<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/01/Solid-Biosciences-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/24/2026/01/Solid-Biosciences-430x241.jpg"/></p>
<p>Solid Biosciences 正在尋求在基因治療中實現更好的遞送，以推動其專有衣殼的應用，並啓動其主要項目的第三階段入組。圖片來源：metamorworks via Shutterstock.com。</p>
<p>Solid Biosciences 正在為可能的 “變革性” 一年做好準備，基於其 2025 年的成功，圍繞新平台構建其產品管線，旨在克服基因治療領域的投資逆風。</p>
<p>首席執行官 Bo Cumbo 在美國舊金山舉行的摩根大通醫療會議上表示，增強的遞送方法將是使基因治療 “真正可投資” 的關鍵，會議時間為 1 月 12 日至 15 日。</p>
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<h4>NPVM: Alaunos Therapeutics Inc 的基因修飾細胞治療針對...</h4>
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<p>這家臨牀階段的基因醫學公司已在行業和學術界獲得超過 50 項協議，包括許可證，用於其專有衣殼 AAV-SLB101 的使用，Cumbo 説道。</p>
<p>近年來，細胞和基因治療領域的投資活動停滯不前，許多公司選擇退出該領域，轉向更有利可圖的模式。然而，市場現在似乎正在再次增強，預計到 2031 年將達到 630 億美元，依據 GlobalData 的一份報告。</p>
<p>GlobalData 是 <em>Pharmaceutical Technology.</em> 的母公司。</p>
<p>AAV-SLB101 衣殼旨在結合骨骼和心臟肌肉上的內分泌受體，同時減少肝臟生物分佈，以解決相關的毒性問題。目前批准的療法中使用的 AAV 載體的主要缺點是肝毒性的發生。</p>
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<p>Cumbo 説道，多個漸進變化的匯聚使該衣殼與以往幾代產品有所不同。</p>
<h2>Solid 的平台技術在臨牀中</h2>
<p>Solid Biosciences 正在其一次性基因治療 SGT-003 中使用 AAV-SLB101，旨在恢復 X 連鎖隱性疾病杜氏肌營養不良症（DMD）中部分功能的肌萎縮蛋白，該病主要影響男性。</p>
<p>安全問題促使 DMD 基因治療領域展開更廣泛的討論，因為 Sarepta 的 Elevidys 與 兩例死亡 的非行走患者有關，導致在 2025 年對其標籤進行修改，限制使用於行走患者。</p>
<p>在 I/II 期 INSPIRE（NCT06138639）試驗中，已有 33 名患者接受了 SGT-003 的治療。該療法在僅使用類固醇的免疫調節方案下通常耐受良好，Cumbo 表示這使得公司與其他公司有所區別。他強調向監管機構證明公司沒有 “窺探” 外部對照數據的重要性，並指出 Solid 與 FDA 的期望高度一致。</p>
<p>與此同時，Solid 已在 III 期隨機對照安慰劑 IMPACT 試驗（NCT07160634）中招募了首位參與者。Cumbo 表示，公司希望向 FDA 展示他們 “沒有走捷徑”，並對獲得加速批准保持樂觀，計劃在年底前開始滾動生物製品許可證申請（BLA）。</p>
<p>在美國以外，他強調 Solid 已獲得英國創新許可和准入通道（ILAP）認證，旨在加快上市時間並加速患者的獲取。他補充説，公司可能會推出英國首個 DMD 基因治療。</p>
<p>在這裏註冊以接收關於 2026 年 JP 摩根醫療會議最新醫療行業趨勢的每日更新 。在這裏註冊以在會議後接收綜合報告 。</p>

SLDB

<p>Solid Biosciences 正在專注於其專有的 AAV-SLB101 外殼，以增強基因治療的遞送，目標是實現變革性的一年。該公司已為其技術簽署了超過 50 項協議，並正在啓動針對杜氏肌營養不良症的基因治療 SGT-003 的 III 期臨牀試驗。首席執行官 Bo Cumbo 強調了改進遞送方法的重要性，以吸引投資回流到該行業。預計到 2031 年，細胞和基因治療市場將達到 630 億美元，Solid 旨在加速其治療的批准，並有可能在英國推出</p>

<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/01/Solid-Biosciences-430x241.jpg" alt="" original-src="https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/01/Solid-Biosciences-430x241.jpg"/></p>
<p>Solid Biosciences is bidding for enhanced delivery in gene therapy as it drives uptake of its proprietary capsid and initiates Phase III enrolment for its lead programme. Image credit: metamorworks via Shutterstock.com.</p>
<div class="lb-trans"><p>Solid Biosciences 正在尋求在基因治療中實現更好的遞送，以推動其專有衣殼的應用，並啓動其主要項目的第三階段入組。圖片來源：metamorworks via Shutterstock.com。</p>
</div><p>Solid Biosciences is positioning itself for a potentially “transformative” year as it builds on its 2025 success and centres its pipeline around novel platforms, aiming to overcome investment headwinds in the gene therapy sector.</p>
<div class="lb-trans"><p>Solid Biosciences 正在為可能的 “變革性” 一年做好準備，基於其 2025 年的成功，圍繞新平台構建其產品管線，旨在克服基因治療領域的投資逆風。</p>
</div><p>Enhanced delivery methods will be key to making gene therapy “really investable again,” said CEO Bo Cumbo at the JP Morgan Healthcare Conference in San Francisco, US, taking place from 12 to 15 January.</p>
<div class="lb-trans"><p>首席執行官 Bo Cumbo 在美國舊金山舉行的摩根大通醫療會議上表示，增強的遞送方法將是使基因治療 “真正可投資” 的關鍵，會議時間為 1 月 12 日至 15 日。</p>
</div><h3>Go deeper with GlobalData</h3>
<div class="lb-trans"><h3>深入瞭解 GlobalData</h3>
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</div><h4>NPVM: Alaunos Therapeutics Inc's Gene-Modified Cell Therapy to Targ...</h4>
<div class="lb-trans"><h4>NPVM: Alaunos Therapeutics Inc 的基因修飾細胞治療針對...</h4>
</div><h5>Go deeper with GlobalData</h5>
<div class="lb-trans"><h5>深入瞭解 GlobalData</h5>
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</div><p>The clinical-stage genetic medicine company has secured more than 50 agreements, including licences, across industry and academia for use of its proprietary capsid AAV-SLB101, said Cumbo.</p>
<div class="lb-trans"><p>這家臨牀階段的基因醫學公司已在行業和學術界獲得超過 50 項協議，包括許可證，用於其專有衣殼 AAV-SLB101 的使用，Cumbo 説道。</p>
</div><p>Investor activity in the cell and gene therapy space has stalled in recent years, with several companies choosing to leave the space in favour of more lucrative modalities. However, the market now appears to be strengthening again and is expected to reach $63bn by 2031, according to a report by GlobalData.</p>
<div class="lb-trans"><p>近年來，細胞和基因治療領域的投資活動停滯不前，許多公司選擇退出該領域，轉向更有利可圖的模式。然而，市場現在似乎正在再次增強，預計到 2031 年將達到 630 億美元，依據 GlobalData 的一份報告。</p>
</div><p>GlobalData is the parent company of <em>Pharmaceutical Technology.</em></p>
<div class="lb-trans"><p>GlobalData 是 <em>Pharmaceutical Technology.</em> 的母公司。</p>
</div><p>The AAV-SLB101 capsid is designed to bind endocrine receptors on skeletal and cardiac muscle, while reducing liver biodistribution to address associated toxicities. The main downside of AAV vectors used in currently approved therapies is occurrences of liver toxicity.</p>
<div class="lb-trans"><p>AAV-SLB101 衣殼旨在結合骨骼和心臟肌肉上的內分泌受體，同時減少肝臟生物分佈，以解決相關的毒性問題。目前批准的療法中使用的 AAV 載體的主要缺點是肝毒性的發生。</p>
</div><p><img src="/wp-content/themes/goodlife-wp-B2B/assets/images/strategic-int.png" alt="" original-src="/wp-content/themes/goodlife-wp-B2B/assets/images/strategic-int.png"/></p>
<p>GlobalData Strategic Intelligence</p>
<div class="lb-trans"><p>GlobalData 戰略情報</p>
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<div class="lb-trans"><h1>美國關税正在變化 - 你會反應還是預見？</h1>
</div><p>Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.</p>
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</div><p>A culmination of multiple incremental changes differentiates the capsid from previous generations, said Cumbo.</p>
<div class="lb-trans"><p>Cumbo 説道，多個漸進變化的匯聚使該衣殼與以往幾代產品有所不同。</p>
</div><h2>Solid’s platform technology in the clinic</h2>
<div class="lb-trans"><h2>Solid 的平台技術在臨牀中</h2>
</div><p>Solid Biosciences is using AAV-SLB101 in its one-time gene therapy SGT-003, designed to restore partial function of the dystrophin protein in the X-linked recessive condition Duchenne muscular dystrophy (DMD), which primarily affects males.</p>
<div class="lb-trans"><p>Solid Biosciences 正在其一次性基因治療 SGT-003 中使用 AAV-SLB101，旨在恢復 X 連鎖隱性疾病杜氏肌營養不良症（DMD）中部分功能的肌萎縮蛋白，該病主要影響男性。</p>
</div><p>Safety concerns have prompted wider discussions in the DMD gene therapy landscape, as Sarepta’s Elevidys was linked to two fatalities in non-ambulatory patients, leading to a label alteration restricting use to ambulatory patients in 2025.</p>
<div class="lb-trans"><p>安全問題促使 DMD 基因治療領域展開更廣泛的討論，因為 Sarepta 的 Elevidys 與 兩例死亡 的非行走患者有關，導致在 2025 年對其標籤進行修改，限制使用於行走患者。</p>
</div><p>In the Phase I/II INSPIRE (NCT06138639) trial, 33 patients have now been dosed with SGT-003. The therapy has been generally well tolerated with a steroid-only immunomodulation regimen, which Cumbo says differentiates the company from the rest of the field. He emphasised the importance of demonstrating to regulators that the company has not “snooped” the external control data, noting that Solid is well aligned with the FDA’s expectations.</p>
<div class="lb-trans"><p>在 I/II 期 INSPIRE（NCT06138639）試驗中，已有 33 名患者接受了 SGT-003 的治療。該療法在僅使用類固醇的免疫調節方案下通常耐受良好，Cumbo 表示這使得公司與其他公司有所區別。他強調向監管機構證明公司沒有 “窺探” 外部對照數據的重要性，並指出 Solid 與 FDA 的期望高度一致。</p>
</div><p>In parallel, Solid has enrolled its first participant in the Phase III randomised placebo-controlled IMPACT trial (NCT07160634). Cumbo said that the company wants to show the FDA that they are “not cutting corners” and remains hopeful of gaining accelerated approval, with plans to start a rolling biologics licence application (BLA) by the end of the year.</p>
<div class="lb-trans"><p>與此同時，Solid 已在 III 期隨機對照安慰劑 IMPACT 試驗（NCT07160634）中招募了首位參與者。Cumbo 表示，公司希望向 FDA 展示他們 “沒有走捷徑”，並對獲得加速批准保持樂觀，計劃在年底前開始滾動生物製品許可證申請（BLA）。</p>
</div><p>Outside the US, he highlighted that Solid has been awarded the UK’s Innovative Licensing and Access Pathway (ILAP) designation, which aims to accelerate time to market and expedite patient access. He added that the company could launch the UK’s first gene therapy for DMD.</p>
<div class="lb-trans"><p>在美國以外，他強調 Solid 已獲得英國創新許可和准入通道（ILAP）認證，旨在加快上市時間並加速患者的獲取。他補充説，公司可能會推出英國首個 DMD 基因治療。</p>
</div><p>Sign up here to receive daily updates <em>on the latest healthcare industry trends emerging from the JP Morgan Healthcare conference 2026.</em> Sign up here to receive a comprehensive report <em>after the conference</em>.</p>
<div class="lb-trans"><p>在這裏註冊以接收關於 2026 年 JP 摩根醫療會議最新醫療行業趨勢的每日更新 。在這裏註冊以在會議後接收綜合報告 。</p>
</div>

JPM26：Solid Biosciences 寄望於其 AAV 衣殼來推動基因療法的成功