Guidance shows how sponsors can build on existing science, reducing redundant testing and accelerating treatments for patients with rare and life-threatening diseases
The U.S. Food and Drug Administration (FDA) today issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.
When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls (CMC) data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells.
"Today’s action reflects the FDA's commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options," said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research (CBER). “By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on. Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible."
This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area.
For sponsors developing genome editing therapies, it complements the agency's Plausible Mechanism Framework, providing the scientific tools and data-sharing strategies that allow sponsors to efficiently establish the evidentiary foundation this approach requires. It also works in tandem with the FDA's recently issued draft guidance, Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing, which recommends methods for evaluating off-target editing risks. This new draft guidance explains how sponsors can use existing public and platform knowledge to streamline regulatory submissions across multiple stages of product development. Together with the FDA’s other recent actions, it provides developers across the cell and gene therapy field with a clear, science-based path for building on existing knowledge and experience, while maintaining the rigorous standards needed to ensure patient safety.
“By outlining how sponsors can intelligently build upon existing nonclinical, clinical, and manufacturing knowledge, we can meaningfully streamline development programs and lower the cost barriers that have historically slowed access to these potentially life-changing treatments,” said Vijay Kumar M.D., Acting Director of the Office of Therapeutic Products in CBER. “Leveraging prior knowledge does not mean lowering the bar; it means raising our collective efficiency while maintaining the highest standards of safety and efficacy. For patients living with serious or rare diseases, time matters. We encourage developers to engage with this guidance because their perspectives are essential to shaping a regulatory framework that works for everyone, and most importantly, for the patients who are counting on us."
"We look forward to working closely with sponsors to help them understand how to effectively implement this guidance and leverage prior knowledge in their development programs,” added Denise Gavin, Ph.D., Director of the Office of Therapeutic Products’ Office of Gene Therapy - CMC.
In all cases, sponsors should provide a scientific rationale demonstrating the applicability of the data being leveraged to their specific product and development context. The FDA encourages sponsors to engage early in product development, even before submitting an IND application, for example through Initial Targeted Engagement for Regulatory Advice on CBER/CDER Products (INTERACT) and pre-IND meetings, to discuss their specific development strategies.
The draft guidance is available for public comment. Comments must be submitted within 90 days of publication in the Federal Register at Regulations.gov. The agency will review and consider comments received before finalizing the guidance.
An unofficial translation of this announcement is provided below.
翻译参考：
FDA发布指南草案助力加速细胞和基因疗法惠及患者
指南说明申办方如何借助现有科学成果，减少重复测试，加快针对罕见病及危及生命疾病患者的治疗进程
2026年6月2日，美国食品药品监督管理局（FDA）发布指南草案，旨在帮助开发者更高效地将有前景的基因疗法推向患者，充分利用现有科学和监管知识。
该指南一旦定稿，将明确申办方如何利用公开可获取的信息及已建立的平台知识——包括化学、制造和控制（CMC）数据、非临床研究结果及临床信息——来简化人类基因治疗产品（使用人体体细胞基因组编辑技术）的监管申报工作。
生物制品评估与研究中心（CBER）代理主任Karim Mikhail表示："今天的举措体现了FDA致力于更快地将安全有效的细胞和基因疗法送达患者手中，尤其是那些几乎没有或完全没有其他治疗选择的罕见病及危及生命疾病患者。通过提供企业如何借助已有知识的指导，我们在不降低患者和公众所依赖的严格科学标准的前提下加速创新。归根结底，这是为了确保基因疗法的承诺以尽可能快速、安全的方式惠及最需要它的患者。"
该草案指南支持广泛的细胞和基因治疗产品的开发，包括使用基因组编辑技术的产品，是FDA在该领域一系列互补行动的组成部分。
对于开发基因组编辑疗法的申办方，本指南与FDA的合理机制框架相辅相成，提供科学工具和数据共享策略，使申办方能够高效建立该方法所需的证据基础。同时，本指南也与FDA近期发布的草案指南《使用下一代测序技术评估人类基因治疗产品中基因组编辑的安全性》指南草案，后者就评估脱靶编辑风险的方法提出建议。本新草案指南解释了申办方如何利用现有公开知识和平台知识，简化产品开发多个阶段的监管申报工作。结合FDA近期其他举措，本指南为细胞和基因治疗领域的开发者提供了清晰、以科学为基础的路径，以便在借鉴现有知识和经验的同时，维持确保患者安全所需的严格标准。
CBER药物治疗产品办公室代理主任Vijay Kumar博士表示："通过阐明申办方如何智能地借鉴现有非临床、临床和生产知识，我们可以切实简化开发项目，降低历史上阻碍这些潜在变革性治疗手段可及性的成本障碍。利用已有知识并不意味着降低标准，而是在维持最高安全性和有效性标准的同时提升整体效率。对于患有严重或罕见疾病的患者而言，时间至关重要。我们鼓励开发者积极参与本指南的制定，因为他们的观点对于构建一个适用于所有人、尤其是寄望于我们的患者的监管框架不可或缺。"
药物治疗产品办公室基因治疗CMC办公室主任Denise Gavin博士补充道："我们期待与申办方密切合作，帮助他们理解如何在其开发项目中有效实施本指南并充分利用已有知识。"
在所有情况下，申办方均应提供科学依据，证明所借鉴数据对其特定产品和开发背景的适用性。FDA鼓励申办方在产品开发早期——甚至在提交研究性新药（IND）申请之前——就积极寻求沟通，例如通过CBER/CDER产品监管建议初步定向接触（INTERACT）机制及IND前会议，讨论其具体开发策略。
该指南草案现已开放公众评议。评议意见须在《联邦公报》发布后90天内通过Regulations.gov提交。FDA将在定稿前审阅并考虑所收到的意见。
Sincerely,
Sarah McMullen
Country Director
FDA China Office
Office of Global Operations
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