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This is Episode 3 of "FDA Fridays," a 4-week special series from Cell & Gene: The Podcast. Host Erin Harris talks to Bambi Grilley, Ph.D., Professor of Pediatrics and the Director of Clinical Research and Early Product Development for the Center of Cell and Gene Therapy (CAGT) at Baylor College of Medicine and Chief Regulatory Officer for ISCT, a leading expert at the forefront of pediatric cell and gene therapy. Dr. Grilley shares her wealth of experience and unique perspective on the most pressing barriers to accessing transformative treatments for children, from logistical and financial challenges faced by families, to systemic and regulatory hurdles in getting therapies from the lab to the clinic.

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Episode 2 of FDA Fridays, the special series from Cell & Gene: The Podcast, features Host Erin Harris' discussion with regulatory expert Monika Swietlicka, Principal, Regulatory Strategy, Halloran Consulting Group about the key strategies and challenges in navigating FDA regulation for cell and gene therapies. Their conversation covers the importance of defining a therapy’s intended role (curative, chronic, or adjunctive) early in development, the different regulatory expectations based on disease context and patient population, and the crucial impact of unmet medical need on regulatory flexibility. Swietlicka emphasizes the necessity of a clear, scientifically sound regulatory narrative, especially when precedent is lacking, and advocates for early, robust investment in CMC to minimize risks. They also explore the complexities of using surrogate endpoints, planning for long-term patient follow-up under uncertainty, and the value of integrating commercial considerations from the outset. Swietlicka calls for cell and gene therapy developers to adopt a commercial mindset and proactive regulatory engagement to achieve clinical and commercial success.

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In Episode 109 of Cell & Gene: The Podcast, Host Erin Harris talks to Will Chou, M.D., CEO of Passage Bio about the promise and challenges of developing gene therapies for frontotemporal dementia (FTD), a neurodegenerative disease with strong genetic underpinnings. Dr. Chou discusses the biological basis of FTD and the complex scientific, manufacturing, and regulatory hurdles that come with targeting the brain. They discuss the latest interim results from the upliFT-D study of Passage Bio’s PBFT02, including safety and tolerability findings, dose cohort comparisons, and the evolving strategies around immunosuppression. Dr. Chou also highlights upcoming milestones for PBFT02 and what they could mean for advancing gene therapies in neurodegenerative diseases.

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This is Episode 1 of a special series of Cell & Gene: The Podcast, "FDA Fridays." This first episode features Host, Erin Harris' conversation with Kate Spratt, Strategic and Non-Clinical Regulatory Consultant at Spratt Advanced Cell & Gene Therapy Regulatory Consulting. Spratt emphasizes the importance of early and continuous engagement with regulatory authorities, especially through structured meetings like INTERACT and pre-IND sessions. Harris and Spratt cover CMC readiness, filing for RMAT/CIT, BLA approvals and regulatory expectations, and more. 

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Cell & Gene: The Podcast Host, Erin Harris, talks to Orca Bio CEO and Co-Founder Nathaniel Fernhoff as he recounts the company’s origins and evolution, tracing its roots to pioneering stem cell research at Stanford University and Dr. Irv Weissman's lab. Seeking to create safer, more effective cures for blood cancers such as AML, ALL, and MDS, Orca Bio developed high-precision cell therapies that utilize purified regulatory T cells and stem cells to minimize complications, such as graft-versus-host disease. Orca Bio spun out of academia to establish its own scalable production platform and clinical pipeline, and Fernhoff explains Orca's flagship product, Orca T, which recently achieved a pivotal milestone by outperforming standard stem cell transplants in a Phase 3 trial.

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On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Ralf Schmid, Ph.D., Associate Director of Preclinical Research at Novartis Biomedical Research, about the evolving use of large animal models in gene therapy development. Dr. Schmid discusses the current reliance on non-human primates (NHPs), their growing logistical and ethical challenges, and the emerging interest in alternatives like genetically engineered pigs and sheep. He outlines key considerations around safety, biodistribution, and immunogenicity that still necessitate large-animal testin —particularly for CNS-targeted AAV therapies — and emphasizes the need for thoughtful study design, responsible sourcing, and diversification in model systems. Dr. Schmid also previews his participation in the upcoming Next Generation Gene Therapy Vectors Summit and reflects on the future of preclinical safety testing in a landscape aiming to balance innovation, rigor, and compassion.

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In this episode of Cell & Gene: The Podcast, George Magrath, CEO of Opus Genetics, shares with Host, Erin Harris, how the company is advancing gene therapies for inherited retinal diseases (IRDs), with a focus on LCA5, a rare and severe form of childhood blindness. Built on the foundational science of Dr. Jean Bennett and leveraging the legacy of Luxturna, Magrath shares how Opus is developing subretinal AAV-based therapies targeting seven distinct mutations. He also explains why gene therapy is particularly well-suited to IRDs, shares compelling early clinical data, and discusses the promise of early intervention in preserving vision and enabling proper neural development. 

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In this episode of Cell & Gene: The Podcast, Host Erin Harris interviews Dr. Gloria Matthews, Chief Medical Officer of Aegle Therapeutics, a clinical-stage regenerative medicine company focused on developing therapies for rare and severe dermatologic and immunologic disorders. Dr. Matthews explains how Aegle is pioneering the use of extracellular vesicles (EVs) derived from mesenchymal stem cells, which offer advantages over traditional cell-based therapies. Their discussion highlights Aegle's lead product, AGLE-102 as well as the regulatory and manufacturing challenges of scaling EV-based therapies. They cover the broader therapeutic potential of EVs in areas such as graft-versus-host disease, ocular, and orthopedic disorders, and more.

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On this episode of Cell & Gene: The Podcast, Host Erin Harris welcomes Monika Swietlicka, Principal, Regulatory Strategy at Halloran Consulting Group to discuss the key regulatory challenges facing cell and gene therapy companies. Swietlicka highlights manufacturing complexity and clinical trial design limitations. On gene editing, she notes that the FDA is focused on off-target effects and demands robust data, including functional validation. She emphasizes the importance of early definition of critical quality attributes and a strong comparability strategy to ensure product consistency. She also underscores the growing role of real-world evidence, especially in rare disease settings with limited clinical trial data.

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Host Erin Harris talks to Generate:Biomedicines' EVP of R&D, Dr. Alex Snyder about the convergence of AI, machine learning (ML), and synthetic biology in the development of next-generation therapies. They cover how AI is transforming drug discovery by enabling the rapid design and optimization of therapeutic candidates, particularly in complex fields like immuno-oncology and cell therapy. Dr. Snyder shares how that in the context of CAR-T therapies, AI-driven approaches are used to design and refine each component of the CAR construct. And, Dr. Snyder sets the stage for a broader conversation about how the integration of AI and synthetic biology is not only accelerating drug development timelines but also expanding the realm of what’s possible in cell-based therapeutics.

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