On June 6-7, the Choroideremia Research Foundation (CRF) hosted its 2019 International Choroideremia Symposium in Philadelphia, Pennsylvania. With the support of the Center for Advanced Retinal and Ophthalmic Therapeutics at the University of Pennsylvania, CRF developed an agenda geared toward answering key questions raised by ongoing research, and developing consensus opinion on paths forward for the organization and its community. The Symposium was attended by 20 international experts in CHM from 5 countries in addition to representatives from biotech industry and the patient community to bring multiple perspectives toward the singular goal of developing treatments to end blindness.
The Symposium was held using an open moderated discussion format, engaging all in attendance to help discuss topics and answer questions using the collective expertise of those in attendance. The first day focused on pre-clinical topics which could provide better understanding of disease mechanisms and, in corollary, additional avenues toward the development of new therapies. Much discussion revolved around the underlying mechanisms of retinal cell dysfunction and death in CHM, which like many retinal diseases requires additional investigations. The group discussed a series of potential therapeutic approaches including the use of stem cells, neuroprotective agents, optogenetics, and reversal of the normal aging process in cells, and how research on these approaches could be supported. Discussions on day 2 of the Symposium reviewed ongoing natural history studies and clinical trials, bringing together leaders in these respective areas to identify lessons learned from these trials and potential next steps. Lastly, a robust discussion was held to begin developing a classification system to stage the progression of CHM in patients, as well as the ideal outcome measures to be used in future clinical trials.
“The 2019 International Choroideremia Symposium was a tremendous success,” says Christopher Moen, Chief Medical Officer of CRF. “The topics reviewed at the Symposium identified several opportunities for research projects which could drive CHM toward future therapies that could work alongside gene replacement therapy. We’re excited at the future of CHM research and fortunate to have such tremendous support from our medical and scientific community.”
About the Choroideremia Research Foundation
CRF was founded in 2000 as a fundraising and patient advocacy organization to stimulate research on CHM. Since its inception, the CRF has provided over $2 million in research awards and is the largest financial supporter of CHM research worldwide. Research funded by the CRF has led to the development of a CHM animal model, the pre-clinical production of gene therapy vectors currently in clinical trials, and the CRF Biobank which stores tissue and stem cell samples donated by CHM patients.