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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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In this episode of Cell & Gene: The Podcast, Host Erin Harris sits down with Peter Altman, Ph.D., CEO of BioCardia, to discuss the company’s evolution from a cardiac biotherapeutic delivery firm to a developer of autologous and allogeneic cell therapies for cardiovascular and pulmonary diseases. Altman highlights BioCardia’s CardiAMP program, a precision medicine approach that pre-screens patients based on their cell profiles to improve trial outcomes and reduce costs. He explains why no cardiac cell therapy has yet received FDA approval, citing challenges in delivery, immune rejection, and arrhythmia risks. Altman outlines BioCardia’s near-term roadmap, including regulatory submissions in the U.S. and Japan and the launch of a second trial focused on patients most likely to benefit.

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In this episode of the Cell & Gene podcast, Host Erin Harris speaks with Alicia Zhou, Ph.D., CEO of the Cancer Research Institute (CRI), about CRI’s mission to advance cancer immunotherapy and the critical role of federal funding, particularly from the NIH, in supporting basic and translational cancer research. Dr. Zhou highlights CRI’s focus on funding early-stage science and clinical trials to move immunotherapy toward curing all cancers. She covers the serious implications of recent U.S. Government funding cuts, including NIH budget freezes and halted grant review processes, which are already impacting biomedical research institutions, graduate programs, and clinical trials. In light of the NIH budget cuts, Dr. Zhou explains how CRI is stepping up by committing an additional $2.5 million from its reserves to fund 10 postdoctoral fellowships.

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On the 100th episode of Cell & Gene: The Podcast, Host, Erin Harris, sits down with Audrey Greenberg, Founder and CEO of AG Capital Advisors. Audrey is the award-winning executive, board director, and strategic advisor with a proven track record of launching, scaling, and successfully exiting multi-billion-dollar ventures. They explore the biggest challenges and opportunities facing CGT biotech leaders today; the funding crisis in biotech, the evolving investment landscape, and whether the traditional VC-driven model is still sustainable. They also examine what it really takes to scale a biotech company from startup to market leader, the role of AI-driven drug discovery, and how big tech’s growing influence could reshape the industry. You'll hear their discussion on leadership challenges in biotech, the need for more diverse voices at the top, and what must change to empower the next generation of women leaders in CGT, and more.

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In this episode of Cell & Gene: The Podcast, Host Erin Harris dives into the world of retinal gene therapy with Kenji Fujita, M.D., Chief Medical Officer of Atsena Therapeutics. Atsena develops treatments for inherited retinal diseases, including X-linked retinoschisis (XLRS), a rare genetic condition that currently has no FDA-approved therapy. They talk through the impact of XLRS on patients, the challenges of gene delivery to the retina, and how Atsena’s AAV vector technology is designed to overcome these hurdles. They also discuss the significance of the Fast Track designation recently granted to Atsena’s XLRS program, the role of patient advocacy in rare disease research, and the key milestones to watch for in the months ahead.

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In this episode, Host Erin Harris sits down with Dr. Simrit Parmar, Founder of Cellenkos, to explore how the company is pioneering umbilical cord blood-derived Regulatory T cell (Treg) cell therapies for autoimmune and inflammatory diseases. Dr. Parmar shares the vision behind Cellenkos, the advantages of cord blood-derived Tregs over other sources, and how their CRANE technology platform enhances precision in targeting diseases. They also discuss key findings from their Phase 1b trial for CK0804, the challenges of scaling up off-the-shelf Treg therapies, and what’s next for Cellenkos in 2025.

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