文章核心观点 - 公司完成了1800万普通股的公开发行,总募集资金6300万美元[1] - 公司还完成了对Eli Lilly的3,523,538股普通股的私募配售,募集资金约1230万美元[2] - 公司的RNA编辑技术平台Axiomer™可以实现单核苷酸的特定编辑,有望开发出新一代RNA疗法[5] 公司信息 - 公司名称为ProQR Therapeutics N.V.,总部位于荷兰莱顿和美国剑桥[1] - 公司专注于基于Axiomer™RNA编辑技术平台开发转化性RNA疗法[5] - 公司正在扩大基于该平台的管线[5] 融资信息 - 公司完成了1800万普通股的公开发行,总募集资金6300万美元[1] - 公司还完成了对Eli Lilly的3,523,538股普通股的私募配售,募集资金约1230万美元[2] - 公开发行由Evercore ISI、Cantor、Raymond James和Oppenheimer & Co.担任联席主承销商[2] - 私募配售不涉及任何承销折扣或佣金[2]

LEIDEN, Netherlands and CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (“ProQR”), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the pricing of its previously announced underwritten public offering of 18,000,000 ordinary shares (the “Offering”) at a public offering price of $3.50 per share, for total gross proceeds of $63.0 million, before deducting under ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (“ProQR”), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it has commenced an underwritten public offering of its ordinary shares (the “Offering”). All of the shares are being offered by ProQR. In addition, ProQR expects to grant the underwriters a 30-day option to purchase up to an ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 07, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present preclinical data for its proprietary Axiomer™ RNA editing technology platform and its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society, October 6-9, 2024, in Montreal, QC, Canada. Gerard Platenburg ...

Table of Contents Exhibit 99.1 PROQR THERAPEUTICS N.V. Index to Unaudited Condensed Consolidated Financial Statements PAGE | --- | --- | |----------------------------------------------------------------------------------------------------------|-------| | | | | Unaudited Condensed Consolidated Statement of Financial Position at June 30, 2024 and December 31, 2023 | | | Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the | | | Three and Six Month Periods ended ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., June 18, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will participate in the RNA Editing Summit, June 18-20, 2024, in Boston, Massachusetts. As part of the conference program, Gerard Platenburg, Chief Scientific Officer and Co-Founder, ProQR Therapeutics, will present on June 20, 2024 at 2:00pm ET: "Developing Axiomer RNA Editing Technology Towards ...

新产品和新技术研发 - 公司在ASGCT年会上展示了AX-0810项目针对NTCP的新的临床前概念验证数据[1] - 公司的Axiomer RNA编辑平台利用ADAR机制，通过编辑富含特定核苷酸的RNA来招募内源ADAR，实现RNA编辑[3] - 公司持有超过10项平台专利，保护使用寡核苷酸广泛招募诱导ADAR的技术[4] - 公司计划在今年下半年分享AX-1412项目的临床前概念和转化数据[16] - 公司预计在2024年底或2025年初将AX-0810进入临床试验[16] 合作和市场扩张 - 公司的策略包括内部开发管道项目和选择性合作伙伴关系，目前重点是AX-0810和AX-1412项目[6] - 公司与Eli Lilly合作在Axiomer平台上共同开发10个靶点，合作已带来1.25亿美元的前期付款[6] 未来展望 - 公司设计了一种转化策略，通过在健康志愿者中进行研究，以获得干净的数据集，适当的样本大小和快速执行[6] - 公司拥有足够的现金储备，可支持至2026年中期，有充足的机会从非稀释性来源延长现金流[17] 其他有价值的信息 - 优化后的EONs在体内编辑方面表现更好，预计将带来更高的编辑水平[21] - AX-0810的概念验证旨在展示RNA编辑，公司将在今年晚些时候宣布发展候选药物[24] - NTCP靶向治疗可以解决胆酸在肝细胞中的浓度问题，可能会导致临床意义的改善[29] - 25%的编辑水平可能足以产生有意义的临床效果[36] - 目前在非人灵长类动物模型中未发现异常安全信号[37]

Table of Contents Exhibit 99.1 PROQR THERAPEUTICS N.V. Index to Unaudited Condensed Consolidated Financial Statements PAGE Unaudited Condensed Consolidated Statement of Financial Position at March 31, 2024 and December 31, 2023 1 Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the Three Month Periods ended March 31, 2024 and 2023 2 Unaudited Condensed Consolidated Statement of Changes in Equity for the Three Month Periods Ended March 31, 2024 and 2023 3 Unaudi ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 20-F (Mark One) ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☐ SHELL COMPANY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ...

Exhibit 99.1 ProQR Announces Year End 2023 Operating and Financial Results • Preclinical and translational pipeline program data for AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 anticipated in 2024, building further momentum toward the clinic • Accelerated development of Axiomer™ for CNS applications via new research partnership with Rett Syndrome Research Trust • Completed divestment of sepofarsen and ultevursen ophthalmic programs to Laboratoires ...