The FDA released draft guidance aimed at helping developers bring promising gene therapies to patients more efficiently by leveraging existing scientific and regulatory knowledge throughout the development process.
The draft guidance, once finalized, will provide recommendations for sponsors developing human gene therapy products that use genome editing in human somatic cells. It outlines how companies can use publicly available information and established platform knowledge—including chemistry, manufacturing and controls (CMC) data, nonclinical study results, and clinical evidence—to streamline regulatory submissions and reduce development burdens.
The new guidance could have a meaningful effect on the clinical development of potential treatments for inherited retinal diseases (IRDs).
"For years, one of the biggest barriers to getting gene therapies approved has been the regulatory pathway. Proving efficacy in rare diseases is difficult when patient populations are small and traditional trial designs simply don't fit," said Jason Menzo, CEO of the Foundation Fighting Blindness. "The FDA's new draft guidance is a meaningful step toward addressing that. By allowing developers to build on existing clinical, manufacturing, and nonclinical knowledge, the agency is signaling that our field has accumulated enough shared scientific foundation to support a more efficient path forward."
"For the IRD community specifically, this shifts the landscape significantly. Decades of patient data, natural history studies, and disease-specific research don’t happen overnight—and they are exactly what the industry needs right now," he added.
The guidance is intended to support a broad range of cell and gene therapy products, including those that utilize genome editing technologies. It forms part of a broader package of FDA initiatives designed to advance the development of innovative therapies.
“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 FDA’s 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.”
According to the agency, the new guidance complements the FDA’s Plausible Mechanism Framework by providing practical approaches for using shared scientific knowledge and data to establish evidence supporting genome editing therapies. It also aligns with the agency’s recently issued draft guidance, "Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing," which outlines recommendations for assessing potential off-target editing risks.
Together, these efforts are intended to provide developers with a clearer, science-based pathway for incorporating prior knowledge into product development while maintaining safeguards to protect patients.
“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, MD, 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,” Dr. Kumar added. “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.”
The FDA noted that sponsors seeking to rely on existing data must provide a scientific rationale demonstrating how the information applies to their specific product and development program. The agency also encourages companies to engage early with regulators, including through Initial Targeted Engagement for Regulatory Advice on CBER/CDER Products (INTERACT) meetings and pre-Investigational New Drug (pre-IND) consultations.
The draft guidance is now available for public comment. Interested stakeholders have 90 days following publication in the Federal Register to submit feedback through Regulations.gov. The FDA said it will review and consider all comments received before issuing a final version of the guidance.
