… 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 …
… 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 …
… with no serious adverse events QR-421a is the second ophthalmology program where clinical activity was predicted … benefit was observed across multiple relevant measures appropriate to the severity of the patient’s disease, … treated eye, <5 dB.steradian change in untreated eye], and optical coherence tomography (OCT) assessment of …
… concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a … endpoints, the dose, dosing interval, and patient population for the Phase 2/3 pivotal trials,” said Aniz … were categorized into “advanced” and “early-moderate” populations based on baseline visual acuity. In advanced …