Patient’s ‘serious’ kidney injury halts gene therapy trial for Duchenne muscular dystrophy

maxresdefault
Image: DMD Pathfinders

The Food and Drug Administration has halted a clinical trial involving a Duchenne muscular dystrophy gene therapy from Solid Biosciences (SLDB) after a patient suffered serious kidney and blood-related injuries, the company said [November 12].

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.

Related article:  Start up Nebula Genomics offers free full genome sequencing, but there's a privacy catch

ADVERTISEMENT

All of the toxicities were deemed related to SGT-001 by the patient’s treating doctor. The patient is being treated and is recovering, Solid said.

In a statement, the company said it “will work with the FDA in an effort to resolve the hold and determine next steps” for the clinical trial.

Read full, original post: Solid Biosciences’ Duchenne gene therapy trial halted after patient suffers serious toxicity

ADVERTISEMENT
ADVERTISEMENT
Outbreak Daily Digest
Biotech Facts & Fallacies
Genetics Unzipped
Infographic: Deaths from COVID-19 are far higher than reported estimates

Infographic: Deaths from COVID-19 are far higher than reported estimates

More than 2.8 million people have lost their lives due to the pandemic, according to a Wall Street Journal analysis ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

Optional. Mail on special occasions.
Send this to a friend