During this episode of Soundbites of the Annual Meeting , we spoke with P.J. Brooks, PhD, who is Deputy Director of the Division of Rare Diseases Research Innovation, part of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). In his conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Dr. Brooks talked about: The impact of developing cell and gene therapies as platforms to treat more than one disease Projects he's in...
Jul 09, 2025•9 min
"Certainly, I've been anxious about data...I did an experiment, I put a lot of time into it, I'm hoping to get good results...we all know that feeling, right? The stakes are just so different here...this kid's life depends on whether this experiment worked." --Kiran Musunuru, MD, PhD One of the highlights of the ASGCT 28th Annual Meeting was the announcement on May 15 of a historic medical breakthrough : a child diagnosed with a rare genetic disorder had been successfully treated for the first t...
Jun 16, 2025•28 min
In honor of Molecular Therapy’s 25th anniversary, this episode is the second 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. In this episode, Dr. Glorioso will discuss the reviews, AAV vector development, back to the future , with author Sheila Mikhail; M34, Inc.; and The deLIVERed promises of gene therapy: Past, present, and future of live...
Jun 11, 2025•48 min
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 Med...
May 01, 2025•48 min
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, n...
Apr 14, 2025•45 min
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 ...
Mar 11, 2025•49 min
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 ...
Mar 04, 2025•37 min
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 trial...
Feb 11, 2025•50 min
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 ...
Jan 14, 2025•40 min
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 publis...
Jan 07, 2025•38 min
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 tak...
Dec 10, 2024•27 min
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 pro...
Dec 03, 2024•29 min
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 ...
Nov 12, 2024•36 min
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....
Nov 05, 2024•52 min
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 p...
Oct 15, 2024•44 min
Dr. Ben Hurlbut, professor at Arizona State University's Lincoln Center for Applied Ethics, joins host Dr. Emily Walsh Martin for a critical conversation in the cell and gene therapy industry in which they discuss how ethics informs and impacts the field and offers a deep dive into the ethical, medical, and social considerations around germline gene editing. Music by: https://www.steven-obrien.net/ -------------------------- Bright New Morning - Steven O'Brien (Used for free under a Creative Com...
Sep 17, 2024•1 hr 5 min
Drs. Beverly Davidson and Bryan Simpson join Molecular Therapy EiC Dr. Roland Herzog to discuss their recent paper, Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in the brain, which was published in Molecular Therapy. ASGCT is proud to present this episode of the Molecular Therapy Podcast in partnership with our upcoming Advancing Gene and Cell Therapy for Cancer conference, which will be held on October 16 and 17 in Philadelphia and online. This landmark hyb...
Sep 10, 2024•36 min
Join host Emily Walsh Martin, PhD, and David Altshuler, MD, PhD, Chief Scientific Officer of Vertex Pharmaceuticals. David came to Vertex after a lengthy career in medicine and academic research. David and Emily discuss how David and the team at Vertex think about innovation in drug development and how that impacts how they select programs and approaches for the diseases they focus on. They also cover what he’d love to see happen with new cell and gene platform technologies being brought forward...
Jul 16, 2024•49 min
Join ASGCT and the Molecular Therapy family of journals for a conversation between Drs. Paloma Giangrande and Marcin Kortylewski. They discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Kortylewski and colleagues titled " Oligo-PROTAC strategy for cell-selective and targeted degradation of activated STAT3 ." If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, September 23-24. This can't-miss event brings together poli...
Jul 09, 2024•34 min
Dr. Daniel Stone speaks with Drs. Mario Mietzsch and Robert McKenna from the University of Florida to discuss a recent article published in the Biomanufacturing in Gene and Cell Therapy special issue of Molecular Therapy Methods & Clinical Development by Drs. Mietzsch, McKenna, and colleagues titled Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard . If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington,...
Jun 18, 2024•35 min
In this Very Special Episode, Emily Walsh Martin, PhD explores the cutting-edge transposase-based technology presented by SalioGen at the ASGCT Annual Meeting in which they achieved gene insertion without double-strand breaks. Join us as Jason Cole, CEO at SalioGen, takes us on a captivating journey through the company's groundbreaking work, including their initial data in Stargardt disease using LNP delivery to photoreceptors. But that's not all! Discover Jason's unconventional path from small ...
Jun 11, 2024•48 min
Mohamed Abou-el-Enein , MD, PhD, MSPH, the new editor-in-chief of Molecular Therapy Methods & Clinical Development ( MTMCD ), sits down with Scientific Editor Rory Bricker-Anthony, PhD, to discuss his vision for the journal. If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, on September 23 and 24 . This can't-miss event brings together policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Nicole Verdun, ...
Jun 04, 2024•21 min
At #ASGCT2024, Dr. Lam (CIRM) spoke during a symposium on developing CGTs with a DEI-forward approach. In this interview, she talks about incorporating DEI into the review criteria for CIRM's funded programs, how to measure DEI, and what she'd like to see in the long term for patients. Meeting registrants can watch this session (and all others) through June 21 on the virtual platform and the app! Host: Lynnea Olivarez, incoming Communications Commmittee Chair Show your support for ASGCT!: https:...
May 14, 2024•6 min
As she begins her tenure as ASGCT President, Paula Cannon, PhD (USC), tells host Lynnea Olivarez, incoming chair of the Communications Committee, why she's excited to take the reins for the Society. Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information.
May 11, 2024•5 min
Jef Akst, managing editor at BioSpace, talks about the major CGT issues she's been covering at #ASGCT2024 including regulatory considerations and AAV manufacturing. She also tells host Lynnea Olivarez what she hopes to be reporting on next year at the ASGCT Annual Meeting in New Orleans! Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information....
May 10, 2024•6 min
Todd Carter, PhD (CSO at Voyager Therapeutics) talks with incoming Communications Committee Chair Lynnea Olivarez about changes in the approach to developing gene therapies for CNS disorders like Huntington's disease. Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information.
May 10, 2024•6 min
ASGCT Board of Directors member Lindsey George, MD, (Children's Hospital of Philadelphia) discusses the dramatic advancements in available treatment options for patients with hemophilia and the responsibility of scientists and clinicians in guiding patients through these once-in-a-lifetime decisions. Host: Lynnea Olivarez, incoming chair of the ASGCT Communications Committee Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information....
May 10, 2024•6 min
Incoming ASGCT Communications Committee chair Lynnea Olivarez catches up with ASGCT Career Development Award winner Ashley Cooney, PhD, (University of Iowa) to discuss her work in vector-delivered airway gene transfer and use of saline solution to "dramatically increase" gene transfer. Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information....
May 10, 2024•4 min
Leah Myers, Executive Director of the FamilieSCN2A Foundation, and Jenny Burke, who sits on the organization's board, talk about the importance of empowering patients and families affected by genetic diseases. Host: Lynnea Olivarez Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information.
May 08, 2024•6 min
Dr. Murry talks about his early work reprogramming injured heart cells, his vision for the future of organ regeneration, and the privilege of getting paid to be curious. Host: Lynnea Olivarez Show your support for ASGCT!: https://asgct.org/membership/donate See omnystudio.com/listener for privacy information.
May 08, 2024•6 min
