Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
Many new gene therapy treatments aren’t as profitable as predicted. But the reasons why don’t quite fit Goldman Sachs’ warning ...
Pharmaceutical Technology on MSN
The innovators working to make in vivo cell therapy a reality
As in vivo cell therapies enter clinical trials, suppliers tackle the technical and logistical barriers to commercial ...
Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science ...
Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...
There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...
Hemophilia usually is an inherited bleeding disorder in which the blood does not clot properly. Patients with severe hemophilia A may have to manage their condition with regular at-home injections or ...
US Food and Drug Administration (FDA) officials credited recent advancements in regulatory science and flexibilities with ...
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