Podcasts

Setting Safety Parameters in Gene Therapy Trials with Thomas Wechsler

Listen to the Molecular Therapy Podcast - January 14, 2025

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.

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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/
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Bright New Morning - Steven O'Brien

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

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