Appendix 1 of
Hauswirth, Mol Vis 4:11, 1998.
Appendix 1. Agenda for 1998 Retinal Gene Therapy Workshop
Retinal Gene Therapy Workshop
Emory University Conference Center
February 21 and 22, 1998
Organized by William W. Hauswirth and Roderick R. McInnes
Sponsored by the Foundation Fighting Blindness
Saturday, February 21
8:15 PM - Gene Therapy for Inherited Retinal Degenerations: Are you Ready?
Phil Noguchi (Food and Drug Administration)
Sunday, February 22
08:00 AM - Welcome
Jerry Chader (Foundation Fighting Blindness)
08:05 AM - A General Introduction to Inherited Retinal Degenerations
Sam Jacobson - Clinical and Pathological Features
Ed Stone - Genetic Basis of Inherited Retinal Disease
08:35 AM - DNA Viral Vectors
Primary Discussant: Jean Bennett
Secondary Discussants: Battacharya, Gage, Kumar-Singh, Li, Muzyczka
Some Questions: What aspects of DNA viral vectors currently look most favorable in the retina, and why? What are the major problems with these vectors, and what are the most promising approaches for overcoming these problems?
09:10 AM - RNA Viral and Nonviral Gene Delivery
Primary Discussant: Rusty Gage
Secondary Discussants: Bennett, Lewin, Li, Muzyczka, Saperstein
Some Questions: What aspects of RNA viral vectors and nonviral gene delivery currently look most favorable in the retina, and why? What are the major problems with these systems, and what are the most promising approaches for overcoming these problems? How important is regulated expression and how might it be best achieved?
09:50 AM - Alternative Gene Therapies
Primary Discussant: John Flannery
Secondary Discussants: Goff, LaVail, Li, Nickerson, Wong, Zack
Some Questions: Are there useful approaches for more general retinal gene therapy in addition to gene replacement or inactivation of specific dominant negative alleles? Do anti-VEGF, anti-apoptotic factor, neurotrophin, growth factor or cytokine genes offer viable alternative strategies?
10:25 AM - Break
10:40 AM - Animal Models
Primary Discussant: Gus Aguirre
Secondary Discussants: Baehr, Farber, Goff, LaVail, Travis, Valle, Wong
Some Questions: What animal models of retinal diseases are most relevant to clinical disease and which are (or will be) the most convincing as preclinical models. Are additional animal models needed?
11:15 AM - Mechanisms of Retinal Degeneration
Primary Discussant: Don Zack
Secondary Discussants: Aguirre, Baehr, Farber, Flannery, Nickerson, Travis, Wong
Some Questions: What information about general retinal biology is needed to better understand retinal degeneration and cell death? How might this data allow more effective gene therapy strategies?
11:50 PM - Target Human Retinal Diseases
Primary Discussant: Ed Stone
Secondary Discussants: Battacharya, Humphries, Lewis, Sieving, Zack
Some Questions: What human retinal diseases should be first considered for gene therapy, and why? What retinal diseases are (or will be) most amenable to gene therapy approaches? Which need further attention, and at what level?
12:25 PM - Lunch
01:25 PM - Proof of Principle
Primary Discussant: Pete Humphries
Secondary Discussants: Bennett, Flannery, Jacobson, Laties, Sieving
Some Questions: What research milestones must be reached prior to a clinical trial? Are additional experimental results needed? What sorts of retinal injection-related or therapeutic gene-related pathology might be expected in humans? Can this be eliminated or minimized?
02:00 PM - Patient Populations and Clinical Criteria
Primary Discussant: Sam Jacobson
Secondary Discussants: Csaky, Laties, Lewis, Saperstein, Sieving, Stone
Some Questions: What patient populations exist that could be used for Phase I and II retinal gene therapy trials. What ethical and logistical problems can be anticipated? What clinical assays exist for gauging the effectiveness of a retinal gene therapy trial? How could they be employed effectively in a Phase I or II trial? What problems exist?
02:45 PM - Other Human Trials
Primary Discussant: Ric Boucher
Secondary Discussants: Csaky, LaVail, Muzyczka, Noguchi, Verma
Some Questions: What lessons can be learned from other human gene therapy trials?
03:20 PM - Role of Industry
Primary Discussant: Marty Glick
Secondary Discussants: Brazzell, Gonda
Some Questions: What is the current role of industry in retinal gene therapy? What should be done to enhance commercial interest and to increase academic-commercial interactions?
03:55 PM - Break
04:10 PM - Alternatives to In Vivo Gene Therapy
Primary Discussant: Ray Lund
Secondary Discussants: Chader, Csaky, Laties, LaVail, Valle
Some Questions: What is the status of research on transplantation alternatives to retinal gene therapy? Is ex vivo therapy a viable alternative? Are the risk/benefit ratios for alternative therapies predictable and, if so, how do they impact on an overall strategy for treating retinal disease vs. gene therapy?
04:45 PM - Workshop Overview
Primary Discussant: Inder Verma
Secondary Discussants: All participants
Some Questions: Is the field on sufficiently firm scientific footing to bring retinal gene therapy to the clinic in the near future?
05:25 PM - Discussion
06:00 PM - Workshop Adjourns