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To treat a rare and devastating disease, AveXis biotechnology has developed a drug that can introduce an active gene into the body and fight against the continuous and increasing deterioration of muscle deterioration, characteristic of the disease.
Sound bewildering? Imagine that you are trying to make it.
The specialty of AveXis is this type of "gene therapy", a promising area that could be considered as a long-term treatment, or even as a cure.
But the manufacturing of these complex and very new products also represents a significant challenge for biopharmaceutical companies and threatens to shape the contours of this emerging industry.
Scientific Director Brian Kaspar told Business Insider in an interview with AveXis that the problem had resulted in the company taking control of the company by nearly $ 9 billion by a major pharmaceutical company last year.
"This is one of the reasons that Novartis came and bought us because we understood, and I think it was unique," Kaspar said.
Lily: Billions of dollars are invested in gene therapy startups. Key executives from three companies described the biggest challenge facing the new field.
What a booming field "does not necessarily appreciate"
To make the powerful and accurate genetic modifications needed to treat hereditary diseases, AveXis and other companies use viruses as vectors.
Depending on their condition, some gene therapies target a very specific part of the body, such as the eye.
However, ophthalmic therapy requires far less virus than AveXis Zolgensma, which is injected into the fluid surrounding the spine and brain to treat rare muscular atrophy of the genetic disorder (SMA).
Zolgensma has not yet been approved in the United States, but a decision by the Food and Drug Administration is expected in May.
See:The highest FDA official responsible for monitoring gene therapy had these 5 suggestions for startups flooding the industry with billions of dollars
Companies must use enough of their gene therapies to test them and get enough evidence of their safety and effectiveness. But they must also be able to manufacture on a fairly large scale to treat all patients with the disease who want their treatment.
And changing the process at this point "could change the product," says Kaspar, "something that the field does not necessarily appreciate."
How AveXis did it
Kaspar has worked for a large part of his career on viruses and their use in gene therapy, including 13 years spent researching them at the Nationwide Children's Hospital of Ohio. In 2012, he started at AveXis as a scientific founder.
But these academic works were not quite on the commercial scale that AveXis would need.
The company has hired experts, starting with industry veteran Andrew Knudten, who is now AveXis' senior vice president of global strategic operations.
Knudten worked in the pharmaceutical manufacturing sector, but not in gene therapy developed by AveXis. He put together a team of experienced engineers in a type of complex drugs called biologics (which includes vaccines and many other common drugs), and they worked closely with Kaspar's group.
The combination of different types of science has proven fruitful, Kaspar said. He had been faced with problems such as "how to grow cells at a bigger and longer level" and "I can move 10 liters but this process will use thousands of liters".
"Well, these people said:" It's not a problem, Brian, "Kaspar said. "So, things that we had theoretically almost as a challenge, someone else discovered them, but in a different setting.
But Kaspar does not recommend other companies to follow exactly the same approach. On the one hand, AveXis has not disclosed their process, he said.
And there is no single solution either, according to Kaspar. It depends on the disease that is attacked and the size of the patient population, for example – the technology may not necessarily evolve as part of a rare eye disease.
"So, just because we understood this in one way or one badysis, people have to go out and really develop it themselves," he said. "And there is no ready-made solution for most of these diseases."
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