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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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