Medical Genetics in Ophthalmology: Through the Lens of Genetics-Informed Therapies OnDemand-Patient Perspectives on Gene Therapy

Video Transcription

Video Summary

Amy Laster from the Foundation Fighting Blindness shares patient perspectives on ocular gene therapies, emphasizing the transition from experimental promise to practical treatment. The Foundation's My Retina Tracker Registry, with over 43,000 participants, accelerates therapy development by collecting genetic and clinical data, facilitating research and trial recruitment. Patients consistently prioritize functional benefits such as independence and navigation, often feeling conventional clinical endpoints don't reflect real-world improvements. There is high interest in therapies, but less than half feel adequately informed, highlighting a need for better patient education. Key patient concerns include therapy safety, accessibility, durability, and understanding long-term effects—especially for pediatric treatments. Patients weigh "is it worth it?" over "will it work?" focusing on quality of life over clinical measures. The rise from few to over 100 clinical trials has increased patient sophistication in decision-making, with a shift toward real-world outcomes driving therapy evaluation. Collaboration with researchers and global patient communities remains crucial for advancing meaningful ocular treatments.

Asset Subtitle

Amy Laster, BS, PhD

Keywords

ocular gene therapies

My Retina Tracker Registry

patient perspectives

functional benefits

therapy safety and accessibility

clinical trials and real-world outcomes