• Strategies to identifying emerging genetic targets beyond the crowded IP space and evaluating their therapeutic potential with different oligonucleotide modalities
• Overcoming preclinical translation challenges by leveraging humanized and disease-specific models to confirm target relevance, address species-specific phenotypes, and predict clinical efficacy
• Integrating bioinformatics and AI-driven approaches to prioritise causal targets, enhance mechanistic validation, and accelerate the selection of disease-relevant candidates 

NEW DATA