<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/01/shutterstock_612999197-430x241.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/24/2026/01/shutterstock_612999197-430x241.jpg"/></p>
<p>斯科特·琼斯博士在检测开发领域工作了超过 35 年，目前领导 BioBridge Global 的研发部门。他的团队与 BBG 的子公司 BBG 先进疗法（BBGAT）紧密合作，开发和验证快速、高灵敏度、符合 GMP 标准的细胞和基因治疗制造商的效力检测。</p>
<p>效力检测是对产品生物功能的定量测量，包括细胞杀伤、免疫调节、伤口愈合、趋化性、血管生成或分化。它们在产品的整个生命周期中都是必需的，从初始开发和临床制造到监管批准和持续商业供应。</p>
<p>“对于细胞和基因治疗，展示产品仍然能够执行其应有的生物功能是非常重要的，” 琼斯博士说。“尽管有明确的制造过程，但这些是活细胞，受到许多变量的影响，这些变量可能会影响效力。”</p>
<p>琼斯博士解释说，效力检测在开发过程中被广泛使用，包括产品稳定性评估、可比性研究和制造过程研究。在监管申请期间，效力检测必须证明治疗能够持续产生预期效果，为监管机构提供产品强度和功能活性的证据。</p>
<p>在 GMP 制造过程中，效力检测是质量控制的一个重要方面，确认每一批次在治疗释放之前保持预期的生物活性。除此之外，琼斯博士解释说，它们还用于评估引入新原材料、生产过程或设施的任何影响。</p>
<h2>将检测与作用机制匹配</h2>
<p>“由于效力检测测量产品实现预期治疗效果的能力，因此它们必须是特定于产品的，” 琼斯博士说。“在 BBG 先进疗法，我们开发与作用机制（MOA）和产品特定适应症相一致的定制效力检测。”</p>
<p>琼斯博士解释说，细胞和基因治疗通常具有多个与效力相关的关键质量属性（CQA），这意味着需要多种不同的效力检测。如果间充质干细胞（MSC）治疗的部分功能是调节免疫系统，他的团队可能会进行细胞基础检测，将 MSC 与免疫细胞混合，以观察它们的反应。</p>
<p>“我们关注细胞激活、增殖以及细胞因子或生长因子的释放等方面。我们还可能测试细胞与其他细胞类型的相互作用，以展示它们能够激活或抑制特定反应，” 他解释道。</p>
<p>“因此，效力测试通常涉及不止一种检测，” 琼斯博士重申。“一些检测专注于细胞间相互作用，其他检测测量培养基中释放的因子，还有一些检测关注细胞的物理特性或表面标记。”</p>
<p>具体的方法取决于产品、其预期用途和所做的声明。目标始终是将检测结果与临床相关性联系起来，使研究人员能够证明细胞是有效的，并将按预期表现。</p>
<h2>效力检测延迟</h2>
<p>“虽然效力检测至关重要，但它们在细胞和基因治疗开发者中很少被优先考虑，” 琼斯博士说。“开发者通常会将这些检测放在次要位置，因为他们的重点是快速将产品推向市场。因此，效力检测可能在背景中显得像一个迫在眉睫的挑战。” 根据琼斯博士的说法，这种犹豫主要是由于开发所需的时间、精力和专业知识的显著性。</p>
<p>因此，效力检测通常在过程中过晚处理，这可能会阻碍进展，并可能导致批准延迟，延续多年。根据琼斯博士的说法，FDA 要求额外数据和检测并不罕见，即使生物制药公司认为他们现有的效力策略已经足够。</p>
<p>随着监管机构收紧对更强效力策略的规定，这一挑战可能会加剧。2023 年，FDA 对效力检测的最新指导更新强调了效力保证策略的重要性，其中包括使用多种互补检测来测量与效力相关的 CQA。该草案可能在未来几年内成为最终版本，提高了检测开发和验证的标准。</p>
<p>效力风险在监管评估中已经成为焦点。前 FDA CBER 主任彼得·马克斯指出，与效力相关的问题是细胞和基因治疗批准延迟的最常见原因之一 [i]，而一项在欧洲的研究估计，几乎一半的先进疗法药物（ATMP）申请在欧洲药品管理局数据库中在评估过程中遇到了与效力相关的问题，[ii]，这通常导致显著的延迟。</p>
<p>这些挑战强调了尽早开始检测开发的重要性。“生物制药公司应该从一开始就考虑这些检测，只要他们知道自己的适应症或作用机制，” 琼斯博士指出。</p>
<h2>早期开始，早期合作</h2>
<p>将检测开发策略融入早期开发可以带来丰厚的回报，使公司能够加速开发并提高产品一致性，从临床前阶段开始。</p>
<p>然而，这可能具有挑战性，特别是当产品受到配方变化的影响或当作用机制尚不明确时。尽管如此，早期开始为监管成功奠定了基础，使开发者能够从监管机构获得更早的反馈，相应调整他们的策略，并在符合 GMP 的条件下测试他们的效力检测。在临床试验中拥有强大可靠的效力策略也能保障产品质量，确保每位患者都能获得有效剂量。</p>
<p>早期和中期生物技术公司通常缺乏有效开发自身效力检测所需的深入专业知识，尤其是因为这些知识在关键窗口之外往往未被充分利用。与专家合作使公司能够在需要时获得所需的专业知识。这使得开发者能够专注于他们的产品，而像琼斯博士这样的专家则利用数十年的效力检测开发经验，设计符合严格监管标准的检测策略。</p>
<p>BBG Advanced Therapies 认识到随着细胞治疗行业的扩展，对这种专业知识的迫切需求。该公司位于德克萨斯州圣安东尼奥，提供一支专门的经验丰富的检测开发科学家团队，创建和验证特定于细胞或基因治疗产品的定制效力检测。BBGAT 致力于帮助创新者自信地加速其效力检测项目，从早期开发到监管批准，并提供支持公司进行起始材料收集和处理、测试、临床试验和生物制造的补充服务。</p>
<p>“与像 BBG Advanced Therapies 这样经验丰富的效力检测开发团队合作，将简化开发过程并确保检测的成功表现，” 琼斯博士说。</p>
<p>“我们的团队利用多种分析平台，如流式细胞术、ELISA、荧光成像、凝胶电泳和核酸定量，以确保与客户的技术能力、灵敏度要求和所需通量的最佳契合。我们还开发了一种专有的、正在申请专利的分析平台，以进一步增强我们为客户提供高价值解决方案的能力。”</p>
<p>BBG Advanced Therapies 提供的名为 Premiva 的新平台可以展示治疗在体内对抗癌细胞的功能。只需几滴治疗剂，这种可定制的动态流动基础专有平台就能为开发者提供对自体/异体细胞的合格和验证的特征描述，包括身份、特异性和效力。</p>
<p>在效力检测如同乌云笼罩开发的世界中，成功在于尽早开始和提前合作。通过从一开始就制定一个强大的、符合监管要求的策略，并与理解科学和监管机构期望的专家合作，开发者可以用信心取代不确定性，用简化的批准取代监管的拖延。</p>
<hr/>
<p>[i] Solvias. The Potency Puzzle: Overcoming Challenges in Potency Assays for Cell and Gene Therapy Development. [ii] Elsallab, Magdi et al. <em>Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals.</em> Molecular Therapy Methods &amp; Clinical Development, Volume 18, 269 – 279</p>

<p>来自 BioBridge Global 的 Scott Jones 博士讨论了为细胞和基因疗法开发效力检测的挑战。这些检测对于展示产品在整个生命周期中的生物功能至关重要，从开发到监管批准。尽管它们的重要性，开发者往往将其优先级降低，导致市场进入的潜在延迟。FDA 更新的指导强调了需要一个强有力的效力保证策略，突出了在监管评估中与效力相关问题的风险。Jones 提倡在产品生命周期中尽早整合检测开发，以减轻这些挑战</p>

<p><img src="https://imageproxy.pbkrs.com/https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/01/shutterstock_612999197-430x241.jpg" alt="" original-src="https://www.pharmaceutical-technology.com/wp-content/uploads/sites/24/2026/01/shutterstock_612999197-430x241.jpg"/></p>
<p>Dr. Scott Jones has spent more than 35 years advancing assay development and currently leads BioBridge Global’s R&amp;D department. His team works closely with BBG’s subsidiary, BBG Advanced Therapies (BBGAT) to develop and validate rapid, highly sensitive, GMP-compliant potency assays for cell and gene therapy manufacturers.</p>
<div class="lb-trans"><p>斯科特·琼斯博士在检测开发领域工作了超过 35 年，目前领导 BioBridge Global 的研发部门。他的团队与 BBG 的子公司 BBG 先进疗法（BBGAT）紧密合作，开发和验证快速、高灵敏度、符合 GMP 标准的细胞和基因治疗制造商的效力检测。</p>
</div><p>Potency assays are a quantitative measure of a product’s biological function, from cell killing and immuno-modulation to wound healing, chemotaxis, angiogenesis, or differentiation. They are needed across the entire lifecycle of a product, from initial development and clinical manufacturing to regulatory approval and ongoing commercial supply.</p>
<div class="lb-trans"><p>效力检测是对产品生物功能的定量测量，包括细胞杀伤、免疫调节、伤口愈合、趋化性、血管生成或分化。它们在产品的整个生命周期中都是必需的，从初始开发和临床制造到监管批准和持续商业供应。</p>
</div><p>“With cell and gene therapies, it’s important to show that the product still performs the biological function it’s supposed to do,” says Dr. Jones. “Even though there is a defined manufacturing process, these are living cells subject to numerous variables that can affect potency.”</p>
<div class="lb-trans"><p>“对于细胞和基因治疗，展示产品仍然能够执行其应有的生物功能是非常重要的，” 琼斯博士说。“尽管有明确的制造过程，但这些是活细胞，受到许多变量的影响，这些变量可能会影响效力。”</p>
</div><p>Dr. Jones explains that potency assays are used across the development process, in product stability evaluations, comparability studies, and manufacturing process studies. During regulatory filings, potency assays must demonstrate that the therapy consistently produces its intended effect, providing regulators with evidence of the product’s strength and functional activity.</p>
<div class="lb-trans"><p>琼斯博士解释说，效力检测在开发过程中被广泛使用，包括产品稳定性评估、可比性研究和制造过程研究。在监管申请期间，效力检测必须证明治疗能够持续产生预期效果，为监管机构提供产品强度和功能活性的证据。</p>
</div><p>During GMP manufacturing, potency assays are an integral aspect of quality control, confirming that each batch retains the intended biological activity before therapies are released. Beyond that, Dr. Jones explains they’re also used to evaluate any impacts of introducing new raw materials, production processes, or facilities.</p>
<div class="lb-trans"><p>在 GMP 制造过程中，效力检测是质量控制的一个重要方面，确认每一批次在治疗释放之前保持预期的生物活性。除此之外，琼斯博士解释说，它们还用于评估引入新原材料、生产过程或设施的任何影响。</p>
</div><h2>Matching assay to Mechanisms of Action</h2>
<div class="lb-trans"><h2>将检测与作用机制匹配</h2>
</div><p>“Since potency assays measure the ability of a product to achieve the intended therapeutic effect, they must be product specific,” Dr. Jones says. “At BBG Advanced Therapies, we develop tailored potency assays aligned with the mechanisms of action (MOA) and specific indication for use of the product.”</p>
<div class="lb-trans"><p>“由于效力检测测量产品实现预期治疗效果的能力，因此它们必须是特定于产品的，” 琼斯博士说。“在 BBG 先进疗法，我们开发与作用机制（MOA）和产品特定适应症相一致的定制效力检测。”</p>
</div><p>Dr. Jones explains that cell and gene therapies usually have multiple potency-related Critical Quality Attributes (CQAs), meaning multiple different potency assays are required. If part of a mesenchymal stem cell (MSC) therapy’s function is to modulate the immune system, his team might run a cell-based assay where the MSCs are mixed with immune cells to see how they respond.</p>
<div class="lb-trans"><p>琼斯博士解释说，细胞和基因治疗通常具有多个与效力相关的关键质量属性（CQA），这意味着需要多种不同的效力检测。如果间充质干细胞（MSC）治疗的部分功能是调节免疫系统，他的团队可能会进行细胞基础检测，将 MSC 与免疫细胞混合，以观察它们的反应。</p>
</div><p>“We look at things like cell activation, proliferation, and the release of cytokines or growth factors. We might also test how cells interact with other cell types to show they can activate or suppress specific responses,” he explains.</p>
<div class="lb-trans"><p>“我们关注细胞激活、增殖以及细胞因子或生长因子的释放等方面。我们还可能测试细胞与其他细胞类型的相互作用，以展示它们能够激活或抑制特定反应，” 他解释道。</p>
</div><p>“Because of this, potency testing usually involves more than one assay,” Dr. Jones reiterates. “Some assays focus on cell-to-cell interactions, others measure released factors in the media, and others look at physical characteristics or surface markers on the cells.”</p>
<div class="lb-trans"><p>“因此，效力测试通常涉及不止一种检测，” 琼斯博士重申。“一些检测专注于细胞间相互作用，其他检测测量培养基中释放的因子，还有一些检测关注细胞的物理特性或表面标记。”</p>
</div><p>The exact approach depends on the product, its intended use, and claims being made. The goal is always to connect the assay results to clinical relevance, enabling researchers to prove that the cells are potent and will perform as expected.</p>
<div class="lb-trans"><p>具体的方法取决于产品、其预期用途和所做的声明。目标始终是将检测结果与临床相关性联系起来，使研究人员能够证明细胞是有效的，并将按预期表现。</p>
</div><h2>Potency assay delays</h2>
<div class="lb-trans"><h2>效力检测延迟</h2>
</div><p>“While potency assays are essential, they are rarely a priority for cell and gene therapy developers,” says Dr. Jones. “Developers often deprioritise these assays because their focus is on bringing products to market quickly. As a result, potency assays can feel like a looming challenge in the background.” According to Dr. Jones, the hesitation is largely due to the significant time, effort, and expertise required for their development.</p>
<div class="lb-trans"><p>“虽然效力检测至关重要，但它们在细胞和基因治疗开发者中很少被优先考虑，” 琼斯博士说。“开发者通常会将这些检测放在次要位置，因为他们的重点是快速将产品推向市场。因此，效力检测可能在背景中显得像一个迫在眉睫的挑战。” 根据琼斯博士的说法，这种犹豫主要是由于开发所需的时间、精力和专业知识的显著性。</p>
</div><p>As such, potency assays are often addressed too late in the process, which can stall progress and potentially cause approval delays that stretch on for years. According to Dr. Jones, it’s not unusual for the FDA to request additional data and assays, even when biopharma companies feel their existing potency strategy is sufficient.</p>
<div class="lb-trans"><p>因此，效力检测通常在过程中过晚处理，这可能会阻碍进展，并可能导致批准延迟，延续多年。根据琼斯博士的说法，FDA 要求额外数据和检测并不罕见，即使生物制药公司认为他们现有的效力策略已经足够。</p>
</div><p>The challenge could only heighten as regulators tighten the rules for more robust potency strategies. In 2023, the FDA’s latest update to its guidance on potency assays emphasised the importance of a potency assurance strategy which includes the use of multiple complementary assays measuring potency-related CQAs. This draft could become final in coming years, raising the bar for assay development and validation.</p>
<div class="lb-trans"><p>随着监管机构收紧对更强效力策略的规定，这一挑战可能会加剧。2023 年，FDA 对效力检测的最新指导更新强调了效力保证策略的重要性，其中包括使用多种互补检测来测量与效力相关的 CQA。该草案可能在未来几年内成为最终版本，提高了检测开发和验证的标准。</p>
</div><p>Potency risks are already front and centre in regulatory assessments. Former FDA CBER director Peter Marks has noted that potency-related problems are among the most common reasons for delayed approvals in cell and gene therapies[i], while a study in Europe has estimated that almost half of advanced therapy medicinal product (ATMP) applications in the European Medicines Agency database encountered potency-related issues during assessment,[ii] often resulting in substantial delays.</p>
<div class="lb-trans"><p>效力风险在监管评估中已经成为焦点。前 FDA CBER 主任彼得·马克斯指出，与效力相关的问题是细胞和基因治疗批准延迟的最常见原因之一 [i]，而一项在欧洲的研究估计，几乎一半的先进疗法药物（ATMP）申请在欧洲药品管理局数据库中在评估过程中遇到了与效力相关的问题，[ii]，这通常导致显著的延迟。</p>
</div><p>Such challenges underscore the criticality of starting assay development early. “[Biopharmas] should think about these assays from the very beginning, as soon as they know their indication or mechanisms of action,” notes Dr. Jones.</p>
<div class="lb-trans"><p>这些挑战强调了尽早开始检测开发的重要性。“生物制药公司应该从一开始就考虑这些检测，只要他们知道自己的适应症或作用机制，” 琼斯博士指出。</p>
</div><h2>Start early, partner early</h2>
<div class="lb-trans"><h2>早期开始，早期合作</h2>
</div><p>Building an assay development strategy into early development can pay dividends, enabling companies to accelerate development and improve product consistency starting from preclinical phase.</p>
<div class="lb-trans"><p>将检测开发策略融入早期开发可以带来丰厚的回报，使公司能够加速开发并提高产品一致性，从临床前阶段开始。</p>
</div><p>This can be challenging, however, especially when a product is subject to formulation changes or when MOAs aren’t yet clear. Nevertheless, starting early builds the foundation for regulatory success, allowing developers to obtain earlier feedback from regulators, adjust their strategy accordingly, and test their potency assays in GMP-compliant conditions. Having a robust and reliable potency strategy during clinical trials also safeguards product quality, ensuring each patient receives an effective dose.</p>
<div class="lb-trans"><p>然而，这可能具有挑战性，特别是当产品受到配方变化的影响或当作用机制尚不明确时。尽管如此，早期开始为监管成功奠定了基础，使开发者能够从监管机构获得更早的反馈，相应调整他们的策略，并在符合 GMP 的条件下测试他们的效力检测。在临床试验中拥有强大可靠的效力策略也能保障产品质量，确保每位患者都能获得有效剂量。</p>
</div><p>Early and mid-stage biotech companies often lack the in-depth expertise needed to develop their own potency assays effectively, especially since such knowledge is often underutilised outside critical windows. Partnering with specialists gives companies access to the expertise they need when they need it. This allows developers to stay focused on their product, while experts like Dr. Jones leverage decades of experience in potency assay development to design assay strategies that meet rigorous regulatory standards.</p>
<div class="lb-trans"><p>早期和中期生物技术公司通常缺乏有效开发自身效力检测所需的深入专业知识，尤其是因为这些知识在关键窗口之外往往未被充分利用。与专家合作使公司能够在需要时获得所需的专业知识。这使得开发者能够专注于他们的产品，而像琼斯博士这样的专家则利用数十年的效力检测开发经验，设计符合严格监管标准的检测策略。</p>
</div><p>BBG Advanced Therapies recognises the urgent need for this expertise as the cell therapy industry expands. Based in San Antonio, Texas, the company offers a dedicated group of experienced assay development scientists that create and validate custom potency assays specific to a cell or gene therapy product. BBGAT is committed to helping innovators confidently accelerate their potency assay programs from early development through regulatory approval and also offers complementary services that support companies with starting material collection and processing, testing, clinical trials, and biomanufacturing.</p>
<div class="lb-trans"><p>BBG Advanced Therapies 认识到随着细胞治疗行业的扩展，对这种专业知识的迫切需求。该公司位于德克萨斯州圣安东尼奥，提供一支专门的经验丰富的检测开发科学家团队，创建和验证特定于细胞或基因治疗产品的定制效力检测。BBGAT 致力于帮助创新者自信地加速其效力检测项目，从早期开发到监管批准，并提供支持公司进行起始材料收集和处理、测试、临床试验和生物制造的补充服务。</p>
</div><p>“Working with an experienced potency assay development group like BBG Advanced Therapies will streamline the development process and ensure successful performance of the assays,” says Dr Jones.</p>
<div class="lb-trans"><p>“与像 BBG Advanced Therapies 这样经验丰富的效力检测开发团队合作，将简化开发过程并确保检测的成功表现，” 琼斯博士说。</p>
</div><p>“Our team utilises a variety of analytical platforms such as flow cytometry, ELISA, fluorescence imaging, gel electrophoresis, and nucleic acid quantification, to ensure an optimal fit with our client’s technical capabilities, sensitivity requirements, and desired throughput. We have also developed a proprietary, patent-pending analytical platform to further enhance our ability to deliver high-value solutions for our clients.”</p>
<div class="lb-trans"><p>“我们的团队利用多种分析平台，如流式细胞术、ELISA、荧光成像、凝胶电泳和核酸定量，以确保与客户的技术能力、灵敏度要求和所需通量的最佳契合。我们还开发了一种专有的、正在申请专利的分析平台，以进一步增强我们为客户提供高价值解决方案的能力。”</p>
</div><p>Named Premiva, the novel platform offered by BBG Advanced Therapies can demonstrate how a therapy will function in the body against cancer cells. With just a few drops of therapeutic agent, this customisable dynamic flow-based proprietary platform can provide developers a qualified and validated characterisation of auto-/allo- cells including identity, specificity, and potency.</p>
<div class="lb-trans"><p>BBG Advanced Therapies 提供的名为 Premiva 的新平台可以展示治疗在体内对抗癌细胞的功能。只需几滴治疗剂，这种可定制的动态流动基础专有平台就能为开发者提供对自体/异体细胞的合格和验证的特征描述，包括身份、特异性和效力。</p>
</div><p>In a world where potency assays loom like a dark raincloud over development, success lies in starting early and partnering early. By crafting a robust, regulator-ready strategy from the outset and working with specialists who understand both the science and regulatory body expectations, developers can replace uncertainty with confidence and regulatory holdups with streamlined approvals.</p>
<div class="lb-trans"><p>在效力检测如同乌云笼罩开发的世界中，成功在于尽早开始和提前合作。通过从一开始就制定一个强大的、符合监管要求的策略，并与理解科学和监管机构期望的专家合作，开发者可以用信心取代不确定性，用简化的批准取代监管的拖延。</p>
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</div><p>[i] Solvias. The Potency Puzzle: Overcoming Challenges in Potency Assays for Cell and Gene Therapy Development. [ii] Elsallab, Magdi et al. <em>Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals.</em> Molecular Therapy Methods &amp; Clinical Development, Volume 18, 269 – 279</p>
<div class="lb-trans"><p>[i] Solvias. The Potency Puzzle: Overcoming Challenges in Potency Assays for Cell and Gene Therapy Development. [ii] Elsallab, Magdi et al. <em>Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals.</em> Molecular Therapy Methods &amp; Clinical Development, Volume 18, 269 – 279</p>
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细胞疗法中的效力检测：探讨其背后的挑战