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PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington’s Disease Program

12 月 9, 2024

December 2, 2024 – PTC to receive $1.0B in cash at closing –– PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones –– PTC to share profits in the U.S. and tiered double-digit royalties on ex-U.S. net sales –– Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of

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Sage Therapeutics ceases development of dalzanemdor in HD

12 月 9, 2024

The Phase 2 DIMENSION Study did not meet its primary endpoint Dalzanemdor was generally well-tolerated; no new safety signals were observed Based on these data, the Company does not plan further development of dalzanemdor CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nov. 20, 2024– Sage Therapeutics, Inc. (Nasdaq: SAGE) today announced topline results from the Phase 2

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LoQus23 Therapeutics announces £35 million financing to advance a new drug aimed at inhibiting somatic expansion in Huntington’s Disease

10 月 2, 2024

Cambridge, UK, 2 October 2024 – LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s Disease, myotonic dystrophy type 1 and similar triplet repeat expansion diseases, today announces the successful close of its £35 million (c.$43 million) Series

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FDA 授予 PTC518 亨廷顿氏病项目快速通道称号

9 月 26, 2024

PTC518 是一种口服药物，可减少导致疾病进展的变异亨廷汀蛋白的生成。 获得 "快速通道 "认证的项目可受益于与美国食品药品管理局的早期互动，并有资格获得优先审评和加速审批。在此之前，PTC 发布了其 2 期研究的 12 个月中期数据，其中包括

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不懈探索纪事：创建基金会

8 月 1, 2024

我是亨廷顿氏症家族的第三代传人，但直到 2024 年，在我第二任父亲 F. Cook 的不懈努力下，我们才得以确认这种疾病是如何传入家族的。我的外曾祖父是一位法国人，名叫查尔斯-诺维尔-卡廷（Charles Novel-Catin，1883-1936 年）、

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Wave 生命科学公司宣布 SELECT-HD 1b/2a 期试验取得积极成果，首次在临床上证明可降低亨廷顿氏症患者的等位基因选择性突变亨廷廷蛋白水平

6 月 25, 2024

今天，Wave Life Sciences 公司宣布了 SELECT-HD 的积极结果，这是我们评估研究疗法 WVE-003 的 1b/2a 期安慰剂对照试验。这些结果表明，WVE-003 能选择性地降低亨廷顿氏病患者体内有毒的突变型亨廷汀（mHTT）蛋白，并保留健康的野生型亨廷汀（wtHTT）蛋白。试验结果在分析中，比较了

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PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington’s Disease Program

12 月 9, 2024

December 2, 2024 – PTC to receive $1.0B in cash at closing –– PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones –– PTC to share profits in the U.S. and tiered double-digit royalties on ex-U.S. net sales –– Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of

阅读更多 "

Sage Therapeutics ceases development of dalzanemdor in HD

12 月 9, 2024

The Phase 2 DIMENSION Study did not meet its primary endpoint Dalzanemdor was generally well-tolerated; no new safety signals were observed Based on these data, the Company does not plan further development of dalzanemdor CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nov. 20, 2024– Sage Therapeutics, Inc. (Nasdaq: SAGE) today announced topline results from the Phase 2

阅读更多 "

LoQus23 Therapeutics announces £35 million financing to advance a new drug aimed at inhibiting somatic expansion in Huntington’s Disease

10 月 2, 2024

Cambridge, UK, 2 October 2024 – LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s Disease, myotonic dystrophy type 1 and similar triplet repeat expansion diseases, today announces the successful close of its £35 million (c.$43 million) Series

阅读更多 "

FDA 授予 PTC518 亨廷顿氏病项目快速通道称号

9 月 26, 2024

PTC518 是一种口服药物，可减少导致疾病进展的变异亨廷汀蛋白的生成。 获得 "快速通道 "认证的项目可受益于与美国食品药品管理局的早期互动，并有资格获得优先审评和加速审批。在此之前，PTC 发布了其 2 期研究的 12 个月中期数据，其中包括

阅读更多 "

不懈探索纪事：创建基金会

8 月 1, 2024

我是亨廷顿氏症家族的第三代传人，但直到 2024 年，在我第二任父亲 F. Cook 的不懈努力下，我们才得以确认这种疾病是如何传入家族的。我的外曾祖父是一位法国人，名叫查尔斯-诺维尔-卡廷（Charles Novel-Catin，1883-1936 年）、

阅读更多 "

Wave 生命科学公司宣布 SELECT-HD 1b/2a 期试验取得积极成果，首次在临床上证明可降低亨廷顿氏症患者的等位基因选择性突变亨廷廷蛋白水平

6 月 25, 2024

今天，Wave Life Sciences 公司宣布了 SELECT-HD 的积极结果，这是我们评估研究疗法 WVE-003 的 1b/2a 期安慰剂对照试验。这些结果表明，WVE-003 能选择性地降低亨廷顿氏病患者体内有毒的突变型亨廷汀（mHTT）蛋白，并保留健康的野生型亨廷汀（wtHTT）蛋白。试验结果在分析中，比较了

阅读更多 "
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