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sesiclenegene cosaparvovec
- Development stage
- Phase 2
- Lead developer
- The Abigail Wexner Research Institute
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intrathecal, Intravenous
01
Overview
Sesiclenegene cosaparvovec is an investigational AAV-based gene therapy designed for the treatment of neuronal ceroid lipofuscinosis type 6 (CLN6), a form of Batten disease. This therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the CLN6 gene to patient cells, aiming to restore or modulate the function of the defective CLN6 transmembrane endoplasmic reticulum protein. The mechanism involves gene transfer and modulation of CLN6 protein activity. Sesiclenegene cosaparvovec has received PRIME designation in the EU and is currently in Phase 2 clinical development for neuronal ceroid-lipofuscinoses.
- Other names
- CLN6 gene therapyCLN-6 gene therapyCLN 6 gene therapy
02
Targets
Gal (Terminal N-linked galactose)CLN6 (Ceroid-lipofuscinosis, neuronal 8 transmembrane ER and ERGIC protein)
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