From birth to gene edicts in 6 months: personalized therapy breaks speed limits

During the boy’s fourth month, the researchers met the Food and Drug Administration to discuss the regulatory approval of a clinical trial – a trial where KJ would be the only participant. They also worked with the Institutional Review Board (IRB) at the Philadelphia children’s hospital to review the clinical protocol, safety and ethical aspects of treatment. The researchers described the unprecedented speed of the surveillance steps as “through alternative procedures”.

In the fifth month, they started toxicological tests in mice. In mice, experimental therapy has corrected KJ’s mutation, replacing the pair of wandering bases with the good GC pair in animal cells. The first dose provided a corrective rate of the entire 42% heporate in animals. At the beginning of the sixth month of KJ, the researchers had safety test results in monkeys: their personalized basic basic therapy, delivered in the form of mRNA via a lipid nanoparticle, produced no toxic effect on the monkeys.

A batch of clinical treatment of treatment has been prepared. During the month seven, additional treatment tests revealed low level of genetic target genetic changes. The researchers submitted FDA documents for the approval of a “new investigation medication” or Ind, for KJ. The FDA approved it in a week. The researchers then started KJ on an immune suppression treatment to ensure that his immune system would not react to genetic publishing therapy. Then, while KJ was only 6 months old, he obtained a first low dose of his personalized gene editing therapy.

“Transformational”

After treatment, he was able to start eating more protein, which would otherwise skyrocket his ammonia levels. But it could not be weaned the drug treatment used to maintain its ammonia levels (nitrogen recovery drug). Without any security concern observed after the first dose, KJ has since obtained two additional doses of gene therapy and is now under a reduced nitrogen recovery medication. With more protein in his diet, he went from 9^th centile in weight at 35^th or 40^th centile. He is now about 9 and a half months old, and his doctors are preparing to allow him to return from the hospital for the first time. Although it will have to be closely monitored and can still need a liver transplant, his family and his doctors have been celebrating improvements so far.