For those who might be interested, I
posted today on the NEU forum regarding the latest setback for gene therapy.
Sarepta has announced the second death of a patient this year from acute liver failure following gene therapy treatment.
Sarepta, which is best known as a leader in PPMO antisense therapies, has had recent commercial success with its first approved gene therapy, Elevidys. Elevidys, which is indicated for the treatment of both ambulatory and non-ambulatory DMD, received accelerated approval from the FDA in 2023.
There have now been a dozen gene-therapy related deaths over the last three years which have occurred during either clinical trials or following marketing approval.
(20min delay)