efficiency: Thirona's splint-free enzymatic ligation method for producing circular RNA avoids splicing byproducts entirely by splitting the IRES sequence and placing fragments on either side of the coding region. The linear precursor forms a secondary structure that brings the ends into proximity for ligation, producing scarless circular RNA without introducing additional sequences beyond the IRES and gene fragments.
✓Biotech partnership timing and triangulation: Arvanus maintained ongoing data-sharing conversations with major pharma partners for years before closing its Novartis androgen receptor degrader deal in 2024, with discussions predating the CEO's 2018 hire. Smaller biotechs should map the gap between investor time horizons and pharma partner horizons when deciding which programs to advance internally versus out-license.

What It Covers

Beyond Biotech's summer recap episode features seven founders and executives from companies including EVOX, Arvanus, Nanobiotics, Step Pharma, Gain Therapeutics, Alterna, and Thirona, covering three core themes: novel drug delivery to the brain, platform science mechanisms, and the financing and partnership strategies keeping each program alive.

Key Questions Answered

•Brain delivery strategy selection: Three distinct approaches exist for CNS drug delivery, each with different trade-off profiles. EVOX uses convection-enhanced delivery directly into the striatum via neurosurgical procedure for a single-dose gene editing application in Huntington's disease. Arvanus achieves blood-brain barrier penetration through medicinal chemistry alone, demonstrated clinically with ARV-102 now in Phase 1 trials.
•Liver clearance as a platform-level problem: Nanobiotics' nanoprimer approach addresses a systemic bottleneck affecting hundreds to thousands of therapies in development, including cell therapies, oncolytic viruses, and in vivo CAR-T. Injecting the nanoprimer IV before the therapeutic agent occupies the reticuloendothelial system long enough to allow the therapeutic to circulate, improving both efficacy and reducing liver toxicity.
•Human genetics as drug target validation: Step Pharma's CTPS1 inhibitor program originated from a 2014 discovery of children with a loss-of-function CTPS1 mutation who could not mount viral immune responses. Because CTPS2 compensates in all other tissue types, selectively inhibiting CTPS1 blocks lymphocyte proliferation in lymphoma while sparing healthy tissue, resolving the toxicity barrier that blocked prior nucleotide synthesis inhibitors.
•Circular RNA manufacturing efficiency: Thirona's splint-free enzymatic ligation method for producing circular RNA avoids splicing byproducts entirely by splitting the IRES sequence and placing fragments on either side of the coding region. The linear precursor forms a secondary structure that brings the ends into proximity for ligation, producing scarless circular RNA without introducing additional sequences beyond the IRES and gene fragments.
•Biotech partnership timing and triangulation: Arvanus maintained ongoing data-sharing conversations with major pharma partners for years before closing its Novartis androgen receptor degrader deal in 2024, with discussions predating the CEO's 2018 hire. Smaller biotechs should map the gap between investor time horizons and pharma partner horizons when deciding which programs to advance internally versus out-license.

Notable Moment

Gene Mac of Gain Therapeutics argues the Parkinson's field must abandon the MDS-UPDRS clinical scale as the sole FDA approval benchmark, contending that emerging technologies can measure disease state and progression more precisely, and that his Phase 2 trial already incorporates outside technology as secondary endpoints to capture what the standard scale misses.

Know someone who'd find this useful?

Episode Transcript

Hello, and welcome to Beyond Biotech, the weekly podcast from Le Biotech. I'm Dylan Kussain, and this is episode 215 for the podcast. Summer is a busy time for everyone, guests and listeners alike. So today, we're taking stock. This is our summer recap, an episode where we pull together the best moments from conversations you might have missed over the last few months. We'll hear from Randy Thiel of Arvanus and Per Lundin of Evox on two very different ways of getting drugs to the brain, and Lauren Levy of Nanobiotics on outsmarting the liver altogether. We'll meet the founders behind these companies, including Andy Parker of Step Pharma and Gene Mac of Gain Therapeutics, and the unlikely paths that got them there. We'll dig into the science of nonsense mutations with Narissa Krayer of Altina, circular RNA with Liu Gao of Theroner, and a cancer target hiding in human genetics. And we'll close with what those leaders think success actually looks like years from today. So sit back and let's revisit some of our favorite moments from the summer. But first, a word from our friends at Inside Biotech. Hi, everyone. My name is Karish Manjugani, and I am the co host of Inside Biotech, the official podcast of Biotech Connection Los Angeles or BCLA. For those who may not know, BCLA is a nonprofit 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 healthcare 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. A drug is only as good as its ability to actually reach the target. It's obvious, but it's also the single hardest unsolved problem in biotech right now, especially when the target is the brain. Over the next few minutes, three very different companies explain three very different answers to that same problem. First, Per Lundin from EVOX. Yes. So for our Huntington's program, for instance, we are using something called convection enhanced delivery. So essentially, we go directly into the brain using a device. So we're essentially for Huntington's, we utilise our exosome delivery system as a way to ensure that we maximise the spread of our gene editor in the context of the striatum. So this is done through a …

Get the full transcript (6,148 words) + summary by email — free

One-time email with the complete transcript and AI summary of this episode. No account needed.

One email, no spam. We’ll also show you what SignalCast does.

Browse all Beyond Biotech transcripts →