… ProQR spins out all Dystrophic Epidermolysis Bullosa activities into newly formed … EBRP to apply our experience in DEB drug development to develop transformational therapies for DEB patients,” said … a loss of the anchoring fibrils resulting in fragile skin. People with DEB live with constant pain and have a high risk …
… cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced details … Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, will give an oral … QR-010 is designed to be self-administered via an optimized eFlow ® Nebulizer (PARI Pharma GmbH). eFlow ® is a …
… ProQR has granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders and … to future milestones and royalties from the products developed. Amylon will initially focus on developing a RNA-based therapy for a rare genetic disease …
… in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, … 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients … expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and …
… are no therapies commercially available The Phase 1/2 open-label trial will assess the safety, tolerability, … 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients … expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and …
… will be instrumental in advancing the company’s novel and proprietary Axiomer ® RNA editing technology. The board now … rare diseases including Leber's congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis, today … Ph.D., has more than two decades of experience in RNA drug development from discovery through drug registration, both in …
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will p
… for AX-0810 AX-0810 is an editing oligonucleotide (EON) development program targeting the SLC10A1 RNA using the … biomarker bile acids 72 hours after treatment. Further optimizations for EONs targeting NTCP have enabled … models and showed translatability across models. ProQR is developing its AX-0810 program targeting NTCP for Cholestatic …