<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·瓊斯博士在檢測開發領域工作了超過 35 年，目前領導 BioBridge Global 的研發部門。他的團隊與 BBG 的子公司 BBG 先進療法（BBGAT）緊密合作，開發和驗證快速、高靈敏度、符合 GMP 標準的細胞和基因治療製造商的效力檢測。</p>
<p>效力檢測是對產品生物功能的定量測量，包括細胞殺傷、免疫調節、傷口癒合、趨化性、血管生成或分化。它們在產品的整個生命週期中都是必需的，從初始開發和臨牀製造到監管批准和持續商業供應。</p>
<p>“對於細胞和基因治療，展示產品仍然能夠執行其應有的生物功能是非常重要的，” 瓊斯博士説。“儘管有明確的製造過程，但這些是活細胞，受到許多變量的影響，這些變量可能會影響效力。”</p>
<p>瓊斯博士解釋説，效力檢測在開發過程中被廣泛使用，包括產品穩定性評估、可比性研究和製造過程研究。在監管申請期間，效力檢測必須證明治療能夠持續產生預期效果，為監管機構提供產品強度和功能活性的證據。</p>
<p>在 GMP 製造過程中，效力檢測是質量控制的一個重要方面，確認每一批次在治療釋放之前保持預期的生物活性。除此之外，瓊斯博士解釋説，它們還用於評估引入新原材料、生產過程或設施的任何影響。</p>
<h2>將檢測與作用機制匹配</h2>
<p>“由於效力檢測測量產品實現預期治療效果的能力，因此它們必須是特定於產品的，” 瓊斯博士説。“在 BBG 先進療法，我們開發與作用機制（MOA）和產品特定適應症相一致的定製效力檢測。”</p>
<p>瓊斯博士解釋説，細胞和基因治療通常具有多個與效力相關的關鍵質量屬性（CQA），這意味着需要多種不同的效力檢測。如果間充質幹細胞（MSC）治療的部分功能是調節免疫系統，他的團隊可能會進行細胞基礎檢測，將 MSC 與免疫細胞混合，以觀察它們的反應。</p>
<p>“我們關注細胞激活、增殖以及細胞因子或生長因子的釋放等方面。我們還可能測試細胞與其他細胞類型的相互作用，以展示它們能夠激活或抑制特定反應，” 他解釋道。</p>
<p>“因此，效力測試通常涉及不止一種檢測，” 瓊斯博士重申。“一些檢測專注於細胞間相互作用，其他檢測測量培養基中釋放的因子，還有一些檢測關注細胞的物理特性或表面標記。”</p>
<p>具體的方法取決於產品、其預期用途和所做的聲明。目標始終是將檢測結果與臨牀相關性聯繫起來，使研究人員能夠證明細胞是有效的，並將按預期表現。</p>
<h2>效力檢測延遲</h2>
<p>“雖然效力檢測至關重要，但它們在細胞和基因治療開發者中很少被優先考慮，” 瓊斯博士説。“開發者通常會將這些檢測放在次要位置，因為他們的重點是快速將產品推向市場。因此，效力檢測可能在背景中顯得像一個迫在眉睫的挑戰。” 根據瓊斯博士的説法，這種猶豫主要是由於開發所需的時間、精力和專業知識的顯著性。</p>
<p>因此，效力檢測通常在過程中過晚處理，這可能會阻礙進展，並可能導致批准延遲，延續多年。根據瓊斯博士的説法，FDA 要求額外數據和檢測並不罕見，即使生物製藥公司認為他們現有的效力策略已經足夠。</p>
<p>隨着監管機構收緊對更強效力策略的規定，這一挑戰可能會加劇。2023 年，FDA 對效力檢測的最新指導更新強調了效力保證策略的重要性，其中包括使用多種互補檢測來測量與效力相關的 CQA。該草案可能在未來幾年內成為最終版本，提高了檢測開發和驗證的標準。</p>
<p>效力風險在監管評估中已經成為焦點。前 FDA CBER 主任彼得·馬克斯指出，與效力相關的問題是細胞和基因治療批准延遲的最常見原因之一 [i]，而一項在歐洲的研究估計，幾乎一半的先進療法藥物（ATMP）申請在歐洲藥品管理局數據庫中在評估過程中遇到了與效力相關的問題，[ii]，這通常導致顯著的延遲。</p>
<p>這些挑戰強調了儘早開始檢測開發的重要性。“生物製藥公司應該從一開始就考慮這些檢測，只要他們知道自己的適應症或作用機制，” 瓊斯博士指出。</p>
<h2>早期開始，早期合作</h2>
<p>將檢測開發策略融入早期開發可以帶來豐厚的回報，使公司能夠加速開發並提高產品一致性，從臨牀前階段開始。</p>
<p>然而，這可能具有挑戰性，特別是當產品受到配方變化的影響或當作用機制尚不明確時。儘管如此，早期開始為監管成功奠定了基礎，使開發者能夠從監管機構獲得更早的反饋，相應調整他們的策略，並在符合 GMP 的條件下測試他們的效力檢測。在臨牀試驗中擁有強大可靠的效力策略也能保障產品質量，確保每位患者都能獲得有效劑量。</p>
<p>早期和中期生物技術公司通常缺乏有效開發自身效力檢測所需的深入專業知識，尤其是因為這些知識在關鍵窗口之外往往未被充分利用。與專家合作使公司能夠在需要時獲得所需的專業知識。這使得開發者能夠專注於他們的產品，而像瓊斯博士這樣的專家則利用數十年的效力檢測開發經驗，設計符合嚴格監管標準的檢測策略。</p>
<p>BBG Advanced Therapies 認識到隨着細胞治療行業的擴展，對這種專業知識的迫切需求。該公司位於德克薩斯州聖安東尼奧，提供一支專門的經驗豐富的檢測開發科學家團隊，創建和驗證特定於細胞或基因治療產品的定製效力檢測。BBGAT 致力於幫助創新者自信地加速其效力檢測項目，從早期開發到監管批准，並提供支持公司進行起始材料收集和處理、測試、臨牀試驗和生物製造的補充服務。</p>
<p>“與像 BBG Advanced Therapies 這樣經驗豐富的效力檢測開發團隊合作，將簡化開發過程並確保檢測的成功表現，” 瓊斯博士説。</p>
<p>“我們的團隊利用多種分析平台，如流式細胞術、ELISA、熒光成像、凝膠電泳和核酸定量，以確保與客户的技術能力、靈敏度要求和所需通量的最佳契合。我們還開發了一種專有的、正在申請專利的分析平台，以進一步增強我們為客户提供高價值解決方案的能力。”</p>
<p>BBG Advanced Therapies 提供的名為 Premiva 的新平台可以展示治療在體內對抗癌細胞的功能。只需幾滴治療劑，這種可定製的動態流動基礎專有平台就能為開發者提供對自體/異體細胞的合格和驗證的特徵描述，包括身份、特異性和效力。</p>
<p>在效力檢測如同烏雲籠罩開發的世界中，成功在於儘早開始和提前合作。通過從一開始就制定一個強大的、符合監管要求的策略，並與理解科學和監管機構期望的專家合作，開發者可以用信心取代不確定性，用簡化的批准取代監管的拖延。</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>來自 BioBridge Global 的 Scott Jones 博士討論了為細胞和基因療法開發效力檢測的挑戰。這些檢測對於展示產品在整個生命週期中的生物功能至關重要，從開發到監管批准。儘管它們的重要性，開發者往往將其優先級降低，導致市場進入的潛在延遲。FDA 更新的指導強調了需要一個強有力的效力保證策略，突出了在監管評估中與效力相關問題的風險。Jones 提倡在產品生命週期中儘早整合檢測開發，以減輕這些挑戰</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·瓊斯博士在檢測開發領域工作了超過 35 年，目前領導 BioBridge Global 的研發部門。他的團隊與 BBG 的子公司 BBG 先進療法（BBGAT）緊密合作，開發和驗證快速、高靈敏度、符合 GMP 標準的細胞和基因治療製造商的效力檢測。</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>效力檢測是對產品生物功能的定量測量，包括細胞殺傷、免疫調節、傷口癒合、趨化性、血管生成或分化。它們在產品的整個生命週期中都是必需的，從初始開發和臨牀製造到監管批准和持續商業供應。</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>“對於細胞和基因治療，展示產品仍然能夠執行其應有的生物功能是非常重要的，” 瓊斯博士説。“儘管有明確的製造過程，但這些是活細胞，受到許多變量的影響，這些變量可能會影響效力。”</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>瓊斯博士解釋説，效力檢測在開發過程中被廣泛使用，包括產品穩定性評估、可比性研究和製造過程研究。在監管申請期間，效力檢測必須證明治療能夠持續產生預期效果，為監管機構提供產品強度和功能活性的證據。</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 製造過程中，效力檢測是質量控制的一個重要方面，確認每一批次在治療釋放之前保持預期的生物活性。除此之外，瓊斯博士解釋説，它們還用於評估引入新原材料、生產過程或設施的任何影響。</p>
</div><h2>Matching assay to Mechanisms of Action</h2>
<div class="lb-trans"><h2>將檢測與作用機制匹配</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>“由於效力檢測測量產品實現預期治療效果的能力，因此它們必須是特定於產品的，” 瓊斯博士説。“在 BBG 先進療法，我們開發與作用機制（MOA）和產品特定適應症相一致的定製效力檢測。”</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>瓊斯博士解釋説，細胞和基因治療通常具有多個與效力相關的關鍵質量屬性（CQA），這意味着需要多種不同的效力檢測。如果間充質幹細胞（MSC）治療的部分功能是調節免疫系統，他的團隊可能會進行細胞基礎檢測，將 MSC 與免疫細胞混合，以觀察它們的反應。</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>“我們關注細胞激活、增殖以及細胞因子或生長因子的釋放等方面。我們還可能測試細胞與其他細胞類型的相互作用，以展示它們能夠激活或抑制特定反應，” 他解釋道。</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，效力測試通常涉及不止一種檢測，” 瓊斯博士重申。“一些檢測專注於細胞間相互作用，其他檢測測量培養基中釋放的因子，還有一些檢測關注細胞的物理特性或表面標記。”</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>具體的方法取決於產品、其預期用途和所做的聲明。目標始終是將檢測結果與臨牀相關性聯繫起來，使研究人員能夠證明細胞是有效的，並將按預期表現。</p>
</div><h2>Potency assay delays</h2>
<div class="lb-trans"><h2>效力檢測延遲</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>“雖然效力檢測至關重要，但它們在細胞和基因治療開發者中很少被優先考慮，” 瓊斯博士説。“開發者通常會將這些檢測放在次要位置，因為他們的重點是快速將產品推向市場。因此，效力檢測可能在背景中顯得像一個迫在眉睫的挑戰。” 根據瓊斯博士的説法，這種猶豫主要是由於開發所需的時間、精力和專業知識的顯著性。</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，效力檢測通常在過程中過晚處理，這可能會阻礙進展，並可能導致批准延遲，延續多年。根據瓊斯博士的説法，FDA 要求額外數據和檢測並不罕見，即使生物製藥公司認為他們現有的效力策略已經足夠。</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>隨着監管機構收緊對更強效力策略的規定，這一挑戰可能會加劇。2023 年，FDA 對效力檢測的最新指導更新強調了效力保證策略的重要性，其中包括使用多種互補檢測來測量與效力相關的 CQA。該草案可能在未來幾年內成為最終版本，提高了檢測開發和驗證的標準。</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>效力風險在監管評估中已經成為焦點。前 FDA CBER 主任彼得·馬克斯指出，與效力相關的問題是細胞和基因治療批准延遲的最常見原因之一 [i]，而一項在歐洲的研究估計，幾乎一半的先進療法藥物（ATMP）申請在歐洲藥品管理局數據庫中在評估過程中遇到了與效力相關的問題，[ii]，這通常導致顯著的延遲。</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>這些挑戰強調了儘早開始檢測開發的重要性。“生物製藥公司應該從一開始就考慮這些檢測，只要他們知道自己的適應症或作用機制，” 瓊斯博士指出。</p>
</div><h2>Start early, partner early</h2>
<div class="lb-trans"><h2>早期開始，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>將檢測開發策略融入早期開發可以帶來豐厚的回報，使公司能夠加速開發並提高產品一致性，從臨牀前階段開始。</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，這可能具有挑戰性，特別是當產品受到配方變化的影響或當作用機制尚不明確時。儘管如此，早期開始為監管成功奠定了基礎，使開發者能夠從監管機構獲得更早的反饋，相應調整他們的策略，並在符合 GMP 的條件下測試他們的效力檢測。在臨牀試驗中擁有強大可靠的效力策略也能保障產品質量，確保每位患者都能獲得有效劑量。</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>早期和中期生物技術公司通常缺乏有效開發自身效力檢測所需的深入專業知識，尤其是因為這些知識在關鍵窗口之外往往未被充分利用。與專家合作使公司能夠在需要時獲得所需的專業知識。這使得開發者能夠專注於他們的產品，而像瓊斯博士這樣的專家則利用數十年的效力檢測開發經驗，設計符合嚴格監管標準的檢測策略。</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 認識到隨着細胞治療行業的擴展，對這種專業知識的迫切需求。該公司位於德克薩斯州聖安東尼奧，提供一支專門的經驗豐富的檢測開發科學家團隊，創建和驗證特定於細胞或基因治療產品的定製效力檢測。BBGAT 致力於幫助創新者自信地加速其效力檢測項目，從早期開發到監管批准，並提供支持公司進行起始材料收集和處理、測試、臨牀試驗和生物製造的補充服務。</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>“與像 BBG Advanced Therapies 這樣經驗豐富的效力檢測開發團隊合作，將簡化開發過程並確保檢測的成功表現，” 瓊斯博士説。</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>“我們的團隊利用多種分析平台，如流式細胞術、ELISA、熒光成像、凝膠電泳和核酸定量，以確保與客户的技術能力、靈敏度要求和所需通量的最佳契合。我們還開發了一種專有的、正在申請專利的分析平台，以進一步增強我們為客户提供高價值解決方案的能力。”</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 提供的名為 Premiva 的新平台可以展示治療在體內對抗癌細胞的功能。只需幾滴治療劑，這種可定製的動態流動基礎專有平台就能為開發者提供對自體/異體細胞的合格和驗證的特徵描述，包括身份、特異性和效力。</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>在效力檢測如同烏雲籠罩開發的世界中，成功在於儘早開始和提前合作。通過從一開始就制定一個強大的、符合監管要求的策略，並與理解科學和監管機構期望的專家合作，開發者可以用信心取代不確定性，用簡化的批准取代監管的拖延。</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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細胞療法中的效力檢測：探討其背後的挑戰