亨廷顿病临床试验更新：2026年8月。
完整摘要 · 中文译文
在本期中，我们着重介绍亨廷顿病治疗管线中的几项重要进展。我们讨论了POINT-HD研究的启动及随后终止，这是RG6496（Hoffmann-La Roche）的首次人体I期研究，RG6496是一种等位基因选择性反义寡核苷酸，旨在选择性降低突变型亨廷顿蛋白，同时基本保留野生型亨廷顿蛋白的表达。我们还讨论了两项III期疗效试验的启动：评估votoplam（Novartis Pharmaceuticals）的INVEST-HD研究，以及评估pridopidine（Prilenia）的PRECISE-HD研究。此外，我们总结了该领域的最新进展，包括LQT-23（LoQus23）、SRP-1005（Sarepta Therapeutics）和HRN001（Harness Therapeutics）的临床前更新；SKY-0515（Skyhawk Therapeutics）的12个月中期临床结果；uniQure宣布AMT-130 I/II期研究的3年数据可能支持寻求加速批准的生物制品许可申请，以及Roche终止tominersen的II期GENERATION HD2研究。最后，我们提供了亨廷顿病已注册和正在进行中的临床试验的最新清单。 疗效试验：评估 votoplam（诺华制药）的 INVEST-HD，以及评估 pridopidine（Prilenia）的 PRECISE-HD。此外，我们总结了该领域的最新进展，包括 LQT-23（LoQus23）、SRP-1005（Sarepta Therapeutics）和 HRN001（Harness Therapeutics）的临床前更新；SKY-0515（Skyhawk Therapeutics）的 12 个月中期临床结果；uniQure 宣布的 AMT-130 I/II 期研究 3 年数据可能支持一项寻求加速批准的生物制品许可申请，以及罗氏终止 tominersen 的 II 期 GENERATION HD2 研究。最后，我们提供了亨廷顿病已注册和正在进行中的临床试验的最新列表。
完整摘要 · 英文原文
In this edition, we highlight several important developments across the Huntington's disease therapeutic pipeline. We discuss the launch and subsequent discontinuation of POINT-HD, the first-in-human phase I study of RG6496 (Hoffmann-La Roche), an allele-selective antisense oligonucleotide designed to selectively lower mutant huntingtin while largely preserving wild-type huntingtin expression. We also discuss the launch of two phase III efficacy trials: INVEST-HD, evaluating votoplam (Novartis Pharmaceuticals), and PRECISE-HD, evaluating pridopidine (Prilenia). In addition, we summarise recent advances across the field, including preclinical updates for LQT-23 (LoQus23), SRP-1005 (Sarepta Therapeutics) and HRN001 (Harness Therapeutics); interim 12-month clinical results from SKY-0515 (Skyhawk Therapeutics); the announcement by uniQure that 3-year data from the phase I/II AMT-130 study may support a Biologics License Application seeking accelerated approval and Roche's discontinuation of the phase II GENERATION HD2 study of tominersen. Finally, we provide an updated listing of registered and ongoing clinical trials in Huntington's disease. In this edition, we highlight several important developments across the Huntington's disease therapeutic pipeline. We discuss the launch and subsequent discontinuation of POINT-HD, the first-in-human phase I study of RG6496 (Hoffmann-La Roche), an allele-selective antisense oligonucleotide designed to selectively lower mutant huntingtin while largely preserving wild-type huntingtin expression. We also discuss the launch of two phase III efficacy trials: INVEST-HD, evaluating votoplam (Novartis Pharmaceuticals), and PRECISE-HD, evaluating pridopidine (Prilenia). In addition, we summarise recent advances across the field, including preclinical updates for LQT-23 (LoQus23), SRP-1005 (Sarepta Therapeutics) and HRN001 (Harness Therapeutics); interim 12-month clinical results from SKY-0515 (Skyhawk Therapeutics); the announcement by uniQure that 3-year data from the phase I/II AMT-130 study may support a Biologics License Application seeking accelerated approval and Roche's discontinuation of the phase II GENERATION HD2 study of tominersen. Finally, we provide an updated listing of registered and ongoing clinical trials in Huntington's disease.