ProQR Gives Preclinical Proof Of Concept Data For AX-0810 Axiomer RNA Editing Program Targeting NTCP For Cholestatic Diseases
ProQR Gives Preclinical Proof Of Concept Data For AX-0810 Axiomer RNA Editing Program Targeting NTCP For Cholestatic Diseases
- ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer RNA Editing Oligonucleotides
- Preclinical proof of concept for the Company's AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT Annual Meeting
- Management webinar May 9, 2024 at 8:00 am EDT
- ProQR 科學家首次在 ADAR RNA 編輯領域發表報告 在活體中 使用 Axiomer RNA 編輯寡核苷酸證明靶點參與（RNA 編輯）導致 NHP 中的生物標誌物發生有意義的變化
- 在 ASGCT 年會上發佈的公司針對膽汁淤積性疾病的 NTCP 的 AX-0810 計劃的臨床前概念驗證
- 管理網絡研討會 2024 年 5 月 9 日美國東部時間上午 8:00
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced new preclinical data for its proprietary Axiomer RNA editing technology platform, including the first preclinical proof of concept data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP. The data are being presented at a poster session today 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5月8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）今天公佈了其專有Axiomer RNA編輯技術平台的新臨床前數據，包括針對NTCP的膽汁淤積性疾病 AX-0810 管道項目的首份臨床前概念驗證數據。這些數據將在今天於2024年5月7日至11日在馬里蘭州巴爾的摩舉行的美國基因與細胞療法學會（ASGCT）年會的海報發佈會上公佈。
"Our Axiomer ADAR RNA editing data at ASGCT show the exciting potential of our technology and for the first time in the field we report proof of target engagement leading to meaningful changes in biomarkers in NHPs," said Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR. "In our AX-0810 program targeting NTCP for cholestatic diseases, we have demonstrated preclinical proof of concept showing that Axiomer RNA editing oligonucleotides can have a meaningful effect on levels of serum biomarker bile acid. We believe this is a significant derisking step as we approach the clinic as our NHP studies closely resemble the design of the first in human trial that will focus on target engagement and biomarkers. We look forward to sharing translational data for our AX-0810 program and to announcing our clinical program candidate later this year, as we progress to enter the clinic in late 2024/early 2025."
ProQR首席科學官兼聯合創始人傑拉德·普拉滕堡表示：“我們在ASGCT的Axiomer ADAR RNA編輯數據顯示了我們技術的驚人潛力，我們首次在該領域報告了靶向參與導致NHP生物標誌物發生有意義變化的證據。”“在我們針對膽汁淤積性疾病的 NTCP 的 AX-0810 項目中，我們已經證明了臨床前概念驗證，表明 Axiomer RNA 編輯寡核苷酸可以對血清生物標誌物膽汁酸水平產生有意義的影響。我們認爲，在我們進入臨床過程中，這是一個重要的降低風險的步驟，因爲我們的NHP研究與第一項以靶標參與和生物標誌物爲重點的人體試驗的設計非常相似。我們期待分享我們的 AX-0810 項目的轉化數據，並期待在今年晚些時候宣佈我們的臨床候選項目，因爲我們將在 2024 年底/2025 年初進入臨床。”
譯文內容由第三人軟體翻譯。