… and Director of the NIH-funded UC Davis Chemical Biology Graduate Program. For over 25 years, work in the Beal laboratory has advanced understanding of the structures and mechanism of action for the ADAR enzymes responsible for adenosine to inosine RNA editing …
… partnership is another validation of our leadership in ADAR-mediated RNA editing, our robust IP estate, and the … 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 …
… to execute on our strategy.” Recent Progress Continue to advance AX-0810 for patients suffering from cholestatic … cardiovascular risk. ProQR plans to present and publish additional preclinical data from these two pipeline programs … Chardan Genetic Medicines Conference panel discussion on “ ADAR RNA Editing: Unlocking New Therapeutic Opportunities ”. …
… initial pipeline targets expected in late 2022/early 2023 Additional pivotal trial recommended by EMA for sepofarsen … (MAA). In light of this feedback and in order to continue advancement of the portfolio of ophthalmic product … platform. ProQR has established a leading IP estate in the ADAR-editing space related to its RNA-editing technology and …
… 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 …
… advancements anticipated through increased knowledge of ADAR biology, selection of models for prediction of Axiomer … 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 …
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3
… effective strategies to increase RNA editing yields of ADARs using chemical modifications and oligonucleotide design. Additionally highlighted was the potential of the Axiomer … molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer ® EONs are …
… 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 …