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Emerging Company Profile
Milo using follistatin, not dystrophin, in muscular dystrophy gene therapy
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Milo: Dismissing dystrophin
Monday, August 6, 2012
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
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