Fractyl Health Developing a One-Time Gene Therapy To Treat Type 2 Diabetes

A one-time treatment for type 2 diabetes is already in the works. Biotech company Fractyl Health is working on a gene therapy as an alternative to drugs like Ozempic and Wegovy.

Gene Therapy for Type 2 Diabetes

Fractyl Health is working on a one-time type 2 diabetes medication, which may also be used to manage weight. The Lexington, Massachusetts-based biotech startup is in the early phases of creating a single-use gene treatment that will work similarly to semaglutide to reduce blood sugar and body weight.

According to Harith Rajagopalan, co-founder and CEO of Fractyl Health and a trained cardiologist, you have the issue of needing to continue therapy for it to be effective. That is the weakness of current treatments like Ozempic and Wegovy.

Both have to be injected weekly to imitate the human hormone GLP-1, which is secreted in the gut in response to eating. One of GLP-1's functions is to signal the pancreas to produce insulin, which controls blood sugar. The pancreas either doesn't generate enough insulin or produces too little in those with type 2 diabetes. Blood sugar levels rise as a result, which over time may permanently harm the kidneys, blood vessels, nerves, and eyes.

However, the medications are expensive and can have unpleasant side effects like nausea, diarrhea, and constipation, which might lead some patients to discontinue taking them. Others can forget to take a dose or decide to stop taking the medication once they've achieved their weight loss objectives.

The blood sugar levels rise, the appetite returns, and frequently the pounds also do when these therapies are stopped.

With a one-and-done remedy that will persist for years, Rajagopalan seeks to solve the problems from current treatments. It seeks to eliminate the requirement for weekly injections by implanting a gene into the pancreas that continuously produces the GLP-1 hormone. Inactivated viruses are used in the method known as gene therapy to deliver a therapeutic gene to pancreatic cells.

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How Does Gene Therapy for Type 2 Diabetes Work?

The one-time therapy by Rajagopalan is made to last for many years. The business seeks to eliminate the requirement for weekly injections by implanting a gene into the pancreas that continually generates the GLP-1 hormone. Inactivated viruses are used in the method known as gene therapy, to deliver a therapeutic gene to pancreatic cells. The utilization of viruses stems from their inherent capacity to transfer genetic material to cells.

Only animals have been used by Fractyl to test this method. The company's researchers were interested in how well the experimental gene treatment could lower fasting blood sugar-a marker used to detect diabetes. They used mice designed to develop type 2 diabetes and administered semaglutide injections on a weekly basis to one group while administering a single infusion of gene therapy to the other.

They discovered that, after 10 weeks, the gene therapy reduced fasting blood sugar by 70%, just a little bit more than semaglutide, which cut blood sugar by 64%.

At the American Diabetes Association conference at the end of June, business scientists presented the findings together with other research showing that the medication significantly lowered body weight in mice by 23% when compared to control animals.

Rajagopalan claims he was surprised by the weight drop. The thighs, waist, or upper arm are the areas where Ozempic and Wegovy are injected. It then moves into the bloodstream and interacts in some way with the brain. Since the pancreas receives gene therapy from Fractyl, business scientists did not anticipate a considerable reduction in body weight.

According to Daniel Drucker, an endocrinologist and professor of medicine at the University of Toronto, one reason is that the gene therapy is causing the pancreas to produce enough GLP-1 that some of it are getting into the bloodstream and communicating with the brain. He suggests that there may also be an unidentified signaling system in the pancreas that instructs the brain to cease eating.

However, Ducker is hesitant to provide treatment intravenously to a human pancreas. He explained that the pancreas is a delicate but crucial organ, and inflammation may result if it is poked or prodded.