Technology/Pipeline
We have exclusive access to a portfolio of gene therapies and are actively developing a therapeutic pipeline, including a range of supporting technologies.
Plos One Article – https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0022850
Acta Neuropathologica – https://link.springer.com/article/10.1007/s00401-020-02191-1
Nature Communications – https://www.nature.com/articles/ncomms11253
ALS – Amyotrophic Lateral Sclerosis
- Genetic and sporadic forms of ALS
- ~50% of Frontotemporal Dementia
- Target – pathogenic TDP-43
- MOA – proteostasis
Dementia – Alzheimer’s Disease
- Target – P38gamma MAP Kinase
- MOA – reduction of excitotoxicity
Epilepsy – Dravet Syndrome
- Target – P38gamma MAP Kinase
- MOA – reduction in excitotoxicity
