The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...
Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.
Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed ...
In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory ...
Instead of fixing one of the many genes that can be mutated, the technique addresses a type of mutation they share, a stop ...
An experimental gene-editing therapy developed by Crispr Therapeutics is showing promise for treating heart disease.
The FDA will mandate its strictest safety warning on Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, ...
Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.
In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...
Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.
Coave Therapeutics has announced its lead gene therapy program, CoTx-10, for the treatment of retinal vascular diseases, such ...
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