AAV vectors are well-suited to gene therapies for DMD because they are effective at ... US has been delayed in the US by an FDA request for potency, but is already underway elsewhere, including ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is ... how various routes of adeno-associated virus (AAV) gene therapy ...

This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. Gene therapies have set a high bar for outcomes in ...

Besides, the aforementioned research, along with the rational design studies, further corroborates the notion that antigenic, tropism and potency determinants overlap in the structural context of the ...

Sarepta is currently testing SRP-9001 in the placebo-controlled EMBARK study, which aims to enrol 120 boys with DMD aged four to seven and is due to read out in the latter half of 2023.

About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV ... or changes in the DMD gene that result in the ...