oral, CNS-penetrant splicing modulator w/ HTT activity - Novartis, Cambridge, MA

oral, CNS-penetrant splicing modulator w/ HTT activity

in Ph. II for Huntington's (prev. SMA)

further characterization of SMA candidate

Nat. Commun.

Novartis, Cambridge, MA

Context. Branaplam (Novartis Institutes for Biomedical Research) is an oral brain-penetrant splicing modulator, originally developed as a small molecule splicing modulator for spinal muscular atrophy (SMA). It turns out…


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