The FDA’s genome editing guidance explains when developers of genome editing therapies may use public science or platform experience to reduce the need for repeated testing.
The FDA has issued new draft guidance that could help some cell and gene therapy developers move through the development process more efficiently by using reliable scientific information that already exists.
The guidance focuses on human gene therapies that use genome editing in somatic cells, with edits made either outside the body or inside the body. Somatic cells are not reproductive cells, so these approaches are not intended to pass genetic changes to future generations.
The FDA said this could be especially helpful for therapies being developed for rare, serious or life-threatening diseases, where patients may have few or no treatment options.
What Counts as Existing Knowledge?
The draft guidance describes two types of existing information. The first is public knowledge, such as peer-reviewed research, regulatory guidance and published standards; and the second is platform knowledge, which comes from developing or manufacturing similar products or using similar technologies.
For example, a company may have experience with a repeatable manufacturing process, delivery system or testing method that is used across more than one gene therapy program. In some cases, that experience may help support a new therapy instead of requiring the company to start every test from scratch.
The FDA is not saying that older information can be reused automatically. Developers would still need to explain why the information applies to the specific therapy they are developing. If the new product differs in important ways, the company may need additional testing or bridging data.
Where This Could Reduce Repeated Work
One major area is manufacturing and product quality. This includes the tests used to confirm that a therapy is made consistently, meets quality standards and remains stable over time. If two products are similar enough, a developer may be able to rely partly on previous experience with testing methods, stability data or manufacturing processes.
The guidance also discusses nonclinical studies, which are studies done before or alongside human trials to help assess potential safety risks. In some situations, developers may be able to use lab data, computer-based methods, animal data or information from related products to shape the amount and type of new testing needed.
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One key safety issue is off-target editing, which happens when a gene-editing tool makes changes in places that were not intended. The FDA says some testing methods and bioinformatics tools may be used across similar products, but the actual results may still need to be specific to each therapy and editing target.
Clinical experience from related products may also help guide new trials. Prior data could help inform dose selection, monitoring plans, safety follow-up or other parts of trial design. The relevance of those data depends on factors such as the disease, patient population, route of administration and similarity of the therapy.
Early FDA Discussions Are Encouraged
The FDA encourages developers to discuss any strategy for using existing public or platform knowledge early, including before submitting an investigational new drug (IND) application. These early meetings can help clarify whether a company’s plan to use existing information is scientifically reasonable and what additional data may still be needed.
The draft guidance is open for public comment and is not legally binding. If finalized, it would describe the FDA’s current thinking on how companies can use existing scientific and regulatory knowledge to support gene therapy development.
For developers, the guidance offers a clearer way to build on what is already known. For patients with rare or serious diseases, the goal is to help promising therapies move forward more efficiently while keeping product-specific safety and effectiveness standards in place.
Genome Editing: FDA Oversight in the Spotlight
Recent genome editing programs are moving from clinical development toward FDA review. For example, Intellia Therapeutics has begun a rolling biologics license application (BLA) submission for lonvoguran ziclumeran, an investigational CRISPR therapy that works directly inside the body to treat hereditary angioedema, a rare condition that can cause severe swelling attacks.
Safety remains the top priority. Earlier this year, the FDA lifted clinical holds on Intellia’s nexiguran ziclumeran Phase III programs after the company added safety measures, including enhanced liver monitoring and updated exclusion criteria.
Beam Therapeutics is also advancing a genome editing program. The company said it has aligned with the FDA on a potential accelerated approval path for BEAM-302, its investigational base editing treatment for alpha-1 antitrypsin deficiency (AATD), a genetic lung and liver disease. The program has also been accepted into an FDA pilot designed to support manufacturing readiness for products moving through expedited development.
FAQs
What does “prior knowledge” mean in the FDA’s genome editing guidance?
Prior knowledge is existing scientific information, such as published studies or data from similar therapies, that developers may use to support a new genome-editing treatment.
Does using prior knowledge mean that a gene therapy can skip safety testing?
No. Developers must still show that a therapy is safe and explain why any existing information is relevant to their specific product.
Why is the FDA releasing multiple genome editing guidances?
Each guidance addresses a different part of genome editing development. The prior-knowledge draft guidance focuses on when existing data may be used. The next-generation sequencing guidance focuses on safety testing for unintended edits. The Plausible Mechanism Framework applies to individualized therapies for rare genetic conditions.
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