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

Drew Weissman, MD, PhD is a pioneer of mRNA technology, which has been used in more than 1 billion administered doses of COVID-19 vaccines. Weissman is the Roberts Family Professor in Vaccine Research, Director of Vaccine Research in the Infectious Disease Division, and the Director of the Institute for RNA Innovation at the University of Pennsylvania and the Perelman School of Medicine. He co-developed the mRNA technology that made the COVID-19 vaccines possible. His inventions were used in the Pfizer and Moderna vaccines, and have the potential to treat a range of medical conditions. He is also promoting global vaccine access by working with governments and universities around the world. Music by: Steven O'Brien https://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/donate See omnystudio.com/listener for privacy information.

Carl June, MD, shares his scientific journey, from medical research officer in the U.S. Navy to leader behind the first FDA-approved gene therapy, during his one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the third episode of Giants of Gene Therapy! Dr. June is a professor in immunotherapy in the Department of Pathology and Laboratory Medicine at University of Pennsylvania's Perelman School of Medicine, the director of the Center for Cellular Immunotherapy, and the director of the Parker Institute for Cancer Immunotherapy at Penn. He was instrumental in bringing CAR T cells to the clinic and receiving the first FDA approval for CAR T cell therapy in 2017. Dr. June is a prolific researcher who has published more than 500 manuscripts. He's received numerous awards and honors and has been the founder or co-founder of many companies involved in T cell therapies. His discoveries launched a new era of cancer treatment options. Dr. June has been an ASGCT member and Annual Meeting keynote speaker. Music by: Steven O'Brien https://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/donate See omnystudio.com/listener for privacy information.

Kathy High, MD, shares her personal and scientific journey, from hemophilia to retinal disease, during her one-on-one conversation with American Society of Gene & Cell Therapy President Hans-Peter Kiem, MD, PhD, in the second episode of ASGCT's Giants of Gene Therapy. Dr. High is the president of therapeutics at AskBio. Before that, she was the co-founder, president, and head of research and development at Spark Therapeutics, where she oversaw the development, approval, and launch of Luxturna, the first AAV gene therapy approved for use in the United States. Dr. High has published extensively in the space of gene therapy and received many highly prestigious awards for her work. She is also a past president of the American Society of Gene & Cell Therapy. Music by: Steven O'Brien https://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/donate See omnystudio.com/listener for privacy information.