Mice Gene-Hacked to Produce Own Ozempic-Like Drugs

Breakthrough offers tantalizing glimpse into future of personalized medicine

Scientists have successfully modified mice, enabling them to generate their own versions of popular GLP-1 medications like Ozempic. This groundbreaking research, published in *Communications Medicine*, could represent a significant shift towards the body producing its own therapeutic compounds.

Liver Cells Engineered for Drug Production

Researchers at Japan’s University of Osaka utilized CRISPR gene-editing technology to introduce a specific gene into the liver cells of obese, pre-diabetic mice. This genetic alteration instructed the cells to produce exenatide, an early diabetes drug and predecessor to current weight-loss injections.

The edited mice demonstrated a notable reduction in food intake and a slower rate of weight gain compared to their unaltered counterparts. This effect was sustained for up to 28 weeks, as detailed in a related press release.

Previous Experiments Pave the Way

This is not the first instance of researchers employing genetic modification to encourage GLP-1 production. In 2017, scientists at the University of Chicago modified human and mouse skin cells to produce GLP-1, subsequently grafting them onto host animals. These subjects exhibited improved insulin secretion and reversed weight gain.

More recently, students from Canada’s University of Ottawa genetically engineered a plant to produce a similar compound, further underscoring the potential of biological systems to create therapeutic agents.

A Radical Departure in Treatment

These combined efforts present a compelling proof of concept for bespoke genetic treatments, potentially transforming how medications are developed and administered. “We hope that our design of a one-time genetic treatment can be applied to many conditions that do not have exact genetic causes,” stated senior author Keiichiro Suzuki.

Acknowledging Existing Concerns

However, not all GLP-1 drugs are created equal. The original exenatide formulation, taken twice daily orally, proved less effective than semaglutide. Both exenatide and its extended-release version have been discontinued due to known risks of pancreatitis. Ozempic has also been linked to this condition, though research remains inconclusive.

It is currently unclear if the Osaka researchers intend to explore compounds closer to semaglutide. Meanwhile, biopharmaceutical company Fractyl Health is also developing a gene therapy aimed at enabling the body to produce its own GLP-1s. This therapy is undergoing the approval process for human trials in Europe, expected to commence next year.

In-Vivo Production and Side Effects

A critical question remains: could producing GLP-1s directly within the body mitigate the risk of pancreatitis and other gastrointestinal issues associated with these drugs? While it’s too early to definitively answer, ongoing research will closely monitor these developments. Recent data indicates that the U.S. saw over 2 million prescriptions for GLP-1 agonists filled in the first three months of 2024, highlighting their widespread use and the importance of understanding their long-term effects (GoodRx, 2024).