Rare Daily Staff

The Foundation Fighting Blindness and ProQR have entered into a partnership that will provide the company up to $7.5 million to develop QR-421a, an experimental treatment for Usher syndrome type 2A, a genetic disease that causes hearing loss and progressive vision loss.

There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A. QR-421a is being developed to target mutations in exon 13 of the USH2A gene, which drive Usher syndrome type 2A.

QR-421a is a first-in-class RNA oligonucleotide. QR-421a is designed to modify the RNA such that functional usherin protein is produced in the retina with the goal of stopping the progression of the disease and potentially gaining peripheral vision.

Under the agreement, the Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic. QR-421a received orphan drug designation from the FDA.

Foundation Fighting Blindness’ Clinical Research Institute has also launched a natural history study in 120 people with USH2A mutations. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.

“Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin Yerxa, CEO of Foundation Fighting Blindness. “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.”

QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials.  The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and pediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene.  This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE-40 mutation, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.

February 13, 2018

Photo: Benjamin Yerxa, CEO of Foundation Fighting Blindness