The Food and Drug Administration has halted a clinical trial involving a Duchenne muscular dystrophy gene therapy from Solid Biosciences after a patient suffered serious kidney and blood-related injuries, the company said Tuesday.
This is the third time that the Cambridge, Mass.-based Solid has run into a serious safety problem with its gene therapy, called SGT-001. The FDA placed similar clinical holds on the same clinical trial after each prior incident, but later allowed the company to proceed with patient dosing.
SGT-001 uses an inactivated virus to deliver a miniaturized but functional version of the dystrophin gene to muscle cells. The gene therapy is designed to be a one-time and potentially curative treatment for all Duchenne patients, regardless of the mutation that causes their disease.
About the Author Reprints
I would like to be part of the HIV/AIDS treatment campaign. I would appreciate an update on new drugs discovery leading to the final cure of the pandemic. Trust you find the above in order, regards – Zanele