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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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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.

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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.

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We’re sharing this Better Biopharma episode on Cell & Gene: The Podcast because Better Biopharma is a sister show in the Life Science Connect family, and this conversation touches so many of the same challenges, pressures, and big-picture shifts our audience is thinking about right now. CGT doesn’t live in a bubble, and neither do the people building it. So we wanted to bring this wider, cross-industry conversation straight to you.

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In episode 119 of Cell & Gene The Podcast, Host Erin Harris talks to Dr. Norman Putzki, Global Head Clinical Development, Novartis, about the FDA approval of Itvisma, now the only gene replacement therapy approved for children, adolescents, and adults with spinal muscular atrophy (SMA). Dr. Putzki walks us through the six-year development journey behind the STEER and STRENGTH Phase 3 programs. And we explore what the expanded age-range label means for patients who were previously left behind, why intrathecal, fixed-dose AAV delivery represents a pivotal advance for safety, efficacy, and scalability. He details how the Itvisma program is informing Novartis’ broader gene therapy strategy across neuromuscular and CNS diseases, and more.

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In Episode 118, Host Erin Harris talks to Alexander Cryer, Ph.D., Instructor in Medicine at Mass General Brigham, about a proof of concept strategy that reprograms tumor cells with mRNA lipid nanoparticles to overactivate the cGAS-STING pathway, forcing cancer cells to produce and export large amounts of the innate immune agonist cGAMP to stimulate surrounding immune cells and drive anti-tumor immunity. Dr. Cryer explains the basic biology of cGAS-STING and how his team restored this pathway in tumor cells and leveraged intratumoral LNP delivery to overcome nucleic acid delivery and targeting challenges. He also discusses future directions, the need to move beyond intratumoral administration with more targeted systemic delivery, and the broader concept of turning the tumor’s own abundant cell population and evolutionarily conserved innate immune pathways into therapeutic allies rather than obstacles.

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On episode 117, Host Erin Harris welcomes Paul Perreault, Strategic Advisor, Board Member and former CEO of CSL, who shares insights from his decade leading the company through global expansion and the COVID-19 pandemic. Perreault highlights the importance of patient-centered culture, strategic focus, and adaptive leadership for cell and gene therapy innovators. He offers practical advice on building a values-driven culture that connects employees to patients, the importance of focusing on core competencies to attract capital, and the necessity of continuous leadership development, even advocating for mentorship and emotional intelligence as growth drivers in fast-evolving organizations.

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In episode 116 of Cell & Gene: The Podcast, Host Erin Harris talks to Dr. Arun Upadhyay, Chief Scientific officer, Head of Research and Development at Ocugen to discuss how the company’s modifier gene therapy platform is redefining treatment possibilities for inherited retinal diseases such as retinitis pigmentosa, Stargardt disease, and geographic atrophy. Dr. Upadhyay explains how Ocugen’s gene-agnostic approach differs from traditional single-gene therapies by targeting shared disease pathways to preserve photoreceptors and slow vision loss across diverse genetic mutations. He also shares key lessons learned from advancing OCU400, the first modifier gene therapy to receive a broad FDA indication for retinitis pigmentosa, including challenges in clinical trial design, endpoint selection, and manufacturing scalability.

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