Retinal Gene Therapy Program
This meeting will bring together as many of the world’s leaders in gene therapy as possible in a small congenial setting. The goals are to discuss the current status of gene therapy and how to move forward to progress the field, considering that initial successes will lead to larger scale trials. The meeting will be divided into general topic areas, with the goal to maximize discussion after initial presentations. It is our goal that all in attendance will be active discussants.
Friday May 10
2:00-4:00 p.m. Current TrialsModerator: Richard Weleber, M.D.
Stargardt's
·StarGen: Isabelle Audo, David Wilson (2:00-2:15)
·ProgStar: Hendrik Scholl (2:15-2:30)
Usher's
·UshStat: Richard Weleber (2:30-2:45)
Age-Related Macular Degeneration
·Retinostat: Andy Lauer (2:45-3:00)
Leber's Congenital Amaurosis: Jean Bennett or Tim Stout (3:00-3:15)
FDA Regulatory Issues: Steve Rose (3:15-3:30)
Discussion Panel and Audience (3:30-4:00)
4:00-5:00 p.m. Preclinical Trials Moderator: Martha Neuringer Ph.D.
·X-linked Retinoschisis: Paul Sieving (4:00-4:15)
·Discussion Audience (4:15-4:30)
·Achromatopsia: Bill Hauswirth (4:30-4:45)
·Discussion Audience (4:45-5:00)
5:00-6:00 p.m. Biotechnology Moderator: Steve Rose, Ph.D.
·Oxford BioMedica: Stuart Naylor (5:00-5:10)
·AGTC: Jeff Chulay (5:10-5:20)
·Sanofi: Soraya Bekkali (5:20-5:30)
·Discussion Panel and Audience (5:30-6:00)
Gala Dinner (featuring Oregon Pinot Noir and Northwest Cuisine)
Keynote Address: "Challenges in developing new therapies for inherited retinal disease"
Tony Moore
Saturday May 11
7:30-10:00 a.m. Endpoints for Gene Therapy Trials Moderator: Mark Pennesi, M.D., Ph.D.
·Optical Coherence Tomography: Don Hood (7:30-7:45)
·Adaptive Optics: Jacque Duncan (7:50-8:05)
·Autofluorescence: Thiran Jaysundra (8:10-8:25)
·Visual Fields: Richard Weleber (8:30-8:50)
·Microperimetry: Art Cideciyan (8:55-9:10)
·Electrorentinograms: David Birch (9:15-9:30)
·Visual Function Testing: Rohit Varma (9:35-9:50)
·15 minute talks followed by 5 minutes of discussion
10:00-12:30 p.m. Vectors and Delivery Moderator: Tim Stout, M.D., Ph.D.
·AAV: Bill Hauswirth (10:00-10:15)
·Discussion: Audience (10:15-10:30)
·Lentivirus: Kyri Mitrophanous (10:30-10:45)
·Discussion: Audience (10:45-11:00)
·Surgical Considerations: Al Maguire (11:00-11:15)
·Discussion: Audience (11:15-11:30)
·Optogenetics: John Flannery (11:30-11:45)
·Discussion: Audience (11:45-12:00)
·Nanoparticles: Muna Naash (12:00-12:15)
·Discussion: Audience (12:15-12:30)
12:30-2:00 p.m. Lunch and Picture
2:00-4:30 p.m. Issues for Multicenter EfficacyTrials Moderator: David Wilson, M.D.
·Genotype Phenotype Variation: Ed Stone (2:00-2:15)
·Discussion: Audience (2:15-2:30)
·NGS: Implications for Diagnostic Screening: John Chiang (2:30-2:45)
·Discussion: Audience (2:45-3:00)
·Gene Therapy in Brazil and China: Juliana Sallum, Helen Sui, Ming Qi (3:00-3:30)
·Discussion: Audience (3:30-3:45)
·Retinal Gene Therapy Study Group: David Wilson (3:45-4:00)
·Discussion: Audience (4:00-4:30)
