eClinical Technology and Industy News

In a World First, a Patient’s Antibody Cells Were Just Genetically Engineered

B cells are prolific producers of antibodies, but for the first time, scientists have modified them to make other proteins to counteract a serious genetic disease.

Excerpt from the Press Release:

Our B cells help prevent us from getting sick. Their job is to make antibodies, immune system proteins that fight off viruses and other foreign invaders. And they make a lot of antibodies—thousands of them every second. What if these antibody factories could be harnessed to make other things the body needs?

That’s the idea behind a trial launched by Seattle-based biotech company Immusoft. The company announced today that its scientists have genetically programmed a patient’s B cells and put them back in his body in an effort to treat disease. It’s the first time engineered B cells have been tested in a person.

The patient has a genetic disorder known as mucopolysaccharidosis type I, or MPS I. His body doesn’t produce an essential enzyme that helps to break down long-chain sugars inside cells. Without this enzyme, these sugars build up in the eyes, heart, bones, and elsewhere.

The effects are life-threatening. Patients have cloudy eyes, respiratory problems, cognitive issues, and enlarged organs. Those with the most severe form of the disease die in childhood. Others may live to their twenties or thirties.

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