ASGCT Podcast Network

This episode, hosted by Daniel Stone, Associate Editor-in-Chief of Molecular Therapy Advances, focuses on the article, Normothermic perfusion of human livers for profiling lentiviral vector pharmacokinetics and transduction. Listen as Robert Carlisle and Brannon Nicholls from the University of Oxford discuss their approach to studying lentiviral vector transduction and pharmacokinetics in the human liver ex vivo. Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

In this episode of Scientists, Unedited, host Eirini Vamva, PhD, talks with Bruce Levine, PhD, about his personal life and career in cell therapy. Dr. Levine talks about finding his way into immunology, learning from pivotal mentors, and following the unexpected opportunities that led him to the forefront of cell therapy. He reflects on the patients who inspired his work and the teamwork behind major breakthroughs. He also shares the story behind a CAR-T song he helped write and talks about some of the things that make him who he is outside the lab, including cycling and his love of wildlife and dancing.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Molecular Therapy Oncology has released a new special collection, Advancements in Pediatric Cancer Therapy. Listen in as guest editor Nirali Shah and Editor-in-Chief Timothy Cripe discuss articles in this special collection, which covers the history, current landscape, and future direction of pediatric cancer therapy. 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 this episode of The Issue, Sara Johnson Davis, Senior Director of Government Affairs at Ardelyx and former Vice President, Head of Patient Advocacy & Policy at Fulcrum Therapeutics, joins host Sanjay D’Souza, PhD, to discuss the essential role of patient advocacy in cell and gene therapy. The conversation covers the evolving role of patient advocacy in drug development, from early research and clinical trial design to policy, commercialization, and access. Sara shares lessons from the sickle cell disease community, reflects on the transition of gene editing from experimental science to approved therapy, and explains why trust, infrastructure, and policy are essential to ensuring transformative treatments actually reach patients. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded live at the ASGCT 2026 Annual Meeting in Boston, Le Cong, PhD joins hosts Jason West, PhD and Sanjay D’Souza, PhD for a discussion on his work in genome engineering and the early development of CRISPR/Cas9. The three then dive into Dr. Cong’s use of artificial intelligence tools developed and currently used by his lab. Dr. Cong is a 2026 recipient of ASGCT’s Outstanding New Investigator Awards. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

This episode focuses on Molecular Therapy’s upcoming special issue, the biological clock of aging and the possibility of lifespan extension. Listen in as Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, discusses the molecular mechanisms that drive senescence, determine longevity, and contribute to healthy aging with the guest editors of the special issue, Dr. Christin Glorioso from NeuroAge Therapeutics and Dr. John Bell from the University of Ottawa. 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 this episode recorded live at the ASGCT 2026 Annual Meeting in Boston, host Eirini Vamva, PhD interviews Anna Maurer, PhD of the University of Michigan. Dr. Maurer shares her story of growing up in Michigan and how her path led her to Harvard Medical School. She discusses her work in AAV and gives advice to individuals looking to enter the field of cell and gene therapy. Music: 'Dreamer' by Steven O'Brien – Released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, host Sanjay D’Souza, PhD, talks with Fyodor Urnov, PhD, about the evolution and future of gene editing. One of the 2026 ASGCT Catalyst Award co-recipients, Dr. Urnov, talks about his work in gene editing and discusses the need for collaboration in the field to bring more cell and gene therapies to patients. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

This episode, recorded at the ASGCT 2026 Annual Meeting in Boston, focuses on Molecular Therapy Advances’ upcoming special issue, Beyond prevalance: How rare diseases are reshaping therapeutic innovation. Listen as Editor-in-Chief of Molecular Therapy Advances, Dr. Mohamed Abou-el-Enein, discusses the evolution of therapeutic development for rare diseases with guest editors Drs. Jennifer Adair, Claire Booth, Terence Flotte, and Donald Kohn. 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.

Scientists, Unedited is a monthly podcast hosted by Eirini Vamva, PhD, that spotlights the people behind scientific breakthroughs – their journeys, motivations, and lives beyond the lab. Each episode begins with personal stories, explores passions outside of science, and closes with an accessible conversation about the guest’s work for a general audience. In this first episode, Dr. Vamva sits down with ASGCT President Matthew Porteus, PhD, for a conversation about the winding path that brought him full circle back to Stanford University. The conversation offers a candid look at the personal experiences and motivations that have shaped Dr. Porteus’s career and his leadership in the field of cell and gene therapy.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community. In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Godwin Iroanya of the University of Missouri. Godwin shares his current work and research and how ASGCT membership has helped his career, including the Molecular Therapy Family of Journals and the Society’s pilot Mentor Program. Music: 'Origami' 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 episode of The Issue, new co-hosts Jason West, PhD, and Sanjay D’Souza, PhD, turn the mics on themselves to introduce one another to listeners. Recorded live at the ASGCT 2026 Annual Meeting in Boston, this episode invites listeners to get to know the new co-hosts as they share their backgrounds, ask each other questions, and share how listeners can get involved with future content of The Issue podcast. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community. In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Dylan Bechtle, Director of Regulatory Policy in North America at Johnson & Johnson. Dylan shares how ASGCT membership has been valuable to his career in the regulatory space, both through its community and its resources for staying up to date with the CGT field. Music: 'Origami' 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 episode, recorded on-site at the ASGCT 2026 Annual Meeting, Molecular Therapy Advances Associate Editor-in-Chief Dr. Dan Stone speaks with Dr. Dan DuBreuil of Sanofi. They break down the science behind a recent paper detailing the development of a secretable frataxin, highlighting its potential to improve treatment efficacy for Friedreich’s ataxia. 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.

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community. In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Dr. Mark Deehan of MassGeneral Hospital. Dr. Deehan discusses being a new member of ASGCT and how receiving an ASGCT Career Development Award, funded by the Foundation for Angelman Syndrome Therapeutics (FAST), has advanced his lab work. Music: 'Origami' 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.

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community. In this episode, host Ben McLeod of the ASGCT Communications Committee sits down with Chrissy Green, the co-founder and president of Finding Hope for Frizzle (FRRS1L). As a patient advocate, Chrissy discusses how her parent-led organization is working to develop a treatment for FRRS1L. In addition, she shares how the ASGCT Annual Meeting has given her access to individuals and organizations who can help with the development of the treatment. Music: 'Origami' 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 episode, Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, speaks with Normand Allaire, Head of Genomics at the Cystic Fibrosis Foundation Therapeutics Lab. They discuss the article, Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3’ CFTR PTC variants, which introduces an ASO-based therapeutic approach to cystic fibrosis for the 10% of patients who lack an approved treatment. 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.

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini podcast series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community. In this episode of Member Minutes, get to know Marco Carpenter, PhD, of the Children's Hospital of Philadelphia. Dr. Carpenter is one of the 2026 ASGCT Career Development Award recipients. Interviewed by Ben McLeod of the ASGCT Communications Committee, Dr. Carpenter also shares why ASGCT membership is valuable to his career. Music: 'Origami' 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.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Friday, May 15, 2026 as we talk with Jennifer Adair, PhD, the 2026 recipient of the Award for Excellence in Advancing Diversity, Equity, and Inclusion. She is joined by members of her lab, Motahareh Arjomandnejad, PhD, Rachel Cunningham, PhD, and two visiting scientists from Uganda who are part of an active NIH partnership with the lab to develop capacity for CGT for the treatment of HIV, Dr. George Ssenyange, MD and Gertrude N. Kiwanuka, PhD. The group shares details of their work and their takeaways from the 2026 Annual Meeting. This soundbite is hosted by Ben McLeod of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Friday, May 15, 2026 as we talk with Dr. Daria Fedyukina from ARPA-H, who was a panelist during the Fireside Chat, Engaging with ARPA-H—Funding priorities and future directions. The discussion centers around Dr. Fedyukina’s role at ARPA-H and some of the funded projects and companies she is working with. This soundbite is hosted by Ben McLeod of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Thursday, May 14, 2026 as we talk with Hans-Peter Kiem, MD, PhD, the 2026 recipient of the George Stamatoyannopoulos Mentorship Award. Dr. Kiem reflects on why receiving this award is so meaningful and also shares his thoughts on where the field of CGT is headed. This soundbite is hosted by Ben McLeod of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Thursday, May 14, 2026 as we talk with Defne Amado, MD, PhD, a 2026 ASGCT Career Development Award recipient. Dr. Amado discusses her research in therapies for ALS and discusses what she’s excited about in the field. This soundbite is hosted by Widler Casy, PhD, of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Thursday, May 14, 2026 as we talk with Craig Martin, Founder and CEO of the Orphan Therapeutics Accelerator. Craig discusses the CGTxchange, a new partnership with ASGCT, and how individuals can get involved with the exchange. This soundbite is hosted by Lynnea Olivarez of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Wednesday, May 13, 2026 as we talk with Dawn Bowles, PhD, who walks us through her work at Duke Department of Surgery. This soundbite is hosted by Widler Casy, PhD, a member of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Wednesday, May 13, 2026 as we talk with Claire Booth, MBBS, PhD, who discusses her keynote presentation and what she hopes audiences take away from it. This soundbite is hosted by Wan Du, PhD of the ASGCT Communications Committee.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Wednesday, May 13, 2026 as we talk with Jon Whitton, PhD, VP, Global Program Head of Genetics Medicine at Regeneron. This discussion centers around Regeneron's recent approval of Otarmeni and the role the Annual Meeting plays in advancing innovation across the field. This soundbite is hosted by Lynnea Olivarez of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Tuesday, May 12, 2026 with Sarah Ellinwood, Cynthia Clayton, and Lori Lennon, panelists from the day’s Communications Symposium, Effective communication for funding, peer review, and beyond. The three discuss practical tools and tips for researchers looking to tailor communications for different stakeholders. This soundbite is hosted by Lynnea Olivarez of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Tuesday, May 12, 2026 as we talk with Dr. Timothy Yu, the 2026 recipient of the Jerry Mendell Award for Translational Science, who discusses N=1 therapies and the work being done to bring these therapies to treat more conditions. This soundbite is hosted by Lynnea Olivarez of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Monday, May 11, 2026 with Dr. Justin Eyquem, Associate Professor of Medicine in the Division of Hematology and Oncology at UC San Francisco, who presented at two workshops held during Annual Meeting, Cell therapy at the clinical frontier: Innovations, automation, clinical translation, and Homology-directed repair (HDR) in translation. This soundbite is hosted by Ben McLeod of the ASGCT Communications Committee. Music: Everything Connected by High Street Music.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Recorded and published daily during #ASGCT2026, Soundbites of the Annual Meeting captures the energy and breadth of the conference through quick-hit conversations, scientific snapshots, attendee perspectives, and highlights from across the field — offering a daily pulse on what’s happening throughout the meeting. Take a listen from Monday, May 11, 2026 with Dr. Andrew Wilber of Southern Illinois University School of Medicine, chair and organizer of the Advancing the next generation of tools for precision gene editing in vivo workshop held during Annual Meeting. This episode is hosted by Ben McLeod of the ASGCT Communications Committee. Music: Everything Connected by High Street Music. Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Join host Emily Walsh Martin, PhD, as she hands the baton to the new co-hosts of The Issue podcast, Sanjay D’Souza, PhD, and Jason West, PhD. Listen in as the three preview some of the abstracts that will be presented at the ASGCT 2026 Annual Meeting, held May 11-15 in Boston, MA. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

In this episode, host Emily Walsh Martin revisits her 2022 Caregiver’s Perspective interview with Amber Freed, founder of SLC6A1 Connect. Amber is a caregiver to her son Maxwell, who has since received treatment for SLC6A1. Emily and Amber discuss Maxwell’s treatment and the role a caregiver plays in that journey. Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Emily Walsh Martin, PhD, spoke with Amber Freed, founder and CEO of SLC6A1 Connect, a patient organization focused on accelerating the development of therapies for SLC6A1 patients like her son Maxwell.Show your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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