Milo: Dismissing dystrophin

Milo using follistatin, not dystrophin, in muscular dystrophy gene therapy

Milo Biotechnology LLC is developing a gene therapy to treat muscular dystrophy that up-regulates follistatin, a protein already present in the body, which could avoid immune reactions that have scuttled previous gene therapies.

The company's only candidate is AAV1-FS, which uses an adeno-associated virus vector to deliver follistatin. It is in Phase I/II testing for Becker muscular dystrophy and inclusion body myositis.

Like Duchenne muscular dystrophy, Becker is an X chromosome-linked disease arising from a mutation in the dystrophin gene. The mutation results in

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