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<p>RAPID 基於 CIRM 之前的工作，包括最近在六個月內開發的針對 CPS1 缺乏症的基因療法。圖片來源：warut pothikit / Shutterstock.com。</p>
<p>美國加利福尼亞再生醫學研究所（CIRM）已批准罕見疾病加速平台與創新與交付（RAPID）資金計劃，撥款 1 億美元用於兩年。</p>
<p>該倡議旨在加速美國針對罕見疾病的平台型基因療法的開發。</p>
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<p>它還旨在創建一個可擴展的模型，以提供這些療法，滿足超過 3000 萬名受罕見疾病影響的人的需求。</p>
<p>RAPID 基於 CIRM 之前的工作，包括最近在六個月內開發的針對氨基甲酰磷酸合成酶 1（CPS1）缺乏症的基因療法。</p>
<p>該計劃將重點加速罕見疾病的治療，大多數目前缺乏批准的療法。</p>
<p>它將資助驗證和創新獎項，其中前者針對已收到美國食品和藥物管理局（FDA）預研究新藥（IND）反饋的項目。</p>
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<p>驗證獎項資助通過首次人體臨牀試驗的活動，而創新獎項旨在減少測試要求或擴大適用性以涵蓋各種技術和罕見疾病。</p>
<p>CIRM 將要求 RAPID 獲獎者在其網絡內實時分享知識，並及時發佈數據。這種方法旨在加速學習和相關項目之間的監管對齊。</p>
<p>CIRM 臨牀前開發副總裁 Shyam Patel 表示：“RAPID 旨在從根本上重塑我們推進罕見疾病治療的方式。通過專注於可擴展的平台技術，我們正在加速個別項目，同時建立一個能夠更快、更高效地開發整個基因疾病類別的基礎設施。</p>
<p>“這些療法不僅有潛力降低終身醫療成本，還將加強合作伙伴關係，簡化臨牀路徑，最終確保有前景的療法能夠惠及那些沒有其他選擇的患者。”</p>

<p>加利福尼亞再生醫學研究所（CIRM）啓動了 RAPID 資金計劃，計劃在兩年內撥款 1 億美元，以加速罕見疾病基因療法的發展。該倡議旨在創建一個可擴展的模型，以滿足超過 3000 萬名受罕見疾病影響的患者的需求。RAPID 將資助驗證和創新獎項，重點支持獲得 FDA 反饋和首次人體臨牀試驗的項目。CIRM 的 Shyam Patel 強調，RAPID 旨在重塑罕見疾病的治療進展，提高效率並促進合作</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 基於 CIRM 之前的工作，包括最近在六個月內開發的針對 CPS1 缺乏症的基因療法。圖片來源：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>美國加利福尼亞再生醫學研究所（CIRM）已批准罕見疾病加速平台與創新與交付（RAPID）資金計劃，撥款 1 億美元用於兩年。</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>該倡議旨在加速美國針對罕見疾病的平台型基因療法的開發。</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>它還旨在創建一個可擴展的模型，以提供這些療法，滿足超過 3000 萬名受罕見疾病影響的人的需求。</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 基於 CIRM 之前的工作，包括最近在六個月內開發的針對氨基甲酰磷酸合成酶 1（CPS1）缺乏症的基因療法。</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>該計劃將重點加速罕見疾病的治療，大多數目前缺乏批准的療法。</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>它將資助驗證和創新獎項，其中前者針對已收到美國食品和藥物管理局（FDA）預研究新藥（IND）反饋的項目。</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>驗證獎項資助通過首次人體臨牀試驗的活動，而創新獎項旨在減少測試要求或擴大適用性以涵蓋各種技術和罕見疾病。</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 將要求 RAPID 獲獎者在其網絡內實時分享知識，並及時發佈數據。這種方法旨在加速學習和相關項目之間的監管對齊。</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 臨牀前開發副總裁 Shyam Patel 表示：“RAPID 旨在從根本上重塑我們推進罕見疾病治療的方式。通過專注於可擴展的平台技術，我們正在加速個別項目，同時建立一個能夠更快、更高效地開發整個基因疾病類別的基礎設施。</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>“這些療法不僅有潛力降低終身醫療成本，還將加強合作伙伴關係，簡化臨牀路徑，最終確保有前景的療法能夠惠及那些沒有其他選擇的患者。”</p>
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加州再生醫學研究所（CIRM）推出了針對罕見疾病的 RAPID 資助計劃