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/donateSee 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, PhDEditor-in-chief of Molecular Therapy Nucleic AcidsSenior Vice President Discovery & Translational Biology, Orbital Therapeutics Ayşegül Yildiz, PhDScientist of TRON’s Cardiovascular Therapeutics Group Tim Beissert, PhDSenior Scientist and Group Leader of TRON’s Vector Development and Gene Transfer TeamShow your support for ASGCT!: https://asgct.org/membership/donateSee 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/donateSee 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/donateSee 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, PhDEditor-in-Chief, Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapeutics Robert Bell, PhDChief Scientific Officer at Ascidian Therapeutics Michael Ehlers, MD, PhDPresident and Chief Executive Officer at Ascidian Therapeutics 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.auShow your support for ASGCT!: https://asgct.org/membership/donateSee 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/donateSee omnystudio.com/listener for privacy information.

This episode features a conversation between Dr. Timothy Cripe and Dr. Joseph Glorioso, who discuss an article published in Molecular Therapy Oncology by Dr. Glorioso and colleagues titled Oncolytic Herpes Simplex Viruses Designed for Targeted Treatment of EGFR-bearing Tumors. Join the editor-in-chief of Molecular Therapy, Dr. Roland Herzog, and ASGCT this January for the next installment of Molecular Therapy Presents: Clinical Gene and Cell Therapy. This transformative field has grown from promising experimental treatments to approved medicines for a wide range of genetic and/or acquired diseases. This virtual event is free for ASGCT members to attend and will highlight several in-depth invited reviews appearing in Molecular Therapy's Clinical Gene and Cell Therapy special issue. Attend the webinar and learn more about cutting-edge developments in the clinical space before the special issue is published in early 2025. Find Molecular Therapy Presents: Clinical Gene and Cell Therapy, and all upcoming ASGCT events at ASGCT.org/events.  In This Episode Timothy Cripe, MD, PhDEditor-in-Chief, Molecular Therapy Oncology and Professor and Chief of Hematology, Oncology, BMT at Nationwide Children's Hospital Dr. Joseph GloriosoProfessor, Department of Microbiology and Molecular Genetics and Department of Human Genetics at the University of Pittsburgh 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.auShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Host Emily Walsh Martin, PhD, is joined by Chris Choi, PhD, Roswell Park Comprehensive Cancer Center's Senior Vice President of Industry Partnerships and Technical Director of the center’s new Good Manufacturing Practice (cGMP) Facility. Dr. Choi is also an associate professor of oncology. This wide-ranging conversation covers cell therapy development and manufacturing and how the growing Roswell Park Center is aiming to support sponsors in the CAR T and cell therapy space. Emily and Chris also discuss their experiences on cross-functional teams and how a program's research, manufacturing, and clinical elements must be knit together for success. 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/donateSee omnystudio.com/listener for privacy information.

This episode features a conversation between Dr. Roland Herzog and Dr. Joseph Glorioso. On January 1, 2025, Dr. Glorioso will begin his 5-year term as the Editor-in-Chief of Molecular Therapy, marking the end of Dr. Herzog’s successful tenure at the helm of the journal. Drs.Herzog and Glorioso engage in a wide-ranging conversation that touches on the history of Molecular Therapy, highlights from the gene and cell therapy field, and how Molecular Therapy will continue to grow and evolve under Dr. Glorioso’s leadership. ASGCT is proud to present this episode of the Molecular Therapy Podcast in partnership with our upcoming Breakthroughs in Muscular Dystrophy conference on November 19th and 20th in Chicago, and virtually This event will provide an unparalleled opportunity to delve into the latest advancements in research on gene and cell therapies for muscular dystrophy. Learn more and register today at ASGCT.org/Breakthroughs. In This Episode: Dr. Roland HerzogEditor-in-Chief of Molecular Therapy and Professor of Pediatrics, Riley Children's Foundation Professor of Immunology, and Director of the Gene and Cell Therapy Program at Indiana University Dr. Joseph GloriosoProfessor, Department of Microbiology and Molecular Genetics and Department of Human Genetics at the University of Pittsburgh 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.auShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Derek Jackson, vice president of cell and gene therapy product development at Pacira BioSciences, joins The Issue to take the program in a different direction. We often focus our discussions on CGT for rare diseases. Most of the time, the teams of scientists working on the program don’t have any personal experience with the disease from their family, friends, or themselves.. but today we will talk about gene therapy development for a much more common disease: knee osteoarthritis. Moreover, the product we will be discussing is not only non-rare but also non-AAV.  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/donateSee omnystudio.com/listener for privacy information.