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 ...

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 September 30, 2023 and December 31, 2022 1 Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the Three and Nine Month Periods ended September 30, 2023 and 2022 2 Unaudited Condensed Consolidated Statement of Changes in Equity for the Nine Month Periods Ended September 30, 20 ...

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, 2023 and December 31, 2022 1 Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the Three and Six Month Periods ended June 30, 2023 and 2022 1 Unaudited Condensed Consolidated Statement of Changes in Equity for the Six Month Periods Ended June 30, 2023 and 2022 3 Una ...

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, 2023 and December 31, 2022 1 Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the Three Month Periods ended March 31, 2023 and 2022 2 Unaudited Condensed Consolidated Statement of Changes in Equity for the Three Month Periods Ended March 31, 2023 and 2022 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, 2022 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 Initial Pipeline Targets and Highlights Axiomer® RNA Editing Platform Technology at R&D Event ● Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025 ● Axiomer activity demonstrated across multiple preclinical in vitro, organoid, and in vivo models; up to 50% editing observed in rodent and non-human primates ● Beyond correction, b ...