Can CRISPR Deliver a Once-and-Done Cure?

Early trial results and pharma deals spur renewed confidence in curative gene editing

13 Nov 2025

The US biotechnology sector is gaining momentum as experimental CRISPR-based treatments move closer to real-world application, signalling a potential shift toward one-time genetic cures for chronic conditions.

The latest catalyst came from early clinical data on CTX310®, an investigational therapy from CRISPR Therapeutics designed to edit genes linked to high cholesterol and triglycerides. In preliminary Phase 1 results, a single infusion produced an average reduction of about 49 per cent in LDL cholesterol and 55 per cent in triglycerides.

For a medical field dominated by daily drugs and injections, the possibility of a one-time intervention has captured public and investor attention. “People want solutions they don’t have to manage every day,” said one researcher involved in the study.

CRISPR Therapeutics is now considering a strategic shift from rare-disease targets to more common cardiovascular and metabolic disorders, highlighting the company’s emphasis on safety and durability. The momentum has spread across the industry, with Verve Therapeutics, which is developing a similar in vivo gene-editing approach, recently acquired by a global pharmaceutical group. The deal reflects growing confidence among large drugmakers in the technology’s near-term potential.

Analysts say gene editing’s long-standing promise as a “holy grail” of medicine is now entering a more tangible phase. However, cost and access remain central questions. Payers are exploring new reimbursement models, including multi-year or performance-based contracts, to offset high upfront prices while ensuring affordability.

Meanwhile, companies such as Beam Therapeutics are working to refine manufacturing systems to improve precision and scalability. Industry experts say these advances could determine how quickly one-time therapies reach broader patient populations.

As more trial data emerge, the next few years will test whether gene editing can safely and sustainably deliver lasting benefits. If proven effective, analysts expect the technology to reshape treatment models for chronic disease and redefine the commercial landscape of global biotechnology.