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Wave Life Sciences-2026-JPM.pdf

上传人: 明**** 编号:1069802 2026-01-17 35页 2.79MB

1、J.P.Morgan Healthcare ConferenceJanuary 13,2026Wave Life Sciences2Forward-looking statementsThis document contains forward-looking statements.All statements other than statements of historical facts contained in this document,including statements regarding possible or assumed future results of opera

2、tions,preclinical and clinical studies,business strategies,research and development plans,collaborations and partnerships,regulatory activities and timing thereof,competitive position,potential growth opportunities,use of proceeds and the effects of competition are forward-looking statements.These s

3、tatements involve known and unknown risks,uncertainties and other important factors that may cause the actual results,performance or achievements of Wave Life Sciences Ltd.(the“Company”)to be materially different from any future results,performance orachievements expressed or implied by the forward-

4、looking statements.In some cases,you can identify forward-looking statements by terms such as“may,”“will,”“should,”“expect,”“plan,”“aim,”“anticipate,”“could,”“intend,”“target,”“project,”“contemplate,”“believe,”“estimate,”“predict,”“potential”or“continue”or the negative of these terms or other simila

5、r expressions.The forward-looking statements in this presentation are only predictions.The Company has based these forward-looking statements largely on its current expectations and projections about future events and financial trends that it believes may affect the Companys business,financial condi

6、tion and results of operations.These forward-looking statements speak only as of the date of this presentation and are subject to a number of risks,uncertainties and assumptions,including those listed under Risk Factors in the Companys Form 10-K and other filings with the SEC,some of which cannot be

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1. 公司定位与资金:Wave Life Sciences致力于RNA药物开发,拥有约6.02亿美元现金,预计资金可支撑至2028年第三季度。 2. 核心技术:拥有差异化的RNA药物平台和专有化学技术(SpiNA和AIMer),涵盖RNAi、RNA编辑及双功能模态。 3. 关键管线进展: - **WVE-007(肥胖)**:INHBE GalNAc-siRNA,单次240mg剂量3个月可减少9.4%内脏脂肪,同时保留肌肉,支持1-2次/年给药。 - **WVE-006(AATD)**:RNA编辑药物,400mg剂量使野生型M-AAT达64%,恢复生理性AAT生产。 - **WVE-008(肝病)**:PNPLA3 AIMer,针对全球900万I148M纯合子患者,计划2026年提交CTA。 4. 战略重点:2026年加速WVE-007的IIa期试验及RNA编辑管线推进,提交WVE-N531(DMD)的NDA。
肥胖新疗法? RNA编辑突破? 肝病有希望?
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