According to a recent article in Archive Today, an international band of amateur biologists, or “biohackers” announced that they intend to eventually market a knock-off of Glybera, a one million dollar per dose gene therapy. Glybera was the first gene therapy approved by the FDA to treat an inherited disease.
In 2015, when it was introduced in Europe, Glybera was the world’s most expensive drug therapy. Now Zolgensma carries that title at a cost of $2.1 million for a one-time treatment of spinal muscular atrophy.
The prototype of the knockoff has humble beginnings as it was partially developed in:
- A Mississippi shed
- A Florida warehouse
- An Indiana bedroom
- On a computer in Austria
- At a cost of under $7,000
The next step is to enlist the help of corporate scientists and universities in order to test the drug on animals.
The Biohack the Planet Conference
An eclectic group of journalists, scientists and researchers met in Las Vegas this week. The presentations included biosafety, body implants and hallucinogens. The biohackers presented their material at the conference.
Experts are Divided
Some experts who have knowledge of the project feel that it is not likely to work. Proponents point to the excessive cost of certain genetic treatments that limit access and impact.
A Canadian doctor specializing in LPLD saw nineteen of his patients treated with Glybera in clinical trials. His opinion of the biohackers’ project is that it is well beyond an amateur’s range of expertise as well as posing a substantial risk.
A Washington University associate law professor posits that the developers of the knockoff might believe that they are serving a good cause.
Glybera’s Brief History
Glybera was FDA approved for a rare blood disease called lipoprotein lipase deficiency (LPLD). As of this writing, an insurer in Germany is the only provider known to have covered the treatment.
LPLD is primarily a childhood disease that causes excessive fat to occur in the blood. Absent any therapy for the disease, patients must adhere to a strict diet or they will experience excruciating pain resulting from pancreatitis. A number of female LPLD patients cannot have children.
UniQure, the manufacturer of Glybera, pulled the drug from the market in 2017. Since that time patients have had very few options.
About the Biohackers
Andreas Sturmer is a biotechnologist in Linz, Austria who had the idea of using the concept of reverse engineering to copy the drug. The concept involves reproducing another manufacturer’s product after a detailed analysis of its composition. Sturmer took his concept to Licina.
Gabriel Licina and the team decided to call their genetic invention “Slybera.” Their intent is to be “taken seriously,” although they readily admit that they need help both financial and otherwise in order to test their concept on animals.
The third party in the group included David Ishee who is a biohacker in Mississippi. The group communicated by messaging on Facebook and making calls through Skype.
About the Process
Before the biohackers were able to produce the corrected copies of genes that are needed by patients, they had to study the authentic Glybera papers to learn more about the genetic sequence.
The group contacted a gene synthesis company and placed an order for a copy of the DNA that they added to a “minicircle”. Once it is added to a cell, a minicircle begins to manufacture lipoprotein lipase enzyme in small amounts.
The Original Glybera vs. Minicircles
Glybera delivers the gene by injecting viruses into the leg muscle. Although complicated, viral delivery is the most common strategy used in gene therapy.
Due to the high cost of viruses, the biohackers must use minicircles as they have the potential to be injected as well.
In comparison to Glybera, Ishee describes the minicircles that he designed as being less efficient. He said the injections of the knockoff drug could be given over a period of one year.
Ishee explained the difference between the original Glybera and the minicircles by using an analogy of constructing a swimming pool. He said that you can use a backhoe and it would take a day or you could use a shovel and there would be no cost but it would take several months.
A gene-therapy expert in production commented that the minicircle technology has exhibited mixed results. The advantage is that they might possibly be reused but they may have less efficacy when it comes to controlling cells so that they follow genetic instructions.
Is It Legal?
An intellectual property spokesman at UniQure stated that the company’s patent is still in force. Therefore selling the drug may be an infringement on UniQure’s intellectual property and the knockoff would face stringent regulations.
Yet David Ishee took a cavalier attitude saying that he was not concerned about regulations because he was not selling the product.
Other biohackers are not at all concerned about using published papers (even if patented) to search for information.
Gabriel Licina indicated that at this moment Slybera is in its early stages. He said that it is not ready for testing on humans and needs further development.