AI Summary
5 min readGene Therapies That Work? A Ground-Level View
Adrian Veres, co-founder and chief science officer at Dyno Therapeutics, has spent years on what he calls "the biggest challenge that's held back gene therapy"—not the gene editing itself, but getting the editing machinery to the right cells in the body. His company uses machine learning to engineer the protein shells of adeno-associated viruses (AAVs), turning them into precision delivery vehicles that can bypass the liver and reach targets like the brain, muscle, and eyes. The work sits at the intersection of biology and AI, and Veres argues that the field is finally converging on solutions that could make genetic medicine routine rather than miraculous.
What Gene Therapy Actually Means
A gene therapy, Veres explains, is simply "acting at the genetic level to improve health." The appeal is obvious: if a disease traces back to a specific genetic error, fixing that error directly should be more powerful than managing symptoms with a pill. But the path from identifying a faulty gene to correcting it has been brutally slow. "We've been wanting to do this for a very long time," Veres says. "It turns out it wasn't so easy to just will it into action."
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What you'll learn
- 1 (00:00) **Episode Introduction: The Promise & Problem of Gene Therapy** - Host Ashlee Vance frames the long-standing hype around gene therapy and its central challenges: cost, production, and delivery to the right body parts.
- 2 (03:50) **Defining Gene Therapy & The Delivery Bottleneck** - Adrian explains gene therapy as intervening at the genetic level to improve health, contrasting it with small molecules that manage symptoms.
- 3 (12:10) **Current Focus: Monogenic Diseases & The Path Forward** - Adrian clarifies that the field is starting with single-gene (monogenic) diseases because the causal biology is crystal clear, but this is just the beginning.
- 4 (14:30) **The Delivery Toolbox: Lipid Nanoparticles vs. Viral Vectors** - Adrian outlines the two main methods for getting therapeutic DNA/RNA into cells: chemical formulations (like LNP) and engineered viruses.
- 5 (17:07) **Engineering the Capsid: The "Origami Box"** - Adrian explains the capsid (protein shell) of AAV in simple terms, describing it as a 700-amino-acid string that folds into an icosahedral box.
- 6 (21:29) **The Dyno Approach: AI + In Vivo Data Loops** - Adrian describes Dyno's core process: creating a million different capsid variants, barcoding them, injecting them into an animal, and sequencing which barcodes end up in which tissues.
- 7 (27:48) **Ground Truth in Animals & The Barcoding System** - Adrian explains why Dyno tests in animals (especially primates) from the very beginning to get "ground truth" data on delivery.
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Show Notes
We have been promised glorious gene therapies for decades. This is supposed to be the stuff of reprogramming the body to blunt or cure diseases with something approaching a permanent fix. While gene therapy progress has been made, treatments remain rare and expensive, and it’s very tough to get the therapies to go into the desired parts of the body.
Our guest this week is here to help. He’s Adrian Veres, the chief scientific officer and co-founder of Dyno Therapeutics. Founded in 2018, Dyno has been pursuing new and better ways to deliver gene therapies via modified viruses.
Dyno was well ahead of the AI meets biotech curve, using AI models to design new viral shells that let genetic instructions get into the right places of the body. For the moment, Dyno has focused on creating delivery mechanisms for companies working on therapies aimed at the brain, eye and muscles.
In this episode, we talk with Veres about the promise and perils of gene therapy technology, some of the most recent cases where gene therapies worked and how Dyno’s technology came to be and functions. And we get Veres’s take on whether or not we’re entering a golden age of biotech on the back of AI.
Due to some poor camera work on my part, Veres, who is a tall man, looks particularly giant. Fear not, he did not harm me and was actually quite nice.
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IN THIS EPISODE
Dyno Therapeutics: https://www.dynotx.com/
The FDA framework for individualized ultra-rare disease therapies: https://www.fda.gov/news-events/press-announcements/fda-launches-framework-accelerating-development-individualized-therapies-ultra-rare-diseases
Core Memory on Baby KJ and the new gene therapy playbook: https://www.cor
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