Newcomer NeuBase Therapeutics is re-engineering antisense oligos to access a broader range of targets, while simultaneously solving the modality’s dosing and delivery issues.
With three approvals since the start of 2016, antisense oligonucleotides (ASOs) have started to hit their stride, yet firstgeneration technologies are limited on multiple fronts.
Most ASOs only target linear RNA, which means they fail to bind the more complex structures often assumed by mutant, diseasecausing RNAs. And ASOs don’t cross the blood-brain barrier, which has meant that Biogen Inc.’s spinal muscular atrophy therapy Spinraza nusinersen must be delivered intrathecally.
NeuBase aims to address those issues, plus several others, with its Peptide-nu