Today's episode takes us into crucial territory where science meets ethics in gene therapy development. Following our recent compelling conversation with Ben Hurlbut on germline genetic editing, we're diving deeper into the ethical dimensions of bringing new treatments to patients.

We'll explore two critical challenges that development teams face: determining the initial dose for therapy and establishing acceptable parameters for off-target effects before clinical testing. While these decisions are grounded in scientific data, they carry profound ethical implications – especially considering that AAV-based therapies are currently "one and done" treatments, where redosing isn't yet possible.

Thomas Wechsler, chair of the ASGCT Ethics Committee, will join us to help us navigate the technical and ethical dimensions of these crucial decisions. His unique perspective will help us understand how teams balance scientific rigor with ethical responsibility when introducing new therapies to the clinic.

Music by: https://www.steven-obrien.net/
--------------------------
Bright New Morning - Steven O'Brien

(Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

Join Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, as she discusses RNA exon editing with Ascidian Therapeutics' leadership: CSO Dr. Robert Bell and CEO Dr. Michael Ehlers. Their conversation explores Dr. Bell's recent MTNA publication "RNA Exon Editing: Splicing the Way to Treat Human Diseases."

Last Call: Submit your abstracts for ASGCT's 2025 Annual Meeting in New Orleans by January 31st, 11:59 PM ET. Present at cell and gene therapy's premier event and get published in Molecular Therapy. Details at annualmeeting.asgct.org/abstracts.

In This Episode

Paloma Giangrande, PhD
Editor-in-Chief, Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapeutics

Robert Bell, PhD
Chief Scientific Officer at Ascidian Therapeutics

Michael Ehlers, MD, PhD
President and Chief Executive Officer at Ascidian Therapeutics

'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.
www.scottbuckley.com.au

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

Sonal Gupta, MD, PhD, SVP and Head of Clinical Development at Affyimmune Therapeutics talks with host Emily Walsh Martin, PhD, to explore the challenges of cell therapy clinical trials and how to move patients quickly and thoughtfully through the steps required to receive therapy in aggressive solid tumor cancers.

But first, "we’ve moved straight from pumpkin spice season into ASGCT Annual Meeting registration season," as Dr. Walsh Martin says. So hop online and join ASGCT, then register and take advantage of the incredible member discounts on Annual Meeting registration. We’ll see you in New Orleans, May 13-17, 2025!

AnnualMeeting.ASGCT.org/register

Music by: https://www.steven-obrien.net/
--------------------------
Bright New Morning - Steven O'Brien

(Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.