About Event

Welcome to the 4th Annual Gene Therapy for Ophthalmic Disorders Summit

The industry-leading, industry-focused forum for learning how to validate novel administration routes to the eye, extrapolate effectively from preclinical models to inform safety and efficacy, and define relevant endpoints for clinical trials.

Across 3 days of in-depth case studies, new data, and focused group discussions, attendees heard from and engaged with the likes of Janssen, Adverum Biotechnologies, Spark Therapeutics, SparingVision and the RD Fund. As well as leveraging insights from these talks and panels: attendees participated in breakout discussions and utilized Q&A time to meet and learn from ophthalmic gene therapy leaders during our 5+ hours of dedicated networking time.


This Summit Brought Together Leading Voices on:

Defining Relevant and Meaningful Endpoints

Investigated the latest trends in endpoint development and regulation, understood their applicability in animal model studies to clinical trials, and discussed the need for validated novel endpoints, with key insights from Adverum Biotechnologies and SparingVision.

Levelling up the Translatability of Preclinical Models

Addressed the best practices in animal model selection and development, optimized the clinical relevancy of animal model studies, and leveraged the latest retinal organoid developments, with expert sessions hosted by Atsena Therapeutics and MeiraGTx.

Exploring Different Delivery Methods to the Front and Back of the Eye

Took a deep dive into the suprachoroidal route, overcame the AAV cargo capacity challenge with novel administration methods, and heard the latest updates on robotics-assisted subretinal delivery, with exciting discussions from Clearside Biomedical and Splice Bio.

Understanding the Immune and Inflammatory Responses to Ocular Gene Therapies

How can improved promoters and enhancers deliver genes effectively without triggering the immune response? Which preclinical models are best suited to test the various variables associated with GTAU? What are the latest trends in immunosuppressant regimens being used in the field? AGTC and Kriya Therapeutics answered these questions, and many more.

Leveraging Insights from Ophthalmology-Focused Venture Capital

Discovered the current investment landscape for gene therapy in the ophthalmology field, and understood what venture capital investors are looking for when it comes to developing preclinical, clinical, and manufacturing packages in the most fruitful, cost-effective manner, with key insights from the RD Fund and Syncona.

Jie Shen - AbbVie

“We are seeing an explosion of gene therapy development effort for ocular diseases that can be ‘one-and-done’ to alleviate treatment burden for our patients.”

Jie Shen, Vice President, Translational Science, AbbVie