We love to hear from our listeners. Send us a message. In Episode 3 of our in vivo-focused special series of Cell &amp; Gene: The Podcast, Ascidian Therapeutics&#x27; Founder, President, and CEO Mike Ehlers, M.D., Ph.D., explains how the company is advancing a new class of genetic medicines built on RNA exon editing to durably correct disease at the transcript level without permanently altering DNA. He also shares why in vivo therapies still need to prove precision, durability, and predictability before they become mainstream, and why the field must move beyond proof of concept to reproducible clinical performance across patients and diseases. He discusses the importance of patient selection, tissue specificity, and matching editing efficiency to biological thresholds, and much more. Subscribe to the podcast! Apple | Spotify | YouTube Visit my website: Cell &amp; Gene Connect with me on LinkedIn

Why In Vivo Therapies Are Moving Toward the Clinic with Ascidian Therapeutics' Dr. Mike Ehlers

Cell & Gene: The Podcast

# Full Transcript

**Podcast:** Cell &amp; Gene: The Podcast

## Participants

- **Erin Harris** (None)
- **Dr. Mike Ehlers** (Ascidian Therapeutics)

## Transcript

Hi everyone, I'm Erin Harris, host of Cell & Gene: The Podcast. Welcome to Episode 3 in our special In Vivo-focused series. If you didn't catch Episodes 1 and 2 yet, definitely do yourself a favor and go back and give them a listen. Episode 1 features my conversation with Precision Biosciences Cassie Gorsuch, and Episode 2, I sit down with Beam Therapeutics Gopi Shanker. They're both definitely worth your time, well worth a listen, so go back and listen to those when you get a chance.

This is Episode 3 and I'm talking to Ascidian Therapeutics founder, president, and CEO Dr. Mike Ehlers. We talk about how the company is advancing a new class of genetic medicines built on RNA exon editing to durably correct disease at the transcript level without permanently altering DNA. Mike also shares why In Vivo therapies still need to prove precision, durability, and predictability before they become mainstream.

We also talk about why the field must move beyond proof of concept to reproducible clinical performance across patients and diseases. And we also discuss the importance of patient selection, tissue specificity, and matching editing efficiency to biological thresholds. And we also cover a lot more in our conversation. This is a terrific episode and I'm certain you will find it interesting and well worth your time. So without further ado, here's my conversation with Ascidian Therapeutics,

Dr. Mike Ehlers, Dr. Mike Ehlers. Mike, thanks for joining me on Cell and Gene, the podcast in this really special series that we're doing for In Vivo therapies. And I'm really happy that Ascidian has taken me up on my request to join us on this special series. So welcome. And then tell us a little bit about Ascidian Therapeutics and its mission.

Well, thanks, Erin, for having me here on Cell and Gene. I really appreciate it. So Ascidian is a clinical stage biotech company where we're building a new class of genetic medicines that's based on RNA exon editing, which is otherwise known as transplicing.

Essentially, rewriting faulty messages rather than rewriting the genome itself. And so the goal with the company, our mission has been relatively simple, but ambitious, which is to durably correct disease at its source while minimizing permanent risk to the patient's DNA.

And our view is that if we can fix the transcript safely and repeatedly at the kilobase scale, we can treat a broad range of diseases that today are genetically understood, but therapeutically largely out of reach. Terrific. Okay. We're going to get to specifically Ascidian's RNA exon editing approach a little bit later in our discussion.

Before we do, I want to talk a little bit more broadly about in vivo therapies and get your perspective on it. So we know they're often sometimes referred to or discussed as the future of Cell and Gen...

Why In Vivo Therapies Are Moving Toward the Clinic with Ascidian Therapeutics' Dr. Mike Ehlers

Transcript

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Why In Vivo Therapies Are Moving Toward the Clinic with Ascidian Therapeutics' Dr. Mike Ehlers

Transcript

Built for investment professionals

Every day you wait, your competitors are findingedge in conversations you're not hearing.

Every day you wait, your competitors are finding
edge in conversations you're not hearing.