Design Therapeutics, Inc. (DSGN) Presents at Cantor World Healthcare Convention 2025 Tran

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Design Therapeutics, Inc. (NASDAQ:DSGN) Cantor Global Healthcare Conference 2025 September 4, 2025 10:55 AM EDT

Company Participants

Pratik Shah – Co-Founder, President, CEO, Principal Financial Officer & Executive Chairperson

Conference Call Participants

Joshua schimmer – Cantor Fitzgerald & Co., Research Division

Presentation

Joshua Schimmer
Biotech Equity Research Analyst

All right. We’re ready to get started. Welcome, everyone. I’m Josh Schimmer from the Cantor Biotech Equity Research team. I’m pleased to introduce from Design Therapeutics, we have Pratik Shah, Chief Executive Officer.

Question-and-Answer Session

Joshua Schimmer
Cantor Fitzgerald & Co., Research Division

We’re going back to some GeneTAC. So Pratik, thanks so much for joining. Why don’t you frame Design Therapeutics and give us a quick overview of how the GeneTAC platform works?

Pratik shah
Co-Founder, President, CEO, Principal Financial Officer & Executive Chairperson

What makes design unique and interesting is that we’ve developed a new class of small molecules that do something remarkable, which is that they dial up or dial down the expression of a single gene in the genome. And the way the molecules do that is they recognize DNA sequences through the minor group interactions, much like a transcription factor would. And they then recruit the appropriate transcriptional machinery to the locus to either dial up or down expression.

And so as you think about the role of individual genes in disease, there are many monogenic disorders where the single gene root cause is known, and we’re working on several such monogenic conditions. One is Friedreich’s ataxia. The other is Fuchs endothelial corneal dystrophy. The third is myotonic dystrophy and then Huntington’s disease as our front-runner examples of monogenic diseases.

In Friedreich’s ataxia, the problem is that the level of expression from the frataxin gene is low. and that’s what causes the disease. And so what we’ve done with

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