ASGCT Podcast Network

"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. Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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.auShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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.auShow your support for ASGCT!: https://asgct.org/membership/donateSee 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/--------------------------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, 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.

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/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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 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. Beverly DavidsonChief Scientific Strategy Officer and Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children's Hospital of Philadelphia Dr. Bryan SimpsonPreclinical Team Lead at Latus Bio '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.

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/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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 GiangrandeEditor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapeutics Dr. Marcin KortylewskiProfessor, Department of Immuno-Oncology at City of Hope '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.

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 StoneAssociate 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 MietzschAssistant Scientist, Department of Biochemistry and Molecular Biology, University of Florida Dr. Robert McKennaProfessor 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.auShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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.auShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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 ChairShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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/donateSee omnystudio.com/listener for privacy information.

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/donateSee omnystudio.com/listener for privacy information.

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/donateSee omnystudio.com/listener for privacy information.

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 CommitteeShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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/donateSee omnystudio.com/listener for privacy information.

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 OlivarezShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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 OlivarezShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

"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 OlivarezShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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!Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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 CommitteeShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Join host Chris Leidli as he sits down with Emily Walsh Martin, PhD, and Lynnea Olivarez to preview the most intriguing and promising abstracts presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting in Los Angeles. Emily and Lynnea share their thoughts on the breathtaking Presidential Symposium before discussing the rest of the program, including clinical implications, the potential impact on rare diseases, and more! Looking for a curated guide through the Annual Meeting? Check out ASGCT's new Session Paths! These six staff-crafted programs provide pathways for some of the most common Annual Meeting attendee profiles: First-time Attendees Cancer Clinical Science Gene Editing Regulatory + Government Affairs Viral Vectors There's still time to register! Join us at the Annual Meeting in Baltimore or from the comfort of your own home with hybrid registration options! Visit https://annualmeeting.asgct.org/register to learn more and register today.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Tune in for an exceptional discussion on chemistry, manufacturing, and controls with Dr. Susan D'Costa, Chief Technical and Commercial Officer at Genezen, a US-based CDMO for viral vectors. "Geek out" with Susan and Emily on the intricacies of defining your CMC strategy and ready yourselves for a tour-de-force of analytical and process development for viral vectors. The future of cell and gene therapy is nearly here! The ASGCT Annual Meeting is less than a month away, and starting on April 22, you can download the abstracts collection to get a sneak peek at the cutting-edge research being presented May 7-11 in Baltimore. Explore the latest breakthroughs and discoveries shaping the field, and don't miss the premier event in cell and gene therapy. Visit www.annualmeeting.asgct.org to learn more and register today! 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 Drs. Timothy Cripe and Marco Davila as they discuss a recent article published in the December issue of Molecular Therapy Oncology by Dr. Davila and colleagues titled, Bispecific CD33/CD123 targeted chimeric antigen receptor T cells for the treatment of acute myeloid leukemia. Dr. Timothy Cripe: Editor-in-Chief of Molecular Therapy Oncology and Professor and Chief of Hematology/Oncology/BMT at Nationwide Children's Hospital Dr. Marco Davila, Rustum Family Endowed Chair in Translational Research, Senior Vice President and Associate Director for Translational Research, Department of Medicine and Immunology, Roswell Park Comprehensive Cancer Center Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals ASGCT members save $385 on registration rates for the ASGCT Annual Meeting whether you're attending in person or virtually! Bundle both ASGCT membership and Annual Meeting attendance and save big on attending the premier event in the field. Register today at annualmeeting.asgct.org. '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.

Join Emily Walsh Martin, PhD for an in-depth interview with Sabrina Mogle, co-founder of RareMoon Consulting. They talk about what drove her to dedicate her career to the orphan drug and advanced therapy space, and how helping clients navigate regulatory conversations on the path from idea to BLA keeps her motivated today. They also discuss her advice for clients in interacting with regulators given the evolving CBER and global regulatory landscape. Do they make it through the whole episode without either providing the standard regulatory wisdom of  “it depends”? Listen to find out! Have you registered for the ASGCT Annual Meeting yet? Meet us in Baltimore, May 7-11, for the only must-attend event in cell and gene therapy. There's no better place to deep dive in this field than the ASGCT Annual Meeting—you will not find the same breadth or depth of CGT research anywhere in the world. Check out the full program and learn more about the meeting at www.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/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Welcome a special ASGCT Annual Meeting preview hosted by Lynnea Olivarez, member of the ASGCT Communications Committee. Today's guest is Emily Mullin, a staff writer at WIRED who covers biotechnology, medicine, and genetics. Emily is a stalwart on the CGT beat, and today, we're looking ahead to the major topics and themes that will be highlighted at the upcoming ASGCT Annual Meeting. The ASGCT Annual Meeting is the field's flagship event, bringing together stakeholders from academia, industry, regulatory agencies, and more. It's a prime opportunity to exchange research, trends, and the latest in the field. Registration is now open and we hope you'll join us in Baltimore, May 7-11. Find Emily Mullin's work at WIRED.com: https://www.wired.com/author/emily-mullin/ Register today for the ASGCT Annual Meeting: https://annualmeeting.asgct.org/ 'Felicity' 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.

Dr. Roland Herzog sits down with Drs. David Schaffer and Adam Schieferecke to discuss a recent article published in the February issue of Molecular Therapy by Drs. Schaffer and Schieferecke and colleagues titled, Evolving membrane-associated accessory protein variants for improved adeno-associated virus production. Read the full article: https://www.cell.com/molecular-therapy-family/molecular-therapy/abstract/S1525-0016(23)00679-2 Dr. Roland Herzog: Editor-in-Chief of Molecular Therapy and Professor of Pediatrics at Indiana University Dr. David Schaffer: Hubbard Howe Jr. Distinguished Professor, Chemical and Biomolecular Engineering, Bioengineering, Molecular and Cell Biology, and the Helen Wills Neuroscience Institute, University of California at Berkeley Dr. Adam Schieferecke: Postdoctoral Scholar, California Institute for Quantitative Biosciences, University of California at Berkeley.  ASGCT members save $385 on registration rates for the Annual Meeting whether you're attending in person or virtually—that's a bigger savings than the cost of membership itself, by the way. Bundle both ASGCT membership and Annual Meeting attendance and save big on attending the premier event in the field. Register today at annualmeeting.asgct.org. Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals '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.

A special ASGCT Annual Meeting preview episode and crossover with the Rx for Biotech podcast featuring guest hosts and ASGCT Communications Committee members Chris Leidli and Catherine Gillespie. They interview ASGCT's Molecular Therapy Scientific Editor Rory Bricker-Anthony, PhD, and preview a scientific symposium at the upcoming ASGCT Annual Meeting on the impact of generative AI on cell and gene therapy (CGT), best practices, and real-world applications for communicators. Dr. Bricker-Anthony, a speaker in the session, shares insight into what attendees can learn from the upcoming session, including current tools and applications in AI for CGT communications. Listen now to get a sneak peek of the upcoming symposium at the ASGCT AnnualMeeting and valuable tips for conference attendees. ASGCT Annual Meeting: annualmeeting.asgct.org RX for Biotech Podcast: soundcloud.com/biotechguru Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Today we have a very special guest on The Issue: Federico Mingozzi, PhD. Federico's first ASGCT podcast could have easily been on Giants of Gene Therapy. Still, we're excited that he could join us for an in-depth chat about his career journey and AAV immunogenicity. Dr. Mingozzi has done seminal work throughout his career in AAV gene therapy, and his 2020 Molecular Therapy review of our current understanding of the immune reaction to these therapies is a must-read for anyone trying to get a handle on the very complicated landscape of immune reactions to viral vectors. Have you registered for the ASGCT Annual Meeting yet? Meet us in Baltimore, May 7-11, for the only must-attend event in cell and gene therapy. There's no better place to deep dive in this field than the ASGCT Annual Meeting—you will not find the same breadth or depth of CGT research anywhere in the world. Check out the full program and learn more about the meeting at www.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/)Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Join Drs. Paloma Giangrande (Editor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapy) and Maxim Berezovski (Professor of Bioanalytical Chemistry at the University of Ottawa). They will discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Berezovski and colleagues titled, Discovery of DNA aptamers targeting SARS-CoV-2 nucleocapsid protein and protein-binding epitopes for label-free COVID-19 diagnostics. Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals '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.

Episode 2 of The Issue on the ASGCT podcast network is an in-depth interview with Dr Tom McCauley, CSO of Omega Therapeutics. Tom and Emily talk about his path from condensed matter physics to leading teams of scientists exploring the next generation of molecular therapeutics. They also dive into their mutual experience in the early days of in vivo gene editing and the challenges one faces with the complexities of these products, particularly before there is much of a roadmap for development. Finally, they dive into the recent public Ad Comm and ultimate approval of Casgevy and how we hope the industry continues to evolve and set standards as we move forward novel approaches. 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. Richard Vile, Professor of Immunology at Mayo Clinic and Dr. Timothy Cripe, Editor-in-Chief of Molecular Therapy Oncology and Professor and Chief of Hematology/Oncology/BMT at Nationwide Children's Hospital. They will discuss a recent article published in the June issue of Molecular Therapy Oncology by Dr. Vile and colleagues titled, "Trap and ambush therapy using sequential primary and tumor escape-selective oncolytic viruses." Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals '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.

Join Dr. Emily Walsh Martin for the first episode of her new podcast, The Issue on the ASGCT podcast network. Emily welcomes Michael Storey, PharmD (Nationwide Children's Hospital), to discuss the path from patient diagnosis to therapy administration for approved gene and cell therapies including the clinical and administrative steps along the way. 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.

Welcome to the first episode of the Molecular Therapy Podcast! Join Dr. Roland Herzog, Editor-in-Chief of Molecular Therapy and Professor of Pediatrics at Indiana University, and Dr. Norbert Pardi, Assistant Professor of Microbiology at the Perelman School of Medicine at the University of Pennsylvania, as they discuss a recent article published in the September issue of Molecular Therapy by Dr. Pardi and colleagues titled, "Development of an mRNA-lipid Nanoparticle Vaccine Against Lyme Disease." Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals '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.

Note: this episode is cross-posted with the Rx for Biotech podcast, hosted by Chris Leidli.  Emily Walsh Martin, PhD, at Tremont Therapeutics Consulting, works with gene and cell therapy companies and investors who are seeking to advance novel therapies in the clinic. ASGCT's Policy Summit is a big deal to her: It provides a full view of the clinical pipeline and helps her guide clients past the drug approval process and into considerations of patient access, manufacturing, and insurance reimbursement. In this special episode, Dr. Walsh Martin shares her highlights from the fifth annual Policy Summit: the chance to see top decision makers, patients, and advocates in the same room; the discussion of the recent CGT Access Model from the Centers for Medicare and Medicaid Innovation (with deputy director Laura McWright); and, of course, the first in-person speaking engagement of Nicole Verdun, MD, director of FDA CBER's new Office of Therapeutic Products (OTP). Hear Dr. Walsh Martin's thoughts on patient-focused development, public access to data, and FDA's support of platform designations for new CGT treatments. Her interviewer is Chris Leidli, host of the Rx for Biotech podcast and member of ASGCT's Communications Committee.  This year's Policy Summit featured its highest-profile line-up yet—and it's still available to view on-demand until October 23! Learn more at asgct.org/advocacy/policy-summit. Not sure if on-demand access is worth it? Let Dr. Walsh Martin convince you why you don't want to miss the event!Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Note: this episode is cross-posted with the Rx for Biotech podcast, hosted by Chris Leidli. Francesca Cook is the Vice President, Pricing and Market Access, at REGENXBIO and past chair of ASGCT's Government Relations Committee. She's attended and helped with ASGCT's annual Policy Summit since its inception five years ago. In this episode, Cook shares her experience and advice around the event—coming up Sept. 18-19 (virtually + in Washington, DC) with its highest-profile line-up yet and more ways to interact with top CGT decision makers. Her interviewer is Chris Leidli, host of the Rx for Biotech podcast and member of ASGCT's Communications Committee. If you work anywhere in the field of cell and gene therapy (CGT)—science, industry, commerce, regulatory—Cook can tell you why you should register for the Policy Summit, a unique opportunity to discuss how your work can be affected by NIH, FDA, patient advocacy groups, private insurers, and more. And if you've already registered, we look forward to seeing you! Cook has some advice for making the most out of the event. Learn more and register at asgct.org/PolicySummitShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Luigi Naldini, MD, PhD, discusses his intercontinental career and the future of gene editing and use of lentiviral vectors during a one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the seventh episode of Giants of Gene Therapy! Dr. Naldini is a Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. He has achieved worldwide renown as the pioneer of lentivirus gene therapy, a tool that offers hope for cures to deadly diseases. Dr. Naldini has been a mentor to many young scientists and a prolific researcher with more than 280 publications. He has founded three biotech startups. He's spoken in more than 150 congresses worldwide. And he's received numerous global awards, including the Grand Officer of the Order for Merit of the Italian Republic (one of the highest-ranking honors in Italy), the Louis-Jeantet Prize for Medicine in Switzerland, and Outstanding Achievement Awards from both the American Society of Gene and Cell Therapy and European Society of Gene and Cell Therapy.  Dr. Naldini has been an ASGCT member since 2000, the year he also attended the 3rd ASGCT Annual Meeting. He's served on the Society's board and committees. Music by: Steven O'Brienhttps://www.steven-obrien.net/ "Making Progress" (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.

Terry Flotte, MD, discusses the early days and possible future of gene therapy, the use of AAV to deliver GT to targeted sites in the body, and his inspiration behind becoming a physician, scientist, and medical school dean. He spoke one-on-one with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the sixth episode of Giants of Gene Therapy! Dr. Flotte is the Provost, Dean, and Celia and Isaac Haidak Professor at UMass Chan Medical School, with faculty appointments in the Department of Pediatrics and the Horae Gene Therapy Center. He is also a practicing pediatric pulmonologist and a prolific researcher who has authored more than 280 papers and been the principal investigator of multiple clinical gene therapy trials. Dr. Flotte is the editor-in-chief of his field's oldest journal family, Human Gene Therapy. In 1995, Dr. Flotte was the principal investigator for the first human use of AAV vectors, in a trial in cystic fibrosis patients. He's currently investigating the use of gene therapy for genetic diseases that affect children, including alpha-1 antitrypsin (AAT) deficiency and lysosomal storage diseases. Dr. Flotte has been an ASGCT member since the Society's founding. He's served on the board and committees, and spoken at the ASGCT Annual Meeting and other events.   Music by: Steven O'Brienhttps://www.steven-obrien.net/ "Making Progress" (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.

Jennifer Doudna, PhD, shares her personal journey to co-inventing CRISPR-Cas9 for gene editing and the promise of her discovery, during a one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the fifth episode of Giants of Gene Therapy! Dr. Doudna has been at UC Berkeley since 2002. She is a professor in the departments of Molecular and Cell Biology and Chemistry, the Li Ka Shing Chancellor's Professor of Biomedical Science. She's also the president and co-founder of the Innovative Genomics Institute. In 2020, Dr. Doudna earned the Nobel Prize in Chemistry for co-inventing CRISPR-Cas9 genome editing technology with Emmanuelle Charpentier, PhD. Outside of her continued work on CRISPR technologies in the lab, Dr. Doudna is a leader in public discussion of the ethical implications of genome editing for human biology and societies. Dr. Doudna has spoken at the ASGCT Annual Meeting and will be a keynote speaker at this year's Annual Meeting in May, talking about “CRISPR Chemistry and Applications in the Clinic.” Music by: Steven O'Brienhttps://www.steven-obrien.net/ "Making Progress" (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.