May 2, 2015 1:00 p.m. – 2:00 p.m. Check-In & Networking 2:00 p.m. - 5:30 p.m. Energy Ballroom Homewood Suites Denver Downtown Convention Center Denver, Colorado

Agenda

1:00 p.m. – 2:00 p.m. Workshop Check-In and Materials Pickup
2:00 p.m. – 2:15 p.m. Welcome Remarks by Dr. Paul A. Sieving, Director, NEI
2:15 p.m – 2:45 p.m. Photoreceptor Regeneration Overview
Drs. David Gamm and Rachel Wong

Overview of categories and framework for discussion
2:45 p.m. – 4:15 p.m. Gaps in Our Knowledge and Barriers to Scientific Progress Category 1: Cell Sources for Photoreceptor Regeneration
  1. What are the best donor source options for exogenous PR replacement?
  2. What host cells can be targeted for endogenous PR replacement?
  3. How can the production of bona fide PRs be optimized?
  4. Are distinct strategies needed for cone versus rod PR production?

Category 2: Delivery and Integration

  1. What are optimal approaches for introducing donor cells into host retina for exogenous PR replacement strategies?
  2. What are optimal approaches for introducing genes and factors into host retina for endogenous PR replacement strategies?
  3. Can survival and synaptogenesis of newly established PRs be optimized?

Category 3: Outcome Assessment

  1. How do we distinguish PR replacement from rescue or other indirect effects?
  2. What technology (not currently available) is most needed to ensure true PR replacement and functional integration in patients?
  3. What are relevant anatomic and functional outcomes?

Category 4: Animal Models and Target Patient Populations

  1. What can we hope to learn from existing and future animal models?
  2. Do suitable animal model systems exist and how do we assimilate findings across them?
  3. What gaps in knowledge of human retinal disease need to be addressed to optimize application of PR replacement therapies?
  4. To what extent should the FDA and clinicians be engaged at this stage of research?
4:15 p.m. – 4:30 p.m. Break
4:30 p.m. – 5:00 p.m. Areas for Open-Ended/Non-Hypothesis Driven/Discovery Research
5:00 p.m. – 5:30 p.m. The Future of Photoreceptor Regeneration & Concluding Remarks
How long will it take to reach milestones?
Last updated: July 18, 2019