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 Tuesday.<\/p>\n
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.<\/p>\n
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.<\/p>\n
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Sarepta Therapeutics (SRPT) and Pfizer (PFE) are also developing their own gene therapies targeted at Duchenne.<\/p>\n
Six patients have been dosed with SGT-001, starting with three at a lower dose; interim results in those patients were previously reported and found to be disappointing. Three more patients were then treated at a higher dose of SGT-001.<\/p>\n
The sixth patient became ill soon after being treated in October, experiencing an over-activation of the immune system, an acute kidney injury, reductions in platelets and red blood cells, and cardio-pulmonary insufficiency, Solid said.<\/p>\n
All of the toxicities were deemed related to SGT-001 by the patients treating doctor. The patient is being treated and is recovering, Solid said.<\/p>\n
Solid reported the patients status to the FDA, which then placed the clinical trial on hold. 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.<\/p>\n
Pfizers Duchenne gene therapy has also been tied to similar immune system over-activation and related kidney toxicity, although its clinical trial remains active.<\/p>\n
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Solid's Duchenne gene therapy trial halted after patient suffers toxicity - STAT<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" 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 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. Continue reading