The US Food and Drug Administration (FDA) has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.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 finalised, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including Chemistry, Manufacturing and Controls (CMC) data, non-clinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells.

Karim Mikhail, B. Pharm., MS, Acting Director of the Center for Biologics Evaluation and Research (CBER), said, "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. 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This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of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 It also works in tandem with the FDA's recently issued draft guidance, Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing, which recommends methods for evaluating off-target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 This new draft guidance explains how sponsors can use existing public and platform knowledge to streamline regulatory submissions across multiple stages of product development.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.

Vijay Kumar, MD, Acting Director of the Office of Therapeutic Products in CBER, said,?“By outlining how sponsors can intelligently build upon existing non-clinical, 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. 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."

Denise Gavin, PhD, Director of the Office of Therapeutic Products’ Office of Gene Therapy–CMC, said, "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.”

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 finalising the guidance.