Advancing Capsida’s Novel Engineered AAV Gene Therapies to the Clinic: Brain-Wide Expression of STXBP1 Corrects Seizure, Cognitive & Motor Haploinsufficiency Phenotypes
Time: 11:30 am
day: POST-CONFERENCE DELIVERY FOCUS DAY
Details:
- Capsida’s high-throughput, automated platform for screening engineered AAV capsids has accelerated the discovery of novel breakthrough capsids from multiple serotypes that cross the BBB following intravenous delivery to efficiently distribute genetic payloads throughout the CNS
- Presenting overview of Capsida’s program targeting a genetic epilepsy caused by mutations in the STXBP1 gene
- Preclinical models indicate that brain-wide expression of STXBP1 corrects seizure, cognitive and motor phenotypes caused by haploinsufficiency of STXBP1 protein
- Cargo delivered by novel capsids achieves therapeutic expression levels in NHP
