… are no therapies commercially available or in clinical development for this disease. QR-110 has received fast track … granted orphan drug designation in the United States and European Union. Interim safety and efficacy trial results … that the first patient has been dosed in the Phase 1/2 open-label trial assessing the safety, tolerability, …
… lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today … models. “The progress the field and ProQR are making in optimizing ADAR for therapeutic use is exciting,” said Peter … income from partnerships, including the potential Lilly opt-in fee of $50 M for 5 additional targets, milestone …
Access based on positive interim analysis of clinical data as well as preclinical data to date PRIME designation provides a pathway for frequent and early interactions with the EMA aimed at supporting accelerated evaluation and approval
… Collaboration New agreement supports the discovery and development of additional assets directed toward high … originally announced in September 2021, applied ProQR’s proprietary Axiomer® RNA editing platform to target disorders … additional targets, along with an option for Lilly to opt in for more,” said Daniel A. de Boer, founder and CEO of …
… two abstracts on programs for Fuchs endothelial corneal dystrophy and Stargardt’s disease at the ARVO annual meeting. … patients have been enrolled in PQ-110-001, a Phase 1/2 open-label trial assessing the safety, tolerability, … treatment to LCA 10 patients that have no other treatment options,” said Daniel A. de Boer, Chief Executive Officer of …
Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence
