… 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 …
… 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 …
… PQ-110-001 Phase 1/2 Trial PQ-110-001 is a first-in-human open-label trial that enrolled 5 children (age 6 - 17 years) … and 6 adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients … of visual function and retinal structure through ophthalmic endpoints, such as visual acuity (BCVA), mobility …
… 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
ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer™ RNA Editing OligonucleotidesPreclinical proof of concept for the Company’
… le Professeur Samuel G. Jacobson, M.D., Ph.D., qui est l’ophtalmologiste qui s’occupe de quatre des patients inclus … » devrait être lancée au cours du premier semestre 2019. À propos de l’amaurose congénitale de Leber 10 L’amaurose … occidental souffrent de LCA10 à cause de cette mutation. À propos du QR-110 Le QR-110 est un premier oligonucléotide …