Gene Therapy Trial for CLN6 Batten Disease
Conditions
- CLN6
- Batten Disease
- Batten's Disease
- Neuronal Ceroid Lipofuscinosis CLN6
- Neuronal Ceroid Lipofuscinosis
Interventions
- DRUG: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
Sponsor
The Charlotte and Gwenyth Gray Foundation
Collaborators