ASGCT Podcast Network

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, and collected under scrutiny! Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/

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 in T cells. Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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 new access model and its current status. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/

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 manufacturing of multifunctional, genome-edited immune cell therapies. Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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 efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques." Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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 both industry professionals and academic innovators. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/

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 initial trial is set to inform future therapeutic opportunities for indications with high unmet medical need. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/

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

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 retargeted fourth-generation lentiviral vector.” Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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 pioneering researchers in gene editing! Breakthroughs in Targeted In Vivo Gene Editing will be held in San Diego and virtually November 20-21, 2025.  Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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-humanized mice,” will appear in MTMCD’s upcoming special issue, Lipid nanoparticles for cell and gene therapy.    If you enjoy this episode, check out our hybrid event this fall that will allow you to engage with pioneering researchers in gene editing! Breakthroughs in Targeted In Vivo Gene Editing will be held in San Diego and virtually November 20-21. Register to secure your spot or submit an abstract through Sept. 12 for the opportunity to present your research!  Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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 advice for scientists who would like to engage more directly with patient communities what's giving her hope right now Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

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 Children’s Hospital.  If you want to learn more about cell and gene therapy access, register for the ASGCT Policy Summit, September 25-26, in Washington, D.C.! We’ll hear from policymakers, industry leaders, and science stakeholders, who will explore solutions to the challenges of accessing therapeutic treatments.  Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au 

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 outcomes for all the role that CGT will play in prevention, rather than just treatment, of diseases Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

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 is different from other treatments Safety results from the unblinded data Implications this vaccine could have for other substance use disorders Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

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 Beverly Davidson, PhD, The Children’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine.  If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there! Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au 

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 involved in that are working towards the platform approach What's giving him hope right now about the future of the field Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes! Did you attend the 28th Annual Meeting? Watch all sessions on demand on the virtual meeting platform through July 18! 

"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 time with a customized CRISPR gene editing therapy. The infant, KJ Muldoon, was born with a rare metabolic disease called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. When he was just six months old, KJ received his personalized therapy at the Children's Hospital of Philadelphia (CHOP), where ASGCT Members Kiran Musunuru, MD, PhD; and Rebecca Ahrens-Nicklas, MD, PhD; led the group of researchers who developed KJ's treatment. KJ is now growing and thriving. He recently left the hospital where he has spent the majority of his life. And the day after presenting the research that led to this first-of-its-kind development, Dr. Musunuru told us that there was so much more to the story. Listen to our first episode of Soundbites of the Annual Meeting with Dr. Kiran Musunuru.  Did you attend the 28th Annual Meeting? Watch all sessions, including Dr. Musunuru's presentation, on demand on the virtual meeting platform through July 18!  Not a member? Join now and receive 19 months of membership for the price of 12.    

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 liver-directed gene therapy; with author Dr. Mark Kay, Stanford University.  If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there!  Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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! 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.www.scottbuckley.com.au

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/ -------------------------- Bright New Morning - Steven O'Brien (Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

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/)

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

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/)

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/)

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

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/)

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, PhD Editor-in-Chief, Molecular Therapy Oncology and Professor and Chief of Hematology, Oncology, BMT at Nationwide Children's Hospital Dr. Joseph Glorioso Professor, 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.au

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/)

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 Herzog Editor-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 Glorioso Professor, 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.au

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/)

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 Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

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 hybrid event brings together innovative treatment approaches and an unparalleled opportunity to immerse yourself in the latest gene and cell therapy research for cancer. Learn more and register today at ASGCT.org/Advancing. In This Episode: Dr. Roland Herzog Editor-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. Beverly Davidson Chief Scientific Strategy Officer and Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia Dr. Bryan Simpson Preclinical Team Lead at Latus Bio 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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. Now is the time to register for the ASGCT Policy Summit, held in Washington DC on September 23-24. Whether you attend in person or virtually, you will not want to miss this year’s program. Great sessions are planned on reimbursement, Medicaid, venture capital, strategic investment outlooks, and the evolution of the regulatory landscape. Advanced registration ends August 23, so register today! ASGCT.org/PolicySummit 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/)

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 policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Dr. Nicole Verdun, to discuss the latest policies impacting this rapidly evolving field.  Register now at https://www.asgct.org/PolicySummit for invaluable insights on navigating the regulatory landscape.  In This Episode: Dr. Paloma Giangrande Editor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapeutics Dr. Marcin Kortylewski Professor, Department of Immuno-Oncology at City of Hope 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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, DC, September 23-24.  This can't-miss event brings together policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Dr. Nicole Verdun, to discuss the latest policies impacting this rapidly evolving field.  Register now at https://www.asgct.org/PolicySummit for invaluable insights on navigating the regulatory landscape.  In This Episode: Dr. Daniel Stone Associate Editor-in-Chief of Molecular Therapy Methods & Clinical Development and Senior Staff Scientist, Infectious Disease Sciences, Vaccine and Infectious Disease Division at Fred Hutch Cancer Center Dr. Mario Mietzsch Assistant Scientist, Department of Biochemistry and Molecular Biology, University of Florida Dr. Robert McKenna Professor and Director of the Center for Structural Biology, Department of Biochemistry and Molecular Biology, University of Florida 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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 molecules to genetic medicines and gain insights into the strategic value of using a target product profile to guide development decisions. Finally, we delve into the transformative potential of genetic medicine, bridging the gap from bench to bedside to payer-to-patient access. And before we dive in, a quick reminder: The ASGCT Policy Summit (Sept 23-24 in DC or online) is an absolute must for R&D scientists, analytical experts, clinical researchers, and program managers seeking a comprehensive understanding of genetic medicine development. Don't miss this crucial conversation between regulators, payors, patients, caregivers, and developers. ASGCT.org/PolicySummit 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/)

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, PhD, to discuss the latest policies impacting this rapidly evolving field. Register now at ASGCT.org/PolicySummit for invaluable insights on navigating the regulatory landscape. Virtual attendance options are also available. Dr. Abou-el-Enein has served as MTMCD's associate editor-in-chief for the past two years, which will allow him to make a smooth transition in leadership. His vision for MTMCD is consistent innovation, evolution, and growth. Dr. Abou-el-Enein firmly believes that MTMCD has the potential to become a leading voice in the gene and cell therapy landscape. He is eager to leverage his experience and expertise to expand the journal’s reach and impact.  Dr. Abou-el-Enein is an associate professor of clinical medicine (oncology), pediatrics, and stem cell biology and regenerative medicine at USC Keck School of Medicine as well as executive director of the USC/CHLA cell therapy program. He is an internationally recognized expert in planning and executing clinical development programs, designing and operating academic GMP facilities, and translating cell and gene therapy products from preclinical research to clinical applications. His research laboratory is dedicated to pioneering advancements in CAR T-cell therapies, including optimizing engineering techniques and devising novel strategies for allogeneic applications and genetic delivery tools to increase accessibility and scalability. 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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

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.

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!

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.

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

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.

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

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

"Storytelling is completely underrated in science," says Benjamin McLeod, a lab scientist-turned-communicator. Listen to him talk about the importance of presenting CGT research in a narrative format. Host: Lynnea Olivarez

In this episode, host Lynnea Olivarez talks with David Segal, PhD, an abstract session co-chair at the ASGCT Annual Meeting. Dr. Segal discusses new technologies that he says have moved from academia to industry, and, hopefully soon, to rare disease patients. Miss a session? Catch it on demand on the virtual platform for 30 days following the conclusion of the meeting!

ASGCT President Dr. Jeff Chamberlain cuts the ribbon at the ASGCT 27th Annual Meeting and, having planned much of this year's event, reflects on nearly three decades of ASGCT. Host: Lynnea Olivarez, ASGCT Communications Committee

Lynnea Olivarez, incoming ASGCT Communications Committee chair, will be live at the ASGCT 27th Annual Meeting talking to Society leaders, speakers, and abstract presenters about their contributions and what they're excited about at the Annual Meeting. These bite-sized pods are perfect for your walk between the hotel and convention center or your commute to work during the Annual Meeting.