ProQR Highlights Upcoming Presentations on Axiomer RNA Editing at ASGCT 27th Annual Meeting
ProQR Highlights Upcoming Presentations on Axiomer RNA Editing at ASGCT 27th Annual Meeting
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.
荷蘭萊頓和馬薩諸塞州劍橋，2024 年 4 月 22 日（GLOBE NEWSWIRE）——致力於通過變革性 RNA 療法改變生活的公司 ProQR Therapeutics NV（納斯達克股票代碼：PRQR）（ProQR）今天宣佈，它將爲其專有的 Axiomer RNA 編輯技術平台提供新的臨床前數據，包括美國基因與學會針對膽汁淤積性疾病 AX-0810 管道項目的首批臨床前數據細胞療法（ASGCT）年會，2024年5月7日至11日，在馬里蘭州巴爾的摩舉行。
"The in vitro and in vivo data that we will be presenting at ASGCT further build upon our experience with Axiomer and highlight the significant potential we see with the platform, including now preclinical proof of concept for cholestatic diseases targeting NTCP," said Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR. "We continue to be impressed by the robust editing efficiency of Axiomer editing oligonucleotides and look forward to leveraging these data as we advance AX-0810 toward the clinic."
ProQR首席科學官兼聯合創始人傑拉德·普拉滕伯格表示：“我們將在ASGCT上提供的體外和體內數據進一步建立在我們使用Axiomer的經驗基礎上，突顯了我們在該平台上看到的巨大潛力，包括現在針對NTCP的膽汁淤積性疾病的臨床前概念驗證。”“Axiomer 編輯寡核苷酸的強大編輯效率仍然給我們留下深刻的印象，並期待在我們將 AX-0810 推向臨床的過程中利用這些數據。”
Wednesday, May 8, 2024 poster presentations:
2024 年 5 月 8 日星期三海報發佈會：
P-705: "Robust and Durable RNA Editing In Vivo with Axiomer Editing Oligonucleotides in Non-Human Primates"
P-705：“在非人類靈長類動物中使用 Axiomer 編輯寡核苷酸在體內進行穩健耐用的 RNA 編輯”
Building on its robust platform data, the Company will present additional non-human primate (NHP) data including with ACTB and NTCP and initial in vitro and in vivo preclinical proof of concept for its AX-0810 program targeting NTCP for cholestatic diseases, including hepatic editing in NHP with strong correlating impact on disease-relevant biomarkers.
基於其強大的平台數據，該公司將提供其他非人類靈長類動物（NHP）數據，包括ACTB和NTCP數據，以及針對膽汁淤積性疾病的NTCP的 AX-0810 計劃的初步體外和體內臨床前概念驗證，包括對疾病相關生物標誌物具有強烈相關影響的NHP中的肝臟編輯。
P-726, partner poster presentation by Eli Lilly: "Complex Metabolism and Prolonged PK/PD of a GalNAc-Conjugated Editing Oligonucleotide (EON) in Mice"
P-726，禮來公司的合作伙伴海報展示：“Galnac偶聯編輯寡核苷酸（EON）在小鼠體內的複雜代謝和延長 PK/PD”
Following presentation at ASGCT, the posters will be available via ProQR's website in the Presentations and Publications section.
在ASGCT上發表後，海報將通過ProQR網站的 “演示和出版物” 部分公佈。
About Axiomer
關於 Axiomer
ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer "Editing Oligonucleotides", or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using 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 – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
ProQR正在開創一種名爲Axiomer的下一代RNA鹼基編輯技術，該技術有可能爲各種類型的疾病生產出一類新藥物。Axiomer “編輯寡核苷酸”（EONs）使用存在於人體細胞中稱爲ADAR（作用於RNA的腺苷脫氨酶）的分子機制，以高度特異和有針對性的方式介導RNA的單核苷酸變化。Axiomer EONs旨在招募和引導內源表達的ADAR，將RNA中的腺苷（A）轉化爲肌苷（I）——肌苷被翻譯成鳥苷（G）——將具有致病突變的RNA校正爲正常（野生型）RNA，調節蛋白質表達，或改變蛋白質，使其具有有助於預防或的新功能治療疾病。
譯文內容由第三人軟體翻譯。