Unlocking Genetically Validated CNS Targets: Leveraging Human Genetic Biology to De-risk Oligonucleotide Development
- Utilizing human genetic resilience datasets to prioritise high-impact CNS targets independent of traditional drugability constraints
- Translating genetic mechanisms into oligo strategies, for expression modulation and splice-switching approaches
- Advancing first-in-class programmes through IND-enabling pharmacology by integrate efficacy and safety signals in neurodegenerative models