Cell & Gene: The Podcast

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In Episode 3 of our in vivo-focused special series of Cell & Gene: The Podcast, Ascidian Therapeutics' Founder, President, and CEO Mike Ehlers, M.D., Ph.D., explains how the company is advancing a new class of genetic medicines built on RNA exon editing to durably correct disease at the transcript level without permanently altering DNA. He also shares why in vivo therapies still need to prove precision, durability, and predictability before they become mainstream, and why the field must move beyond proof of concept to reproducible clinical performance across patients and diseases. He discusses the importance of patient selection, tissue specificity, and matching editing efficiency to biological thresholds, and much more. 
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In episode 2 of this four-part in vivo-focused special series of Cell & Gene: The Podcast, Host Erin Harris sits down with Gopi Shanker, Ph.D., Chief Scientific Officer at Beam Therapeutics, for an in-depth conversation on the rapidly evolving landscape of in vivo gene editing and precision genetic medicines. Dr. Shanker discusses how next-generation base editing technologies are advancing beyond traditional CRISPR approaches by enabling precise single-base changes without creating double-stranded DNA breaks. They discuss the growing momentum behind in vivo delivery strategies, including targeted LNP technologies designed to reach tissues beyond the liver, as well as the operational and patient-centric advantages these approaches may offer compared to ex vivo therapies. 
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In episode 130 of Cell & Gene: The Podcast, Dr. Naji Gehchan, Chief Medical and Development Officer at Kyverna Therapeutics, joins Host Erin Harris to outline the company’s mission to apply CAR T-cell therapy beyond oncology to treat severe autoimmune diseases, highlighting promising Phase 2 data from its investigational therapy, Miv-cel, in stiff person syndrome (SPS). Dr. Gehchan explains that Miv-cel works by deeply depleting pathogenic B cells and enabling a broad immune reset, differentiating it from conventional therapies that require ongoing management. As Kyverna advances a rolling BLA submission, Dr. Gehchan positions this milestone as potentially historic, both for SPS patients and for the broader cell therapy field, signaling an inflection point where CAR T could transform autoimmune disease care in the same way it has oncology.
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This is Episode 1 of a four-episode in vivo-focused special series of Cell & Gene: The Podcast. Host Erin Harris speaks with Cassie Gorsuch, Ph.D., CSO at Precision Biosciences, about the rapid evolution of in vivo gene editing and the scientific, translational, and regulatory hurdles shaping the field. Dr. Gorsuch discusses how Precision Biosciences approaches in vivo therapeutic development through its Arcus platform, with programs targeting chronic hepatitis B and Duchenne muscular dystrophy. They cover the broader challenges facing in vivo gene editing, including delivery limitations outside the liver, balancing specificity and efficiency, mitigating off-target risks, and translating promising preclinical in vivo data into clinical success.
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In episode 129 of Cell & Gene: The Podcast, Host Erin Harris reconnects with Zachary Roberts, M.D., Ph.D., EVP of Research & Development and Chief Medical Officer at Allogene Therapeutics, to explore how allogeneic CAR-T is evolving from a scalability promise into an earlier-line, potentially curative intervention. Their conversation centers on the ALPHA3 trial, where MRD-guided treatment is redefining when and how CAR-T can be deployed, alongside broader implications for diagnostics, patient access, and the shift toward off-the-shelf therapies in community settings.
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