AAV-2-hNGF

AAV-2-hNGF (adeno-associated virus serotype 2 delivering human nerve growth factor) is an investigational gene therapy designed to deliver the human nerve growth factor (hNGF) gene to target tissues. Nerve growth factor (NGF) is a neurotrophin that plays a critical role in the survival, maintenance, and regeneration of specific neuronal populations, particularly cholinergic neurons, as well as exerting cardioprotective and angiogenic effects. AAV-2-hNGF has been investigated across multiple therapeutic areas. In neurology, as CERE-110 (developed by Ceregene and Sangamo Therapeutics), it was evaluated in clinical trials for Alzheimer's disease, where it was stereotactically delivered to the nucleus basalis of Meynert to rescue degenerating cholinergic neurons. In ophthalmology, Oyster Point Pharma (acquired by Viatris) developed an AAV-NGF gene therapy (OC-101/MR-146) delivered via intralacrimal gland injection to treat neurotrophic keratopathy by promoting corneal nerve regeneration and tear film enrichment. Additionally, academic researchers have explored intramyocardial delivery of AAV-2-hNGF to prevent diabetic cardiomyopathy and microangiopathy by preserving cardiac microvasculature and improving left ventricular function.

Other names

AAV-2-NGFAAV2-NGFAAV 2-NGFAAV2-hNGFAAV-2-hNGFAAV 2-hNGF