Why A Gene Therapy Pioneer Is Raising Concerns About Treatments He Championed
FEB 7, 2018
Jim Wilson has been one of gene therapy's pioneers. Now he's raising concerns about the safety of a technology he helped develop at least when it is pushed to its limits. In two new studies his team conducted in monkeys, high doses of a certain type of gene were toxic.
"I recognize the fact that these are limited numbers [of animals], and in fact that studies were not designed to evaluate safety," Wilson says in an interview with Forbes, reproduced below. "But the fact is, we observed the toxicity with that limited number, which is, from my point of view, even more worrisome."
link to full story
FEB 7, 2018
Jim Wilson has been one of gene therapy's pioneers. Now he's raising concerns about the safety of a technology he helped develop at least when it is pushed to its limits. In two new studies his team conducted in monkeys, high doses of a certain type of gene were toxic.
"I recognize the fact that these are limited numbers [of animals], and in fact that studies were not designed to evaluate safety," Wilson says in an interview with Forbes, reproduced below. "But the fact is, we observed the toxicity with that limited number, which is, from my point of view, even more worrisome."
We conducted two different animal studies in non-human primates and in pigs to study the efficacy of high-dose intravenous AAV to target the central nervous system. We did not expect to see any safety concerns because of the incredible safety track record that AAV has had.
But what we indeed saw in both studies was that, at very high doses, the animals developed within the first few days after the vector was injected evidence for some liver damage and also activation systemically of inflammation, the consequences of which, was the development of a bleeding disorder or coagulopathy.
But what we indeed saw in both studies was that, at very high doses, the animals developed within the first few days after the vector was injected evidence for some liver damage and also activation systemically of inflammation, the consequences of which, was the development of a bleeding disorder or coagulopathy.
link to full story
Comment