<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2025/11/Genscript-2025-430x241.jpg?x-oss-process=image/auto-orient,1/interlace,1/resize,w_1440,h_1440/quality,q_95/format,jpg" alt="" original-src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2025/11/Genscript-2025-430x241.jpg"/></p>
<p>安全性、耐用性和可扩展性是推动细胞和基因治疗领域投资决策的主要因素，行业代表表示。图片来源：Jam Bensilum / GlobalData。</p>
<p></p>
<p>随着对创新体内策略的信心增强，一些投资者表示，这些策略可能是细胞和基因治疗（CGT）领域的未来。</p>
<p>在最近几个月，体内嵌合抗原受体（CAR）-T 领域发生了几笔高调交易。上个月，百时美施贵宝收购 了 Orbital Therapeutics，交易金额为 15 亿美元，紧接着在 9 月，吉利德科学收购 了 Interius BioTherapeutics，交易金额为 3.5 亿美元。在 7 月，AbbVie 收购了 Capstan Therapeutics，其体内 CAR-T 疗法的交易金额高达 21 亿美元。</p>
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<p>在 2025 年 11 月 20 日举行的 GenScript 生物科技全球论坛上，Sofinnova Partners 的 Matthieu Coutet 表示，制药公司和投资者正在向 CGT 领域的体内方法转变。Coutet 补充说，如今，制药公司对投资自体细胞治疗公司的兴趣非常有限。</p>
<p>尽管热情高涨，体内基因治疗仍处于初期阶段，人体数据有限，RA Capital Management 的合伙人 Laura Stoppel 表示。</p>
<p>Stoppel 表示，她对自身免疫疾病的同种异体方法持 “谨慎乐观” 态度，因此 RA Capital Management 正在该领域进行投资，但仍需要更多数据。</p>
<p>谈到自身免疫疾病时，她表示：“我认为这是一个同种异体治疗实际上有潜力与自体细胞治疗的疗效相匹配的地方，尤其是当你不需要这些细胞的长期持久性时。”</p>
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<p>安全性仍然是 CGT 领域的一大障碍，系统性疗法使投资者保持谨慎。</p>
<p>尽管如此，专家表示，CGT 在传统肿瘤学领域之外仍有扩展的空间。例如，眼科被确定为药物开发的关键领域。BioGeneration Ventures 的普通合伙人 Daniela Couto 强调，Complement Therapeutics 正在研究干性年龄相关性黄斑变性（AMD）和地理性萎缩（GA），这是一个在 “巨大适应症” 中满足未满足需求的可投资 CGT 生物科技的例子。</p>
<p>“如果我们能做到一次性基因治疗，我们将改变治疗地理性萎缩患者的方式，” 她说。小组成员还描述了投资者和制药公司对局部给药基因治疗的青睐。</p>
<p>投资者考虑的另一个因素是替代治疗选择的可用性。随着成本效益高的 T 细胞连接剂的发展，Couto 表示，投资细胞治疗变得困难。她补充说，投资者主要倾向于原位给药方式，因为其成本和生产复杂性。</p>
<p>此外，Stoppel 强调，生物科技公司在制造能力方面的准备和投资是投资时考虑的关键因素。她还表示，需要更多创新来优化制造流程。</p>
<p><em>《制药技术》上的细胞与基因治疗报道由 Cytiva 支持。</em></p>
<p><em>编辑内容独立制作，遵循</em> 最高标准 <em>的新闻诚信。主题赞助商不参与编辑内容的创作。</em></p>
<h2>制药技术卓越奖 - 参赛的好处</h2>
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<p>投资者越来越关注细胞和基因治疗（CGT）领域的新技术，特别是体内 CAR-T 疗法。最近的高调收购包括百时美施贵宝以 15 亿美元收购 Orbital Therapeutics，以及吉利德科学以 3.5 亿美元收购 Interius BioTherapeutics。尽管充满热情，体内基因治疗仍处于早期阶段，且人类数据有限。安全性、可扩展性和制造能力是关键的投资考虑因素。专家们认为，CGT 在肿瘤学之外的扩展潜力巨大，眼科被认为是一个有前景的领域</p>

<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2025/11/Genscript-2025-430x241.jpg" alt="" original-src="https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2025/11/Genscript-2025-430x241.jpg"/></p>
<p>Safety, durability and scalability are some of the main factors driving investment decisions in the cell and gene therapy space, say industry representatives. Image credit: Jam Bensilum / GlobalData.</p>
<div class="lb-trans"><p>安全性、耐用性和可扩展性是推动细胞和基因治疗领域投资决策的主要因素，行业代表表示。图片来源：Jam Bensilum / GlobalData。</p>
</div><p></p>
<div class="lb-trans"><p></p>
</div><p>As confidence grows for innovative in vivo strategies, some investors are saying they could be the future of the cell and gene therapy (CGT) sector.</p>
<div class="lb-trans"><p>随着对创新体内策略的信心增强，一些投资者表示，这些策略可能是细胞和基因治疗（CGT）领域的未来。</p>
</div><p>In recent months, several high-profile deals have taken place within the in vivo chimeric antigen receptor (CAR)-T space. Last month, Bristol Myers Squibb acquired Orbital Therapeutics for $1.5bn, shortly after Gilead Sciences bought Interius BioTherapeutics for $350m in September. In July, AbbVie acquired Capstan Therapeutics and its in vivo CAR-T therapy for up to $2.1bn.</p>
<div class="lb-trans"><p>在最近几个月，体内嵌合抗原受体（CAR）-T 领域发生了几笔高调交易。上个月，百时美施贵宝收购 了 Orbital Therapeutics，交易金额为 15 亿美元，紧接着在 9 月，吉利德科学收购 了 Interius BioTherapeutics，交易金额为 3.5 亿美元。在 7 月，AbbVie 收购了 Capstan Therapeutics，其体内 CAR-T 疗法的交易金额高达 21 亿美元。</p>
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</div><h4>LOA and PTSR Model - Gene Therapy For Gastrointestinal Tract Cancer...</h4>
<div class="lb-trans"><h4>胃肠道癌症的基因治疗 - LOA 和 PTSR 模型...</h4>
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<div class="lb-trans"><h4>制药中的免疫肿瘤学：基于 CAR-T 细胞的组合</h4>
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</div><p>During a panel discussion on innovation and investment at the GenScript Biotech Global Forum London 2025 held on 20 November, Matthieu Coutet from Sofinnova Partners said pharma and investors are moving towards in vivo approaches in the CGT space. Coutet added that these days, pharma are showing very little interest in investing in autologous cell therapy companies.</p>
<div class="lb-trans"><p>在 2025 年 11 月 20 日举行的 GenScript 生物科技全球论坛上，Sofinnova Partners 的 Matthieu Coutet 表示，制药公司和投资者正在向 CGT 领域的体内方法转变。Coutet 补充说，如今，制药公司对投资自体细胞治疗公司的兴趣非常有限。</p>
</div><p>Despite the enthusiasm, in vivo gene therapy is still in its infancy with limited data in humans, said Laura Stoppel, partner at RA Capital Management.</p>
<div class="lb-trans"><p>尽管热情高涨，体内基因治疗仍处于初期阶段，人体数据有限，RA Capital Management 的合伙人 Laura Stoppel 表示。</p>
</div><p>Stoppel said she is “cautiously optimistic” about allogeneic approaches in autoimmune disease, and hence RA Capital Management is investing in the space, but more data is still needed.</p>
<div class="lb-trans"><p>Stoppel 表示，她对自身免疫疾病的同种异体方法持 “谨慎乐观” 态度，因此 RA Capital Management 正在该领域进行投资，但仍需要更多数据。</p>
</div><p>Speaking about autoimmune diseases she said: “I think that’s a place where allogeneic actually has the potential to match the efficacy of autologous cell therapy, especially if you don’t need long term persistence of these cells.”</p>
<div class="lb-trans"><p>谈到自身免疫疾病时，她表示：“我认为这是一个同种异体治疗实际上有潜力与自体细胞治疗的疗效相匹配的地方，尤其是当你不需要这些细胞的长期持久性时。”</p>
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</div><p>Safety also remains a hurdle in the CGT space, with systemic therapies prompting investor caution.</p>
<div class="lb-trans"><p>安全性仍然是 CGT 领域的一大障碍，系统性疗法使投资者保持谨慎。</p>
</div><p>Nonetheless, experts said there is scope to expand beyond the traditional fields of oncology with CGTs. For example, ophthalmology was identified as a key area for drug development. Daniela Couto, general partner at BioGeneration Ventures, highlighted Complement Therapeutics’ investigation in dry age-related macular degeneration (AMD) and geographic atrophy (GA) as an example of an investable CGT biotech working in an area of unmet need in a “huge indication”.</p>
<div class="lb-trans"><p>尽管如此，专家表示，CGT 在传统肿瘤学领域之外仍有扩展的空间。例如，眼科被确定为药物开发的关键领域。BioGeneration Ventures 的普通合伙人 Daniela Couto 强调，Complement Therapeutics 正在研究干性年龄相关性黄斑变性（AMD）和地理性萎缩（GA），这是一个在 “巨大适应症” 中满足未满足需求的可投资 CGT 生物科技的例子。</p>
</div><p>“If we could do a gene therapy, one and done, we would change the way we’re treating patients with geographic atrophy,” she said. Panellists also described favourability among investors and pharma for locally administered gene therapies.</p>
<div class="lb-trans"><p>“如果我们能做到一次性基因治疗，我们将改变治疗地理性萎缩患者的方式，” 她说。小组成员还描述了投资者和制药公司对局部给药基因治疗的青睐。</p>
</div><p>Another factor that investors are considering is the availability of alternate treatment options. With the development of cost-effective T-cell engagers, Couto said it is difficult to justify investing in cell therapies. She added that investors are largely drawn towards in situ delivery modalities, due to cost and complexity of production.</p>
<div class="lb-trans"><p>投资者考虑的另一个因素是替代治疗选择的可用性。随着成本效益高的 T 细胞连接剂的发展，Couto 表示，投资细胞治疗变得困难。她补充说，投资者主要倾向于原位给药方式，因为其成本和生产复杂性。</p>
</div><p>In addition, Stoppel highlighted that a biotech’s preparedness and investment in manufacturing capabilities is a key aspect of consideration when investing. She also said there is a need for more innovation to optimise manufacturing processes.</p>
<div class="lb-trans"><p>此外，Stoppel 强调，生物科技公司在制造能力方面的准备和投资是投资时考虑的关键因素。她还表示，需要更多创新来优化制造流程。</p>
</div><p><em>Cell &amp; Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva</em>.</p>
<div class="lb-trans"><p><em>《制药技术》上的细胞与基因治疗报道由 Cytiva 支持。</em></p>
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<div class="lb-trans"><p><em>编辑内容独立制作，遵循</em> 最高标准 <em>的新闻诚信。主题赞助商不参与编辑内容的创作。</em></p>
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<div class="lb-trans"><h2>制药技术卓越奖 - 参赛的好处</h2>
</div><p>Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today!</p>
<div class="lb-trans"><p>获得应有的认可！制药技术卓越奖 表彰创新、领导力和影响力。通过参赛，您展示了自己的成就，提升了行业形象，并将自己定位于推动制药进步的顶尖领导者之中。不要错过脱颖而出的机会——今天就提交您的参赛作品！</p>
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投资者押注于更新的技术和细胞与基因疗法的迹象