… upfront payment and equity investment ProQR is eligible to additionally receive up to approximately $ 1. 25 billion in … oligonucleotides, or EONs, designed to recruit endogenous ADAR enzymes (Adenosine Deaminases Acting on RNA) to a selected target …
… been enrolled in a Phase 1/2 clinical trial of QR-110 in adults and children with LCA 10. Interim data are expected in … the vision loss associated with Usher syndrome type 2A. ADAR A-to-I RNA editing expert Dr. Peter A. Beal was appointed to ProQR’s scientific advisory board. LEIDEN, the Netherlands, May 09, 2018 (GLOBE …
Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and miceFunctional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favora
… ProQR Announces Additional Sepofarsen Illuminate Trial Analyses and Provides … the Company’s runway and deliver on its commitment to advance RNA therapies for diseases with high unmet need. … in 2014, it has established a leading IP estate in the ADAR editing space, a first industry partnership, and with …
Divestment of sepofarsen and ultevursen ophthalmic programs supports ProQR’s strategic focus on Axiomer® RNA editing platform technology and continued advancement of pipeline
€129 M cash and cash equivalents as of June 30, 2023 providing runway into mid-2026
… molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer ® EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA …
… platform uses the body’s own editing machinery called ADAR to make single nucleotide edits in RNA,” said Gerard … as targets in our collaboration with Eli Lilly. With broad applicability, and a leading patent portfolio in the ADAR editing space, Axiomer ® …
AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024$4.5 million in milestones
… for directing site-specific A-to-I editing by endogenous ADAR enzymes Presenter: Antti Aalto, Ph.D., senior … an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …