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Mammoth’s one-dose CRISPR delivery to muscle raises hopes for wider, safer genetic treatments.

15 May 2025

A single shot to the arm could one day rewrite the DNA in your muscles. That future feels closer after Mammoth Biosciences pulled off a feat that has stumped researchers for years: delivering CRISPR to skeletal muscle through one non-invasive injection.

The milestone, revealed at the American Society of Gene and Cell Therapy’s annual meeting, pushes gene editing past its most comfortable territory. Until now, CRISPR has mostly stayed in the liver, which soaks up treatments with relative ease. Muscle tissue, spread across the body and harder to reach, has been a tougher target.

Mammoth’s approach uses an ultra-compact CRISPR system packed into an adeno-associated virus, a workhorse of genetic delivery. In tests on non-human primates, the method successfully edited muscle genes without surgery, offering a proof of concept for tackling diseases such as Duchenne muscular dystrophy.

“This is the kind of innovation the field’s been waiting for,” said one biotech analyst. “It changes the calculus for how and where CRISPR can go.”

The achievement comes as competition in gene editing heats up. Big players are still hunting for ways to overcome CRISPR’s delivery problem, often called its Achilles’ heel. Mammoth’s success could make it a prime target for partnerships or even acquisition.

Challenges remain. Human trials will be critical to prove safety, lasting effects, and regulatory approval. But the implications are significant. If CRISPR can reach skeletal muscle safely and systemically, it could pave the way for more accessible treatments across a wider range of genetic conditions.

For now, Mammoth’s breakthrough hints at a shift in what gene editing can be: not just precise, but pervasive. The leap from the lab to the clinic is still ahead, but the muscle barrier may finally be cracking.