Drug intelligence / Profile preview

QR-111

Development stage: Unknown
Lead developer: Ultragenyx Pharmaceutical
Modality: Gene Therapies

Overview

QR-111 is an investigational drug that was under development by Fovea Pharmaceuticals, a company later acquired by Sanofi-Aventis. It is being investigated for the treatment of Leber congenital amaurosis, a rare inherited eye disease that causes severe vision loss. The drug is currently in Phase II clinical trials and is believed to be a gene therapy, aiming to correct genetic defects underlying the condition.

Targets

SGSH (N-sulphoglucosamine sulphohydrolase)

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