Drug intelligence / Profile preview

NeuroD1 gene therapy

Development stage
Preclinical
Lead developer
NeuExcell Therapeutics
Modality
Gene Therapies
Administration
Intracerebral, Intraspinal
01

Overview

NeuroD1 gene therapy is an experimental **AAV-based in vivo neural reprogramming therapy** that delivers the **NeuroD1 transcription factor** to reactive astrocytes, typically using an **AAV9 vector under a GFAP promoter**, to convert astrocytes into functional neurons after central nervous system injury. The therapeutic concept has been developed primarily in academic settings and remains **preclinical**, with studies in rodent and non-human primate models of **ischemic stroke, ischemic brain injury, spinal cord injury, subarachnoid hemorrhage, and neonatal hypoxic-ischemic brain injury**. Its proposed mechanism is direct astrocyte-to-neuron conversion with associated neuronal replacement, neuroprotection, and circuit repair, leading in published preclinical work to recovery of neuronal populations and improvement in motor and cognitive outcomes.

Other names
NeuroD1 gene therapyNeuroD-1 gene therapyNeuroD 1 gene therapyAAV9 GFAP NeuroD1 gene therapyAAV-9 GFAP NeuroD1 gene therapyAAV 9 GFAP NeuroD1 gene therapyNeuroD1 AAV gene therapyNeuroD-1 AAV gene therapyNeuroD 1 AAV gene therapy
02

Targets

TBX3 (T-box transcription factor TBX3)DNATCF4 (Transcription factor 4)

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