Usher syndrome candidate designated orphan drug

An investigational drug from ProQR for the treatment of retinitis pigmentosa associated with Usher syndrome has received orphan drug designation from the FDA and the European Medicines Agency, according to a company press release.

QRX-411 targets the pseudo-exon 40 mutation in the USH2A gene and has shown promising preclinical data in mRNA restoration, the release said.

In addition to QRX-411, ProQR has one other compound for treatment of visual loss associated with Usher syndrome under study preclinically, as well as two other compounds in preclinical programs, one for treatment of Stargardt’s disease and one for Fuchs’ endothelial corneal dystrophy, according to the release. One drug aimed at treating Leber’s congenital amaurosis type 10 is in early clinical trial.

“The severe genetic retinal diseases we are targeting do not have any available therapies, especially disease modifying therapies focused on restoring vision or impeding progression of the disease,” CEO of ProQR Daniel A. de Boer said in the release. “We believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases.”

An investigational drug from ProQR for the treatment of retinitis pigmentosa associated with Usher syndrome has received orphan drug designation from the FDA and the European Medicines Agency, according to a company press release.

QRX-411 targets the pseudo-exon 40 mutation in the USH2A gene and has shown promising preclinical data in mRNA restoration, the release said.

In addition to QRX-411, ProQR has one other compound for treatment of visual loss associated with Usher syndrome under study preclinically, as well as two other compounds in preclinical programs, one for treatment of Stargardt’s disease and one for Fuchs’ endothelial corneal dystrophy, according to the release. One drug aimed at treating Leber’s congenital amaurosis type 10 is in early clinical trial.

“The severe genetic retinal diseases we are targeting do not have any available therapies, especially disease modifying therapies focused on restoring vision or impeding progression of the disease,” CEO of ProQR Daniel A. de Boer said in the release. “We believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases.”