<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 正在寻求在基因治疗中实现更好的递送，以推动其专有衣壳的应用，并启动其主要项目的第三阶段入组。图片来源：metamorworks via Shutterstock.com。</p>
<p>Solid Biosciences 正在为可能的 “变革性” 一年做好准备，基于其 2025 年的成功，围绕新平台构建其产品管线，旨在克服基因治疗领域的投资逆风。</p>
<p>首席执行官 Bo Cumbo 在美国旧金山举行的摩根大通医疗会议上表示，增强的递送方法将是使基因治疗 “真正可投资” 的关键，会议时间为 1 月 12 日至 15 日。</p>
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<p>这家临床阶段的基因医学公司已在行业和学术界获得超过 50 项协议，包括许可证，用于其专有衣壳 AAV-SLB101 的使用，Cumbo 说道。</p>
<p>近年来，细胞和基因治疗领域的投资活动停滞不前，许多公司选择退出该领域，转向更有利可图的模式。然而，市场现在似乎正在再次增强，预计到 2031 年将达到 630 亿美元，依据 GlobalData 的一份报告。</p>
<p>GlobalData 是 <em>Pharmaceutical Technology.</em> 的母公司。</p>
<p>AAV-SLB101 衣壳旨在结合骨骼和心脏肌肉上的内分泌受体，同时减少肝脏生物分布，以解决相关的毒性问题。目前批准的疗法中使用的 AAV 载体的主要缺点是肝毒性的发生。</p>
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<p>Cumbo 说道，多个渐进变化的汇聚使该衣壳与以往几代产品有所不同。</p>
<h2>Solid 的平台技术在临床中</h2>
<p>Solid Biosciences 正在其一次性基因治疗 SGT-003 中使用 AAV-SLB101，旨在恢复 X 连锁隐性疾病杜氏肌营养不良症（DMD）中部分功能的肌萎缩蛋白，该病主要影响男性。</p>
<p>安全问题促使 DMD 基因治疗领域展开更广泛的讨论，因为 Sarepta 的 Elevidys 与 两例死亡 的非行走患者有关，导致在 2025 年对其标签进行修改，限制使用于行走患者。</p>
<p>在 I/II 期 INSPIRE（NCT06138639）试验中，已有 33 名患者接受了 SGT-003 的治疗。该疗法在仅使用类固醇的免疫调节方案下通常耐受良好，Cumbo 表示这使得公司与其他公司有所区别。他强调向监管机构证明公司没有 “窥探” 外部对照数据的重要性，并指出 Solid 与 FDA 的期望高度一致。</p>
<p>与此同时，Solid 已在 III 期随机对照安慰剂 IMPACT 试验（NCT07160634）中招募了首位参与者。Cumbo 表示，公司希望向 FDA 展示他们 “没有走捷径”，并对获得加速批准保持乐观，计划在年底前开始滚动生物制品许可证申请（BLA）。</p>
<p>在美国以外，他强调 Solid 已获得英国创新许可和准入通道（ILAP）认证，旨在加快上市时间并加速患者的获取。他补充说，公司可能会推出英国首个 DMD 基因治疗。</p>
<p>在这里注册以接收关于 2026 年 JP 摩根医疗会议最新医疗行业趋势的每日更新 。在这里注册以在会议后接收综合报告 。</p>

SLDB

<p>Solid Biosciences 正在专注于其专有的 AAV-SLB101 外壳，以增强基因治疗的递送，目标是实现变革性的一年。该公司已为其技术签署了超过 50 项协议，并正在启动针对杜氏肌营养不良症的基因治疗 SGT-003 的 III 期临床试验。首席执行官 Bo Cumbo 强调了改进递送方法的重要性，以吸引投资回流到该行业。预计到 2031 年，细胞和基因治疗市场将达到 630 亿美元，Solid 旨在加速其治疗的批准，并有可能在英国推出</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 正在寻求在基因治疗中实现更好的递送，以推动其专有衣壳的应用，并启动其主要项目的第三阶段入组。图片来源：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 正在为可能的 “变革性” 一年做好准备，基于其 2025 年的成功，围绕新平台构建其产品管线，旨在克服基因治疗领域的投资逆风。</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>首席执行官 Bo Cumbo 在美国旧金山举行的摩根大通医疗会议上表示，增强的递送方法将是使基因治疗 “真正可投资” 的关键，会议时间为 1 月 12 日至 15 日。</p>
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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>这家临床阶段的基因医学公司已在行业和学术界获得超过 50 项协议，包括许可证，用于其专有衣壳 AAV-SLB101 的使用，Cumbo 说道。</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>近年来，细胞和基因治疗领域的投资活动停滞不前，许多公司选择退出该领域，转向更有利可图的模式。然而，市场现在似乎正在再次增强，预计到 2031 年将达到 630 亿美元，依据 GlobalData 的一份报告。</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 衣壳旨在结合骨骼和心脏肌肉上的内分泌受体，同时减少肝脏生物分布，以解决相关的毒性问题。目前批准的疗法中使用的 AAV 载体的主要缺点是肝毒性的发生。</p>
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<p>GlobalData Strategic Intelligence</p>
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<div class="lb-trans"><h1>美国关税正在变化 - 你会反应还是预见？</h1>
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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 说道，多个渐进变化的汇聚使该衣壳与以往几代产品有所不同。</p>
</div><h2>Solid’s platform technology in the clinic</h2>
<div class="lb-trans"><h2>Solid 的平台技术在临床中</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疗 SGT-003 中使用 AAV-SLB101，旨在恢复 X 连锁隐性疾病杜氏肌营养不良症（DMD）中部分功能的肌萎缩蛋白，该病主要影响男性。</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>安全问题促使 DMD 基因治疗领域展开更广泛的讨论，因为 Sarepta 的 Elevidys 与 两例死亡 的非行走患者有关，导致在 2025 年对其标签进行修改，限制使用于行走患者。</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）试验中，已有 33 名患者接受了 SGT-003 的治疗。该疗法在仅使用类固醇的免疫调节方案下通常耐受良好，Cumbo 表示这使得公司与其他公司有所区别。他强调向监管机构证明公司没有 “窥探” 外部对照数据的重要性，并指出 Solid 与 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>与此同时，Solid 已在 III 期随机对照安慰剂 IMPACT 试验（NCT07160634）中招募了首位参与者。Cumbo 表示，公司希望向 FDA 展示他们 “没有走捷径”，并对获得加速批准保持乐观，计划在年底前开始滚动生物制品许可证申请（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>在美国以外，他强调 Solid 已获得英国创新许可和准入通道（ILAP）认证，旨在加快上市时间并加速患者的获取。他补充说，公司可能会推出英国首个 DMD 基因治疗。</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>在这里注册以接收关于 2026 年 JP 摩根医疗会议最新医疗行业趋势的每日更新 。在这里注册以在会议后接收综合报告 。</p>
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JPM26：Solid Biosciences 寄望于其 AAV 衣壳来推动基因疗法的成功