Cell & Gene: The Podcast

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On episode 126 of Cell & Gene: The Podcast, Host Erin Harris welcomes Catriona Jamieson, M.D., Ph.D, Director of the UC San Diego Sanford Stem Cell Institute and Professor of Medicine at the School of Medicine. They explore how shifting the medical mindset from “common horses” to rare “zebras” is unlocking earlier detection and more effective treatment of diseases like myelofibrosis. Dr. Jamieson explains how subtle, often overlooked symptoms can mask serious underlying conditions, and how advances in genomic testing are enabling clinicians to identify disease-driving mutations sooner. They cover groundbreaking progress in targeted therapies, which aim to stop cancer progression and overcome treatment resistance. 
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On episode 125 of Cell & Gene: The Podcast, Host Erin Harris talks to Halloran Consulting Group's Monika Swietlicka to discuss how the FDA is increasing flexibility in cell and gene therapy development without lowering evidentiary standards, emphasizing a risk-based, holistic approach. They also explore what this means for developers, from platform strategies and global regulatory divergence to the growing role of patient advocacy and the need for early, integrated planning.
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On episode 124 of Cell & Gene: The Podcast, Aurora Therapeutics' CEO Dr. Edward Kaye discusses the company’s strategy for translating CRISPR gene editing into scalable, commercially viable medicines for rare diseases. Aurora is initially targeting phenylketonuria (PKU) using a platform approach that leverages shared components, such as lipid nanoparticles and base editors, while customizing guide RNAs for specific mutations. Dr. Kaye explains how trials, regulatory flexibility, and optimized manufacturing could make it possible to treat many genetic variants efficiently and cost-effectively. Ultimately, Aurora aims to build a repeatable model that expands gene editing access to larger rare-disease populations while keeping patients at the center of development.
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In episode 123 of Cell & Gene: The Podcast, Host Erin Harris talks to Dr. Panteli Theocharous, FIBMS, M.S., Ph.D., FRCPath, about the patient journey in cell therapy trials. They pinpoint key friction points, such as delayed referrals, unpredictable vein-to-vein timelines, and burdensome long-term follow-up, while sharing actionable strategies for simplification. These strategies range from upstream trial design and streamlined consent processes to standardized logistics, hybrid monitoring models, honest risk communication, and engaging patients as true partners in real-world evidence generation.
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In episode 122 of Cell & Gene: The Podcast, Host Erin Harris talks to Paul Romness, CEO of OS Therapies, to learn the company’s mission to address the severe unmet need in pulmonary metastatic osteosarcoma (PMO), a rare pediatric cancer with no established standard of care once it metastasizes. Romness explains how OS Therapies’ off-the-shelf HER2-targeted immunotherapy aims to significantly improve outcomes by stimulating a robust immune response with minimal side effects. He highlights results from a multicenter Phase 2B trial showing markedly improved overall survival rates compared to historical outcomes, details the company’s constructive regulatory interactions with the FDA, and underscores the value of comparative canine biomarkers in development.
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