The Eye on the Future Forum (EFF) is a platform to exchange knowledge and engage in dialogue on innovative clinical development approaches for genetic ophthalmic diseases, with a strong focus on inherited retinal diseases (IRD). We aim to learn what can be improved in the clinical stages of IRD drug development in order to make the patient and site experience better.
EFF brings together key leaders from around the world in addition to patients and advocacy groups to share their insights in the IRD field. We aim to understand each unique perspective so we can collaborate together to change the future of IRD treatment.
ProQR Therapeutics, a biopharmaceutical company which develops RNA medicines for severe genetic rare diseases. ProQR is based in Leiden, The Netherlands, and Cambridge, MA, USA.
Established in 2012 with a close-to-home mission of helping patients who suffer from rare genetic diseases, ProQR is currently working on several drugs, of which the main are:
- Sepofarsen for LCA10 (due to the p.Cys998X mutation in the CEP290 gene)
- QR-421a for Usher syndrome type 2 (mutations in exon 13 of the USH2A gene)
- QR-1123 for adRP (P23H mutation in the RHO gene)
For more information about ProQR, visit www.proqr.com.
The exciting fall EFF program is happening on 23 September 2020.