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SKY-AS
- Development stage
- Preclinical
- Lead developer
- Skylark Bio
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
SKY-AS is an investigational adeno-associated virus (AAV) gene therapy designed to treat Angelman syndrome, a rare monogenic neurodevelopmental disorder caused by a loss of function of the maternally inherited *UBE3A* gene. Developed by Skylark Therapeutics, SKY-AS utilizes a proprietary engineered capsid to deliver a functional copy of the *UBE3A* gene directly to neurons in the central nervous system. This approach is designed to enable broad transduction and targeted microdose delivery, restoring or supplementing UBE3A protein expression to address the underlying genetic cause of the disease. The program is currently in the IND-enabling stage of development.
02
Targets
UBE3A (Ubiquitin-protein ligase E3A)
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