CRISPR Developers Eye Safer Path to the Clinic

Sector turns to next-generation delivery and risk controls as in vivo gene editing moves toward wider clinical use

5 Jun 2025

A serious safety event in Intellia Therapeutics’ late-stage trial is prompting gene-editing companies to refine delivery technologies and strengthen safety oversight as in vivo CRISPR therapies edge closer to commercialisation.

The Massachusetts-based biotech reported a grade 4 enzyme spike in one patient receiving its lipid nanoparticle-delivered CRISPR therapy. The reaction resolved without treatment, but the disclosure sent its shares lower and renewed debate over the safest path to in-body gene editing.

In vivo CRISPR, which edits genes directly inside patients, is viewed as a faster and more scalable alternative to ex vivo approaches that require removing and modifying cells in a laboratory. Intellia’s programme had been regarded as a critical test for the technology’s readiness for mainstream use.

The trial remains on course, with early efficacy data still positive. Analysts say such incidents are part of the learning curve for emerging therapeutic platforms. “Transparency under pressure is how new platforms mature,” said one biotechnology analyst.

Rivals are already adjusting course. Verve Therapeutics is advancing precision delivery for cardiovascular targets, while Editas Medicine is refining its CRISPR tools for blood disorders, both aiming to minimise immune responses and improve targeting.

As the field moves from proof-of-concept to large-scale trials, investors and regulators are increasingly focused on execution: consistent manufacturing, accurate delivery, and robust patient monitoring. Companies are investing in next-generation delivery vehicles and trial designs intended to capture more granular safety data.

Industry observers expect the pace of development to accelerate, with lessons from Intellia’s trial shaping protocols across the sector. If delivery challenges can be addressed, analysts forecast in vivo CRISPR could transition from experimental use to a standard treatment option within the next decade.