ASGCT Podcast Network

In this episode,  Molecular Therapy  Editor-in-Chief Dr. Joseph Glorioso and Dr. Rita Perlingeiro (University of Minnesota) will discuss an article recently published in  Molecular Therapy  titled, “ Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies .” Music:  'Electric Dreams'...

In this episode of The Issue, Emily Walsh Martin is joined by Dr. Ralph Brandenberger, Master Principal; Head of CMC at Dark Horse Consulting for a discussion on due diligence – the most important time in a company’s existence, be it for funding, partnership, or acquisition. Listen in as Ralph and Emily peel back the curtain on what to expect, how to prepare, and how to stay calm, cool...

Dr. Marcin Kortylewski (Oligonucleotide therapies and applications Section Editor of  Molecular Therapy Nucleic Acids ) sits down with Dr. Norman Michael Drzeniek and Niklas Kotzian from the Berlin Institute of Health at Charité. Join them as they discuss their new article, mRNA-based CAR T cell engineering: Unmodified mRNA enables high CAR expression without innate immune activation i...

In this episode, Melissa Majerol, Model Lead of the Cell and Gene Therapy Access Model at the Center for Medicare and Medicaid Innovation (CMMI) at CMS, breaks down how the CMMI Access Model program aims to simplify and accelerate patient access to innovative cell and gene therapies, starting with recently approved therapies in Sickle Cell disease. Listen in as we dive into the specifics of this n...

Dr. Daniel Stone (Associate Editor-in-Chief, Molecular Therapy Advances ) sits down with Dr. Krishanu Saha, Anna Tommasi, and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their team’s recent publication, Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT, and learn more about their novel method to improve the manu...

Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso joins researchers Kathy Steece-Collier (Michigan State University), Jeffrey Kordower (Arizona State University), and Fredric Manfredsson (Barrow Neurological Institute) to discuss their groundbreaking work on RNA interference for Parkinson’s disease. This episode focuses on their recent article titled "Disease-modifying, multidimensional...

Host Dr. Emily Walsh Martin welcomes Dr. Niki Paulk, CEO and Founder of Siren Biotechnology, for a timely conversation on turning scientific promise into commercial success. Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship, covering essential fundraising strategies from Seed and Series Rounds to Community Rounds and beyond. This episode is a must-listen for b...

Host Emily Walsh Martin welcomes Dr. Jim Burns, CEO of Ensoma, to discuss the company's novel technological approach to treating both rare diseases and solid tumor oncology. Dr. Burns provides a detailed overview of Ensoma's platform and its application in their ongoing clinical trials, including the recently announced first patient infusion for chronic granulomatous disease. Learn how this initia...

Join all four Editors-in-Chief of the Molecular Therapy family of journals for a special 2025 Retrospective. Editors Dr. Glorioso, Dr. Giangrande, Dr. Abou-el-Enein, and Dr. Cripe dive into the most exciting advancements in gene and cell therapy from the past year. Music:  'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.  www.scottbuckley.com.au Show your support for ASGCT!...

Editor-in-Chief of Molecular Therapy , Dr. Joseph Glorioso, is joined by Rachael Nimmo, PhD, former director of the Cell Technology Group at Oxford Biomedica, and Kyriacos Mitrophanous, PhD, Chief Innovation Officer at Oxford Biomedica, to discuss an article recently published in Molecular Therapy  by Nimmo and colleagues titled “ Efficient in vivo generation of CAR T cells using a reta...

Join Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids , as she discusses a recently published MTNA article, Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader , with its authors Drs. Krishanu Saha and Namita Khajanchi (UW-Madison). If you enjoy this episode, check out our hybrid event this fall that will allow you to engage with pioneerin...

Join Dr. Mohamed Abou-el-Enein, Editor-in-Chief of Molecular Therapy Methods & Clinical Development, as he discusses a recent article published in MTMCD with co-authors Drs. Daniel Stone, Harrison Dulin, and Keith R. Jerome from the Fred Hutchinson Cancer Center. The article, “ Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humaniz...

On this episode of Soundbites of the Annual Meeting , we talked to Kimberly Haugstad, CEO of RareRising and a longtime rare disease advocate who is also the parent of a child with severe hemophilia. In her conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Kimberly discusses: why patients, as the "lived experience experts" are so important to rare disease communications adv...

This episode is the fourth in a series hosted by Molecular Therapy  Editor-in-Chief Joseph Glorioso, PhD, 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 review, How to democratize cell and gene therapy: A global approach , with author Rayne Rouce, MD, Texas...

On this episode of Soundbites of the Annual Meeting , we talked to Mimi Lee, MD, PhD, a physician-scientist and program manager at the Advanced Research Projects Agency for Health (ARPA-H). In her conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Dr. Lee discusses: ARPA-H's unique approach to funding and structuring CGT development breaking down silos to improve health out...

On this episode of Soundbites of the Annual Meeting , we talked to Stephen Kaminsky, PhD, professor of research in genetic medicine at Weill Cornell Medical College, and lead author of a phase 1 clinical trial of a new vaccine for cocaine addiction. In his conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Dr. Kaminsky discusses: How the vaccine works and how its approach i...

This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso, PhD, 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, “Current trends in gene therapy to treat inherited disorders of the brain,” with author Bev...

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 impac...

"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 b...

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...

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...

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 h...

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 transiti...

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? Jo...

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 de...