Gene Therapy Delivery Decoded: Surgical Routes, Vector Targeting, & Immune Mitigation in the Eye

Gene therapy is no longer just a treatment; it’s a delivery system. By delivering a gene instead of a protein, developers can enable continuous in-eye production of therapeutic proteins, potentially reducing injection burden for years. But this approach hinges on getting the right vector to the right cells, through the right route, with minimal immune response. This workshop will explore gene therapy as a delivery modality, comparing subretinal, intravitreal, and suprachoroidal approaches through the lens of real-world feasibility, safety, and vector precision.

Key Topics to be Explored:

  • How does gene therapy function as sustained drug delivery, and what conditions require protein production inside specific retinal cells like rods and cones?
  • Why does subretinal delivery remain the method of choice for precision targeting and what are the surgical trade-offs, including risk of retinal detachment?
  • What’s the role of AAV serotype selection (e.g., AAV2 vs. AAV8) in targeting the correct retinal layers, and how does that impact safety and efficacy?
  • How do intravitreal and suprachoroidal injections compare in terms of anatomical access, procedural simplicity, and delivery to deep retinal tissues?
  • What strategies are being developed to mitigate immunogenicity, and how are vector surfaces being modified to reduce inflammation?
  • Why is systematic vector testing still lacking, and how do current manufacturing challenges limit the scalability of gene therapy delivery systems in ophthalmology?