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

Print Friendly, PDF & Email
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:  Video: Alzheimer’s and the disappointing history of amyloid research

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

Outbreak Featured
Infographic: Autoimmune diseases — 76 identified so far — tend to target women over men. Here is a master list

Infographic: Autoimmune diseases — 76 identified so far — tend to target women over men. Here is a master list

There are many autoimmune diseases, and taken together they affect as much as 4.5 percent of the world’s population. This ...
Are GMOs and pesticides threatening bees?

Are GMOs and pesticides threatening bees?

First introduced in 1995, neonicotinoids ...
glp menu logo outlined

Newsletter Subscription

* indicates required
Email Lists
glp menu logo outlined

Get news on human & agricultural genetics and biotechnology delivered to your inbox.