A new gene therapy for Duchenne muscular dystrophy (DMD) has barely squeaked by an FDA advisory panel, prompting researchers to work on improvements.
when given this gene. Sarepta and three other companies, Pfizer , Solid Biosciences, and Généthon, went on to test delivering a micro- or minidystrophin gene into the muscles of young boys.
Jessica Curran says this appears to be happening to Conner: Last fall, the sixth grader began using an electric scooter to conserve his strength in long school hallways. UCLA neurologist Perry Shieh, who is seeing declines in DMD patients he’s treated in the Sarepta, Pfizer, and Solid trials, says, “The parents are asking: ‘What is next for my child?’”
“There are many overlapping, complementary strategies” that would allow AAV redosing, Byrne says. His group will soon launch a small study to see whether AAV antibodies can be lowered enough in Conner Curran and other DMD gene therapy patients to potentially allow them to be retreated. Although these immunosuppressive approaches carry their own risks, “compared to the consequences of disease … we feel it’s justified,” Byrne says.
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