Gyrate Atrophy Scientific Symposium
Gyrate Atrophy Scientific Symposium
Research Summary
On February 1-2, 2020 a small group of highly esteemed researchers met to discuss the state of research on gyrate atrophy. This meeting was hosted by Drs. David Valle and Mandeep Singh of Johns Hopkins University. Dr. Valle led the pioneering research on the use of a diet restricted in arginine as a therapy for gyrate atrophy and developed the first mouse models of the disease. This is the first time a meeting focused specifically on gyrate atrophy (GA) has ever been held! Below you will find the program, a photo and some of the highlights of the meeting. My overall impression is that there are a number of very promising research efforts for GA from retinal epithelium transplants to gene therapy to stem cell approaches. Patients and families have a vital role to play in connecting researchers. It was through our connections with other families, that we were able to invite researchers from Australia, the Netherlands and the United Kingdom who, unknown to the US researchers, are working on GA . The Netherlands group just launched their website here which will include a strong focus on gyrate atrophy. See the agenda and a photo below.
This meeting resulted in two leading research teams deciding to begin some research on GA! We hope to support both teams to develop a “proof of concept” that could be used to unlock additional funding.
Agenda:
Location:
Scientific Symposium on Gyrate Atrophy
Mt. Washington Conference Center
(located at Johns Hopkins @ Mt. Washington)
5801 Smith Ave
Baltimore, MD 21209
Sat, Feb 1 – Conference Dinner including Industry/ VC
| 8:00 | Basic Concept | Speaker | Institution |
|---|---|---|---|
| 8.00 – 8.25 |
Gyrate atrophy overview |
David Valle | Johns Hopkins University |
| 8.25 – 8.50 |
Role of proline in the retina and retinal pigment epithelium | Jianhai Du | West Virginia University |
| 8.50 – 9.15 |
Ocular phenotype of gyrate atrophy | Stephen Tsang |
Columbia University |
| 9.15 – 9.35 |
Imaging endpoints for gyrate atrophy | Michael Ip |
University of California, Los Angeles |
| 9.35 – 10.00 |
JH First-in-Human initiative | Sashank Reddy |
Johns Hopkins Technology Ventures |
| 10:00 | Coffee break | ||
| 10:20 | Therapy development | ||
| 10.20 – 10.45 | Ocular drug development overview |
Benjamin Yerxa | Foundation Fighting Blindness |
| 10.45 – 11.10 | Therapy development experience: Leber congenital amaurosis | Jean Bennett | University of Pennsylvania |
| 11.10 – 11.35 |
Therapy development experience: Choroideremia | Alun Barnard | Oxford, UK |
| 11.35 – 12.00 |
Probiotic therapy | MK Nizam |
University of Tasmania, Australia |
| 12.00 – 12.25 |
Retinal pigment epithelium stem cell therapy | Kapil Bharti |
National Eye Institute, USA |
| 12.25 – 14.00 | Lunch roundtable – Group discussion on research priorities | All | |
| Ajourn |
Discussants:
King Wai Yau (JHU)
Anand Swaroop (NEI) – photoreceptor cells YES
Jefferson Doyle (Wilmer)
Don Zack (Wilmer)
Jim Phang (NIH) – Proline metabolism YES
Malay & Karabi Acharya
Jim Handa (Wilmer) Mandeep Singh (Wilmer)
Spencer Lowndes
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