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EPY201
- Development stage
- Preclinical
- Lead developer
- EpilepsyGTx
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intracerebral
01
Overview
EPY201 is an investigational gene therapy developed by EpilepsyGTx for the treatment of focal refractory epilepsy. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a gene construct encoding an engineered Kv1.1 potassium channel under the control of a CAMK2A promoter. The therapy is designed to be administered directly into the area of the brain responsible for seizure activity, aiming to restore normal neuronal excitability and reduce seizures by stimulating Kv1.1 potassium channels[1][2][4]. As of July 2024, it remains in preclinical development with plans for phase I/IIa clinical trials[2].
- Other names
- AAV9-CAMK2A-EKCAAV-9-CAMK2A-EKCAAV 9-CAMK2A-EKCEPY-201EPY201EPY 201
02
Targets
CAMK2A (Calcium/calmodulin-dependent protein kinase II alpha)KCNA1 (Voltage-gated potassium channel subfamily A member 1)
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