Thursday, August 9, 2012
Researchers at the University of Rochester and Isis Pharmaceuticals Inc. have come up with an efficient way to treat symptoms of
myotonic dystrophy type 1 in mice using systemically delivered antisense
oligonucleotides.1 The finding has fueled an early stage development
deal between Isis and Biogen Idec Inc.
Bennett and Thornton first ran cell
culture screens for ASOs that knocked down the expression of a model transcript
bearing the DM1-associated trinucleotide repeats. The most potent hits were
then tested in a mouse model expressing a model transcript with the
DM1-associated trinucleotide repeats.
Dollars and antisense
In June, Isis granted Biogen rights to ASO
therapeutics for DM1 in exchange for $12 million up front and up to $59 million
in preclinical and clinical milestones.
Osherovich, L. SciBX 5(31);
Published online Aug. 9, 2012
1. Wheeler, T.M. et al. Nature; published online Aug. 2, 2012;
Contact: Charles Thornton, University of Rochester, Rochester,
2. Muntoni, F. & Wood,
Rev. Drug Discov. 10, 621-637 (2011)
3. Wheeler, T.M. et al. Science 325, 336-339 (2009)
4. Mulders, S.A.M. et al. Proc. Natl.
Acad. Sci. USA 106, 13915-13920 (2009)
AND INSTITUTIONS MENTIONED
Biogen Idec Inc. (NASDAQ:BIIB), Weston, Mass.
Isis Pharmaceuticals Inc. (NASDAQ:ISIS), Carlsbad, Calif.
Prosensa B.V., Leiden, the Netherlands
Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands
University of Rochester, Rochester, N.Y.