A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire

In this episode of The Issue, CEO of Life Biosciences Jerry McLaughlin joins hosts Sanjay D’Souza, PhD and Jason West, PhD for a discussion on how epigenetic reprogramming could restore function in aging and injured cells. He explains the science behind ER-100, an investigational gene therapy now in a Phase 1 trial for optic neuropathies, and explores the broader promise of targeting age-related diseases to help improve quality of life. 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/donate See omnystudio.com/listener for privacy information.

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