Guillaume Hautbergue
Founding Director & Chief Scientific Officer Crucible Therapeutics
Seminars
Friday 23rd January 2026
Advancing SRSF1-Interfering Genetic Therapies for the Treatment of C9orf72-ALS/FTD & Other Neurodegenerative Diseases
3:30 pm
- Strategies to identify 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