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Emugen Dravet Syndrome Program
- Development stage
- Preclinical
- Lead developer
- Emugen Therapeutics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intracerebroventricular, Intravenous
01
Overview
Emugen Therapeutics is developing an AAV-based gene therapy for the treatment of Dravet Syndrome, a severe form of epilepsy characterized by SCN1A haploinsufficiency. The therapy utilizes a proprietary platform that targets "poison exons"—non-coding sequences that, when included in mRNA, lead to non-functional protein production. By modulating the splicing of the SCN1A gene to exclude these poison exons, the therapy aims to restore functional levels of the Nav1.1 sodium channel protein. The program is currently in the IND-enabling stage of development.
- Other names
- Dravet Syndrome ProgramSCN1A-Nav1.1-AAV gene therapySCN-1A-Nav1.1-AAV gene therapySCN 1A-Nav1.1-AAV gene therapy
02
Targets
SCN1A (Voltage-gated sodium channel protein type 1 subunit alpha)
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