In honor of Molecular Therapy’s 25th anniversary, this episode is the first in a series hosted by Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years by expert authors. In this episode, Dr. Glorioso will discuss two of these reviews, “Gene therapy then and now: A look back at changes in the field over the past 25 years,” with author Dr. Terry Flotte, UMass Chan Medical School, and “From concept to cure: The evolution of CAR-T cell therapy,” with author Dr. Carl June, University of Pennsylvania.

Want to learn about more recent developments in the field directly from the people who are doing the research? There's still time to register and join us for ASGCT’s 2025 Annual Meeting, May 13 to 17, in New Orleans or virtually! If you become a member of the Society, you can save $465 on your registration for the premier event in the field. Check out the full program and register for the meeting today!

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

See omnystudio.com/listener for privacy information.

On this episode of The Issue, Host Emily Walsh Martin, PhD, is tackling her absolute favorite subject: the importance of long-term data!

Emily is joined by Dolan Sondhi, PhD, of Weill Cornell Medical College, to discuss her recent paper, “Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease.”

We hope to see all of your long-term data at the ASGCT Annual Meeting, the premier event in cell and gene therapy, next month! Join us in New Orleans May 13-17, where we'll learn from more than 2,100 abstracts submitted for presentation. For information about the program, registration, and more, visit: https://annualmeeting.asgct.org/.

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/)

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

See omnystudio.com/listener for privacy information.

Today, Niki Gallo, principal consultant of regulatory affairs at Halloran Consulting, joins host Emily Walsh Martin for a deep dive into timeline management pitfalls, the nuances of INDs, and the rapidly evolving FDA landscape. Niki brings to the program her extensive experience shepherding innovative therapeutics through the regulatory process and timely insights into the complexities of transitioning from the bench to the bedside.

Whether you’re from industry or academia, today’s conversation will help wrap all our heads around testing novel therapies for the first time in humans.

The ASGCT Annual Meeting is almost here! Join us in New Orleans May 13-17 for the premier event in CGT and save $465 by joining ASGCT and using the incredible discounts the Society provides to members. Visit annualmeeting.asgct.org to check out the full program, plan your trip, and register for the ASGCT Annual Meeting.

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.

Drs. Ayşegül Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids by Drs. Yildiz. Beissert, and colleagues titled “Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression”.

Are you registered for ASGCT’s 2025 Annual Meeting in New Orleans? Join us May 13-17 for the premier event in our field and save $465 as an ASGCT member. Check out the full program, plan your trip, and register for the ASGCT Annual Meeting by visiting annualmeeting.asgct.org.

In This Episode

Paloma Giangrande, PhD
Editor-in-chief of Molecular Therapy Nucleic Acids
Senior Vice President Discovery & Translational Biology, Orbital Therapeutics

Ayşegül Yildiz, PhD
Scientist of TRON’s Cardiovascular Therapeutics Group

Tim Beissert, PhD
Senior Scientist and Group Leader of TRON’s Vector Development and Gene Transfer Team

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

See omnystudio.com/listener for privacy information.

Join host Emily Walsh Martin and guest Brendan Hayes, senior director of education and innovative therapies at the National Bleeding Disorders Foundation, formerly known as the National Hemophilia Foundation. for an illuminating conversation about trust, innovation, and the power of patient advocacy in advancing cell and gene therapies.

Together, they dive into how patient communities shape the development of new therapies, the historical context that influences decision-making in clinical trials, and the unique considerations facing those who might choose to pursue cell and gene therapies.

You can also hear Brendan in a panel discussion titled The Journey from Early-stage Trials to Approval at ASGCT’s Empowering Patients 2025: A Cell and Gene Therapies Summit. Hosted live on March 12 and 13, you can register for this free, patient-centered virtual event by visiting ASGCT.org/EP2025.

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.

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.