<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/02/PT-01-02-2026-shutterstock_1415242190-430x241.jpg?x-oss-process=image/auto-orient,1/interlace,1/resize,w_1440,h_1440/quality,q_95/format,jpg" alt="" width="430" height="241" original-src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/02/PT-01-02-2026-shutterstock_1415242190-430x241.jpg"/></p>
<p>RAPID 基于 CIRM 之前的工作，包括最近在六个月内开发的针对 CPS1 缺乏症的基因疗法。图片来源：warut pothikit / Shutterstock.com。</p>
<p>美国加利福尼亚再生医学研究所（CIRM）已批准罕见疾病加速平台与创新与交付（RAPID）资金计划，拨款 1 亿美元用于两年。</p>
<p>该倡议旨在加速美国针对罕见疾病的平台型基因疗法的开发。</p>
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<p>它还旨在创建一个可扩展的模型，以提供这些疗法，满足超过 3000 万名受罕见疾病影响的人的需求。</p>
<p>RAPID 基于 CIRM 之前的工作，包括最近在六个月内开发的针对氨基甲酰磷酸合成酶 1（CPS1）缺乏症的基因疗法。</p>
<p>该计划将重点加速罕见疾病的治疗，大多数目前缺乏批准的疗法。</p>
<p>它将资助验证和创新奖项，其中前者针对已收到美国食品和药物管理局（FDA）预研究新药（IND）反馈的项目。</p>
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<p>验证奖项资助通过首次人体临床试验的活动，而创新奖项旨在减少测试要求或扩大适用性以涵盖各种技术和罕见疾病。</p>
<p>CIRM 将要求 RAPID 获奖者在其网络内实时分享知识，并及时发布数据。这种方法旨在加速学习和相关项目之间的监管对齐。</p>
<p>CIRM 临床前开发副总裁 Shyam Patel 表示：“RAPID 旨在从根本上重塑我们推进罕见疾病治疗的方式。通过专注于可扩展的平台技术，我们正在加速个别项目，同时建立一个能够更快、更高效地开发整个基因疾病类别的基础设施。</p>
<p>“这些疗法不仅有潜力降低终身医疗成本，还将加强合作伙伴关系，简化临床路径，最终确保有前景的疗法能够惠及那些没有其他选择的患者。”</p>

<p>加利福尼亚再生医学研究所（CIRM）启动了 RAPID 资金计划，计划在两年内拨款 1 亿美元，以加速罕见疾病基因疗法的发展。该倡议旨在创建一个可扩展的模型，以满足超过 3000 万名受罕见疾病影响的患者的需求。RAPID 将资助验证和创新奖项，重点支持获得 FDA 反馈和首次人体临床试验的项目。CIRM 的 Shyam Patel 强调，RAPID 旨在重塑罕见疾病的治疗进展，提高效率并促进合作</p>

<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/02/PT-01-02-2026-shutterstock_1415242190-430x241.jpg" alt="" original-src="https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/02/PT-01-02-2026-shutterstock_1415242190-430x241.jpg"/></p>
<p>RAPID builds on CIRM’s previous work, including a recent genetic therapy developed within six months for CPS1 deficiency. Credit: warut pothikit / Shutterstock.com.</p>
<div class="lb-trans"><p>RAPID 基于 CIRM 之前的工作，包括最近在六个月内开发的针对 CPS1 缺乏症的基因疗法。图片来源：warut pothikit / Shutterstock.com。</p>
</div><p>The California Institute for Regenerative Medicine (CIRM) in the US has approved the Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) funding programme, setting aside $100m over two years.</p>
<div class="lb-trans"><p>美国加利福尼亚再生医学研究所（CIRM）已批准罕见疾病加速平台与创新与交付（RAPID）资金计划，拨款 1 亿美元用于两年。</p>
</div><p>The initiative aims to speed up the development of platform-based genetic therapies for rare diseases in the US.</p>
<div class="lb-trans"><p>该倡议旨在加速美国针对罕见疾病的平台型基因疗法的开发。</p>
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</div><p>It also aims to create a scalable model to deliver these therapies, addressing the needs of more than 30 million people affected by rare conditions.</p>
<div class="lb-trans"><p>它还旨在创建一个可扩展的模型，以提供这些疗法，满足超过 3000 万名受罕见疾病影响的人的需求。</p>
</div><p>RAPID builds on CIRM’s previous work, including a recent genetic therapy developed within six months for carbamoyl phosphate synthetase 1 (CPS1) deficiency.</p>
<div class="lb-trans"><p>RAPID 基于 CIRM 之前的工作，包括最近在六个月内开发的针对氨基甲酰磷酸合成酶 1（CPS1）缺乏症的基因疗法。</p>
</div><p>The programme will focus on accelerating treatments for rare diseases, most of which currently lack approved therapies.</p>
<div class="lb-trans"><p>该计划将重点加速罕见疾病的治疗，大多数目前缺乏批准的疗法。</p>
</div><p>It will fund validation and innovation awards, of which the former target projects that have received US Food and Drug Administration (FDA) pre-investigational new drug (IND) feedback.</p>
<div class="lb-trans"><p>它将资助验证和创新奖项，其中前者针对已收到美国食品和药物管理局（FDA）预研究新药（IND）反馈的项目。</p>
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</div><p>Validation awards fund activities through the completion of first-in-human clinical trials while innovation awards aim to minimise testing requirements or broaden applicability to various technologies and rare diseases.</p>
<div class="lb-trans"><p>验证奖项资助通过首次人体临床试验的活动，而创新奖项旨在减少测试要求或扩大适用性以涵盖各种技术和罕见疾病。</p>
</div><p>CIRM will require RAPID awardees to share knowledge in near-real time within its network and publish data promptly. This approach is intended to accelerate learning and regulatory alignment across related projects.</p>
<div class="lb-trans"><p>CIRM 将要求 RAPID 获奖者在其网络内实时分享知识，并及时发布数据。这种方法旨在加速学习和相关项目之间的监管对齐。</p>
</div><p>CIRM preclinical development associate vice-president Shyam Patel said: “RAPID is designed to fundamentally reshape how we advance treatments for people with rare diseases. By focusing on scalable platform technologies, we’re accelerating individual projects while building an infrastructure that enables faster, more efficient development across entire categories of genetic conditions.</p>
<div class="lb-trans"><p>CIRM 临床前开发副总裁 Shyam Patel 表示：“RAPID 旨在从根本上重塑我们推进罕见疾病治疗的方式。通过专注于可扩展的平台技术，我们正在加速个别项目，同时建立一个能够更快、更高效地开发整个基因疾病类别的基础设施。</p>
</div><p>“These therapies not only have the potential to reduce lifetime healthcare costs, but also will strengthen partnerships, streamline pathways to the clinic, and ultimately ensure that promising therapies reach patients who otherwise have no options.”</p>
<div class="lb-trans"><p>“这些疗法不仅有潜力降低终身医疗成本，还将加强合作伙伴关系，简化临床路径，最终确保有前景的疗法能够惠及那些没有其他选择的患者。”</p>
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加州再生医学研究所（CIRM）推出了针对罕见疾病的 RAPID 资助计划