The US Food and Drug Administration (FDA) has debuted draft guidance for gene therapy developers, focused on the use of genome editing technologies in non-clinical studies.
This framework, introduced by the FDA’s Center for Biologics Evaluation and Research (CBER), sets out to provide sponsors developing both ex vivo and in vivo genome editing therapies with standardised methods to assess the safety and efficacy of their investigational new drug (IND)-seeking candidates.
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The guidance places a key focus on how next-generation sequencing (NGS) methods can be used to determine and account for potential safety risks associated with off-target gene editing, while providing a framework to approaching sequencing strategies and sample selection. The document also focuses on the optimal parameters for analysis and reporting.
This regulatory framework complements the prior guidance on this topic released by the FDA from January 2024.
According to FDA commissioner, Marty Makary, genome editing holds “extraordinary” promise for treating genetic diseases that were previously incurable, adding that the agency is serious about moving this concept forward to more patients.
To get a temperature check on industry sentiments towards this draft guidance, the FDA has opened the floor to public comments.
The FDA’s guidance debuts amid the backdrop of the agency’s wider focus on rare disease, which has seen it home in on genome editing and RNA-based therapies in the plausible mechanism pathway framework and other policy changes. Experts previously interviewed by Clinical Trials Arena’s sister publication, Pharmaceutical Technology, applauded the FDA’s efforts to shift the regulatory paradigm around rare disease, noting that clarity in this area will likely instil confidence in sponsors and investors.
Genome editing comes to the forefront
In recent years, there has been a growing interest in harnessing genome editing to treat diseases – especially those considered rare or ultra-rare.
One company that has been particularly active in this space is Eli Lilly, which recently outlaid $1.12bn to co-develop and commercialise gene editing therapies for hearing loss with Seamless Therapeutics.
In June 2025, Lilly also landed a deal to acquire gene editing medicines developer, Verve Therapeutics for $1.3bn – which is currently developing drugs designed to address the drivers of atherosclerotic cardiovascular disease.
AstraZeneca has also hedged its bets on the modality through a $555m deal with Algen Biotechnologies, which will see the latter company discover novel therapeutic targets for immunological indications.
According to a report from GlobalData, parent company of Clinical Trials Arena, two-thirds of genome editors use CRISPR technology, which has captured the attention of the pharma industry following the milestone approval of Vertex Pharmaceuticals’ CRISPR/Cas9-based sickle cell disease gene therapy, Casgevy (exagamglogene autotemcel). However, the pipeline is still mostly early-stage, with development focused on high-impact areas like cancer and rare diseases.
