The FDA has cleared an investigational new drug application for QR-1123, an investigational oligonucleotide treatment candidate for vision loss with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, according to a press release from ProQR Therapeutics.
The company plans to begin a first-in-human phase 1/2 trial of QR-1123 by the end of this year, with a single-dose escalation open-label arm and a multiple-dose double-masked arm, the release said. Safety, tolerability, pharmacokinetics and efficacy will be evaluated.
“We are pleased to have an open IND
Uncategorized