Biohackers say they are creating a knockoff of a million-dollar gene therapy

Biohackers say they are creating a knockoff of a million-dollar gene therapy
August 30, 2019
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From Biohackers are pirating a cheap version of a million-dollar gene therapy on MIT Technology Review:

The drug being copied is Glybera, a gene therapy that was the world’s most expensive drug when it came on the market in Europe in 2015 with a price tag of $1 million per treatment. Glybera was the first gene therapy ever approved to treat an inherited disease.

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“This was developed in a shed in Mississippi, a warehouse in Florida, a bedroom in Indiana, and on a computer in Austria,” says Gabriel Licina, a biohacker based in South Bend, Indiana. He says the prototype gene therapy cost less than $7,000 to create.

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The gene therapy that the biohackers say they are copying, Glybera, was approved for people with an ultra-rare blood disease called lipoprotein lipase deficiency. But it didn’t prove cost-effective and was pulled from the market in 2017 by its manufacturer, UniQure. To date, only one insurer, in Germany, is known to have paid for the treatment.

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To make their knockoff, the biohackers checked the original Glybera papers for the information about the genetic sequence of the gene that patients require corrected copies of. They placed an order with a gene synthesis company for a copy of the DNA, which was added to a circular genetic construct called a “minicircle.” When added to a cell, the mincircle will begin manufacturing small amounts of the lipoprotein lipase enzyme.

That is an important difference from the original Glybera, which employed an injection of viruses into the leg muscle to deliver the gene. Viral delivery is a complex undertaking, but it is the most commonly used strategy in gene therapy. The biohackers don’t have access to viruses because of their high cost, but they say minicircles can potentially be injected, too.

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As the clinician scientists, we lost control of the project completely ... They decided that they were going to charge a million dollars a shot.