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hEN1 (AAV)
- Development stage
- Preclinical
- Lead developer
- BrainEver
- Modality
- Gene Therapies
- Administration
- Intracerebral
01
Overview
hEN1 (AAV) is an adeno-associated virus (AAV)-based gene therapy being developed by BrainEver for the treatment of Parkinson's disease. The therapy involves the continuous delivery of the human Engrailed-1 (EN1) gene via an AAV vector to target neurons in the brain, specifically midbrain dopaminergic neurons. EN1 is a homeoprotein transcription factor essential for the survival and maintenance of these neurons, which are progressively lost in Parkinson's disease. By restoring or augmenting EN1 expression and function, the therapy aims to protect and rejuvenate dopaminergic neurons, potentially halting disease progression and improving motor functions. The program is currently in the late discovery phase, with ongoing preclinical studies focused on activity, safety, and the selection of a viral delivery system for long-term local expression.
- Other names
- hEN1hEN-1hEN 1AAV-hEN1AAV-hEN-1AAV-hEN 1Engrailed-1 gene therapyEngrailed1 gene therapyEngrailed 1 gene therapy
02
Targets
EN1 (Homeobox protein engrailed-1)
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