Shots:

• The US FDA has granted ODD for branaplam in HD. In preclinical trials- branaplam demonstrated a reduction in levels of the mutant huntingtin protein. Additionally- the therapy showed a reduction in huntingtin mRNA in SMA patients
• Novartis expects to initiate the P-IIb study for branaplam in HD patients in 2021
• Branaplam (qw- PO) is an RNA splicing modulator- currently under investigation for the treatment of SMA

 ­ Ref: Novartis | Image: Novartis