Pittsburgh developer of next-gen antisense oligonucleotide therapies says data generated from a pharmacokinetic study in non-human primates (NHPs) and in vitro pharmacodynamics studies in patient-derived Huntington’s disease (HD) cell lines supports its decision to advance the development of its lead programs in Huntington’ disease and myotonic dystrophy type 1, and expand its pipeline into other indications . . .
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