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 科学家首次在 ADAR RNA 编辑领域发表报告 在活体中 使用 Axiomer RNA 编辑寡核苷酸证明靶点参与（RNA 编辑）导致 NHP 中的生物标志物发生有意义的变化
- 在 ASGCT 年会上发布的公司针对胆汁淤积性疾病的 NTCP 的 AX-0810 计划的临床前概念验证
- 管理网络研讨会 2024 年 5 月 9 日美国东部时间上午 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.
荷兰莱顿和马萨诸塞州剑桥，2024年5月8日（GLOBE NEWSWIRE）——致力于通过变革性RNA疗法改变生活的公司ProQR Therapeutics NV（纳斯达克股票代码：PRQR）（ProQR）今天公布了其专有Axiomer RNA编辑技术平台的新临床前数据，包括针对NTCP的胆汁淤积性疾病 AX-0810 管道项目的首份临床前概念验证数据。这些数据将在今天于2024年5月7日至11日在马里兰州巴尔的摩举行的美国基因与细胞疗法学会（ASGCT）年会的海报发布会上公布。
"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首席科学官兼联合创始人杰拉德·普拉滕堡表示：“我们在ASGCT的Axiomer ADAR RNA编辑数据显示了我们技术的惊人潜力，我们首次在该领域报告了靶向参与导致NHP生物标志物发生有意义变化的证据。”“在我们针对胆汁淤积性疾病的 NTCP 的 AX-0810 项目中，我们已经证明了临床前概念验证，表明 Axiomer RNA 编辑寡核苷酸可以对血清生物标志物胆汁酸水平产生有意义的影响。我们认为，在我们进入临床过程中，这是一个重要的降低风险的步骤，因为我们的NHP研究与第一项以靶标参与和生物标志物为重点的人体试验的设计非常相似。我们期待分享我们的 AX-0810 项目的转化数据，并期待在今年晚些时候宣布我们的临床候选项目，因为我们将在 2024 年底/2025 年初进入临床。”