Cell & Gene: The Podcast

Scaling CRISPR for Rare Disease with Aurora Therapeutics' Dr. Edward Kaye

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Simplifying Friction in Cell Therapy Clinical Trials with Dr. Panteli Theocharous

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Inside a Breakthrough HER2 Immunotherapy for PMO with OS Therapies’ Paul Romness

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Building Safer CRISPR Medicines for CVD with Scribe Therapeutics' Benjamin Oakes

We love to hear from our listeners. Send us a message.In episode 121 of Cell & Gene: The Podcast, Host Erin Harris talks to Scribe Therapeutics' CEO and Co-Founder Benjamin Oakes about building next‑generation CRISPR and epigenetic editing tools to move genetic medicine beyond rare disease into common cardiometabolic indications. Oakes shares Scribe’s engineered CasX platform and epigenetic silencers, preclinical data from its various programs, and why exquisite specificity and low-dose LNP delivery are essential to treating patients safely. They also explore Scribe’s partnerships with Sanofi and Lilly, the company’s cardiometabolic-first strategy co-developed with Dr. Jennifer Doudna, and Oakes’ conviction that genetic medicines can fundamentally reshape healthspan and the future of preventive cardiovascular care.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Advancing Off-the-Shelf CRISPR CAR-T Therapies into the Community Setting with Caribou Biosciences' Rachel Haurwitz

We love to hear from our listeners. Send us a message.Welcome to episode 120 of Cell & Gene: The Podcast. Host Erin Harris is joined by Rachel Haurwitz, CEO of Caribou Biosciences, to discuss the company’s progress in developing CRISPR-edited, off-the-shelf CAR-T therapies for hematologic malignancies. Their conversation centers on Vispacell, Caribou’s allogeneic CD19 CAR-T for second-line large B-cell lymphoma. Haurwitz explains how Caribou has systematically optimized its allogeneic platform using clinical and translational data. They also cover pivotal Phase 3 trial planning, regulatory considerations, and what to expect next from Caribou’s broader pipeline, including its BCMA-targeted program in multiple myeloma.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn