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.

In this enlightening conversation, Luigi Naldini, a pioneer in gene therapy and a professor at San Raffaele University, shares his remarkable journey from Torino to groundbreaking research. He discusses the innovative use of lentiviral vectors and their potential to cure genetic disorders and cancer. Naldini addresses challenges in making gene therapies accessible in low-income countries and emphasizes the importance of diverse representation in genomic research. He also offers insights for aspiring scientists and explores the joy of balancing work with personal passions like art and music.

Terry Flotte, MD, a leading figure in gene therapy and Dean at UMass Chan Medical School, shares his journey from childhood inspiration to groundbreaking research. He discusses the pioneering work in AAV gene therapy for cystic fibrosis, emphasizing safety and technological evolution. Alongside ASGCT President Hans-Peter Kiem, MD, PhD, they explore the future of gene therapy, touching on exciting advancements aimed at treating genetic disorders. Flotte's passion for pediatrics and personal interests, including baseball and obscure films, add a delightful touch.

Jennifer Doudna, a renowned scientist and co-inventor of CRISPR-Cas9, shares her inspiring journey from rural Hawaii to winning the 2020 Nobel Prize in Chemistry. She discusses the groundbreaking evolution of CRISPR technology and its revolutionary potential in health and agriculture. Doudna emphasizes the importance of ethical considerations in gene editing and advocates for accessibility in scientific advancements. Additionally, she highlights the need for mentorship and diversity in STEM, all while balancing her professional pursuits with personal passions like hiking.

Drew Weissman, a pioneer of mRNA technology and professor at the University of Pennsylvania, shares his revolutionary journey in vaccine development. He details the critical breakthroughs that led to the successful mRNA vaccines and discusses the ongoing innovations aimed at combating diseases like HIV and malaria. Drew emphasizes the importance of global vaccine access and the role of mentorship in science. His insights offer hope for future advancements in gene therapy, showcasing the potential of mRNA technology to transform medicine.

Carl June, MD, a pioneer in immunotherapy and director of the Center for Cellular Immunotherapy at UPenn, shares his groundbreaking journey in gene therapy. He details his transition from engineering to medicine and recounts the pivotal moments that led to FDA approval for CAR T-cell therapy. Dr. June shares inspiring patient stories, discusses future innovations in cell therapies, and addresses the challenges of work-life balance in with scientific careers. He even reveals his passion for extreme biking as a metaphor for perseverance in both life and science.

Kathy High, MD, a pioneer in gene therapy and former president of Spark Therapeutics, shares her inspiring journey from working with hemophilia to advancing retinal disease treatments. Joined by Hans-Peter Kiem, MD, PhD, they delve into the challenges of developing gene therapies, including the complexities of AAV vectors and the hurdles of clinical trials for children. They also discuss recent innovations like synthetic AAVs, the importance of collaboration in biotech research, and offer wisdom for early-career hematologists pursuing their passions.