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Pushing the Frontiers of Inherited Retinal Disease Trials with Prof. Michel Michaelides
In this episode of the BroadEye Podcast, hosts Shawn Maloney and Dr. Bruno Fernandes welcome Prof. Michel Michaelides, one of the most active clinical researchers in ophthalmology today. Prof. Michaelides leads a large inherited retinal disease clinic—seeing 30–40 children and adults each week—and splits her time between patient care, advanced imaging research, and steering more than ten ongoing clinical trials.
Why This Matters
Inherited retinal diseases affect hundreds of thousands worldwide, and for most there is still no approved treatment. Over the past two years, we’ve seen multiple Phase 1/2 gene-therapy trials move into registrational studies, alongside novel antioxidant approaches and AI-driven biomarkers. Today’s conversation cuts through the jargon to give you a clear picture of where the field stands—and where it’s headed.
Key Takeaways
Beyond RPE65: While Luxturna paved the way, newer AAV-based therapies for X-linked RP (RPGR) and other targets are now in Phase 2 and Phase 3 trials, with early data showing promising safety and efficacy.
Imaging & Function: Prof. Michaelides is pioneering next-generation retinal imaging devices and novel functional assays to capture meaningful changes in vision—critical for shortening trial timelines.
Antioxidant Strategies: High-dose N-acetylcysteine trials are under way, offering a non-viral approach to slow photoreceptor degeneration across multiple genetic subtypes.
AI in Stratification: Machine-learning algorithms on retinal scans can help predict progression rate and identify the best candidates for specific therapies, improving both trial design and patient outcomes.
Balancing Dose & Safety: Finding the sweet spot between transgene expression and immune activation remains a core challenge—Prof. Michaelides shares lessons learned from dose-escalation cohorts.
Phase 3 & Beyond: With over a hundred patients enrolled in pivotal studies, we’re closing in on potential approvals—but long-term follow-up and real-world evidence will determine ultimate impact.
About the Guest
Prof. Michelle Michaelides is a clinician-scientist specializing in inherited retinal diseases. She leads dedicated adult and pediatric IRD clinics, oversees the development of advanced imaging and functional biomarkers, and serves as principal investigator on over ten clinical trials spanning gene therapies, antioxidant agents, and AI-guided stratification tools. Prof. Michaelides regularly lectures at international conferences and collaborates with industry and academic partners to accelerate the translation of research into real-world treatments.
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By Bruno Fernandes and Shawn Maloney
5
33 ratings
In this episode of the BroadEye Podcast, hosts Shawn Maloney and Dr. Bruno Fernandes welcome Prof. Michel Michaelides, one of the most active clinical researchers in ophthalmology today. Prof. Michaelides leads a large inherited retinal disease clinic—seeing 30–40 children and adults each week—and splits her time between patient care, advanced imaging research, and steering more than ten ongoing clinical trials.
Why This Matters
Inherited retinal diseases affect hundreds of thousands worldwide, and for most there is still no approved treatment. Over the past two years, we’ve seen multiple Phase 1/2 gene-therapy trials move into registrational studies, alongside novel antioxidant approaches and AI-driven biomarkers. Today’s conversation cuts through the jargon to give you a clear picture of where the field stands—and where it’s headed.
Key Takeaways
Beyond RPE65: While Luxturna paved the way, newer AAV-based therapies for X-linked RP (RPGR) and other targets are now in Phase 2 and Phase 3 trials, with early data showing promising safety and efficacy.
Imaging & Function: Prof. Michaelides is pioneering next-generation retinal imaging devices and novel functional assays to capture meaningful changes in vision—critical for shortening trial timelines.
Antioxidant Strategies: High-dose N-acetylcysteine trials are under way, offering a non-viral approach to slow photoreceptor degeneration across multiple genetic subtypes.
AI in Stratification: Machine-learning algorithms on retinal scans can help predict progression rate and identify the best candidates for specific therapies, improving both trial design and patient outcomes.
Balancing Dose & Safety: Finding the sweet spot between transgene expression and immune activation remains a core challenge—Prof. Michaelides shares lessons learned from dose-escalation cohorts.
Phase 3 & Beyond: With over a hundred patients enrolled in pivotal studies, we’re closing in on potential approvals—but long-term follow-up and real-world evidence will determine ultimate impact.
About the Guest
Prof. Michelle Michaelides is a clinician-scientist specializing in inherited retinal diseases. She leads dedicated adult and pediatric IRD clinics, oversees the development of advanced imaging and functional biomarkers, and serves as principal investigator on over ten clinical trials spanning gene therapies, antioxidant agents, and AI-guided stratification tools. Prof. Michaelides regularly lectures at international conferences and collaborates with industry and academic partners to accelerate the translation of research into real-world treatments.