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The federal government recently awarded millions of dollars in research funding to develop gene therapies for rare diseases, on an ambitious timeline that calls for human trials in three years.
Two of the seven recipients are based in Philadelphia. One of them went to scientists at the Children’s Hospital of Philadelphia to continue their work on gene therapy that they recently used to treat a baby with a rare genetic disorder. The other award was given to GEMMABio, the gene therapy company founded by pioneer Jim Wilson, who had a three-decade-long career at the University of Pennsylvania.
Wilson explained that patients with rare genetic diseases have problems or mutations in their genetic code that cause health problems, and the traditional approach has been to change the patient’s genes. However, many patients with rare diseases have unique mutations, so each patient might need their own, individualized gene therapy.
Wilson and his group will work with an AI gene editing company to develop treatments that need not be unique to each patient.
“What we’re trying to achieve in this program is to find a way to do that efficiently and do it at scale to ensure access,” Wilson said.
He added that he is “delighted” the government decided to fund this research, because the pharmaceutical business will not fund these kinds of innovative treatments for rare diseases. This is where the federal government is stepping in.
“They’re basically filling a gap with the hope that if we show that we can do it and we can do it at scale, then we can pass the baton to more traditional drug developers.”
The award from the government’s Advanced Research Projects Agency for Health will fund Wilson’s research on two diseases: Maple syrup urine disease, a condition where the body cannot break down certain parts of proteins, causing these chemicals to build up in the blood, and requiring a restrictive and special diet; and homozygous familial hypercholesterolemia, a genetic heart condition where cells are less able to remove cholesterol from the blood, which increases the risk of heart disease in childhood.
Right now, these diseases can be treated, but not cured.
Wilson will also tackle the problem of how to get a gene therapy into a patient’s body, which he said “defines 40 years of my career.”
He said that while gene therapy is “the ultimate drug” because it addresses the cause of the problem, it is much larger than other drugs so patients cannot just swallow them. Up until now, he said every gene therapy has used a virus to deliver the treatment to a patient’s body, which works, but the patient’s immune system will also respond to the virus, which makes it hard to deliver the same drug again.
“If that dose was too low or it went away, you’re essentially vaccinated against your own therapy,” he said.
Wilson’s research under this award would use small fat-based molecules called lipid nanoparticles to deliver gene therapies.