… 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 …
… 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 …
… lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today … These presentations will highlight the Company’s proprietary Axiomer RNA editing platform and pipeline … ADAR RNA Editing Platform - Editing oligonucleotides optimization for therapeutic use Presenter: Peter A. Beal, …
… 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 …
… Study of Homozygous F508del Cystic Fibrosis Patients Topline data to be presented today at NACFC LEIDEN, the … QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a … orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 …