12:00 AM
Aug 06, 2012
 |  BioCentury  |  Emerging Company Profile

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 a lack of dystrophin protein, which normally acts as a scaffold and shock absorber in muscle fibers.

Becker muscular dystrophy has a slower rate of progression and is less common than DMD.

There are no drugs approved for Becker. Standard of care is corticosteroids, which only delays disease progression in the short term.

According to scientific co-founder Brian Kaspar, adverse immune responses led to the failure of gene therapies that attempted to deliver dystrophin to treat the underlying cause of DMD.


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