ASP2767

ASP2767 is an adeno-associated virus (AAV) vector-based gene therapy developed by Astellas Pharma for the treatment of optic neuropathy associated with open-angle glaucoma. Originally developed by Quethera (as QTA0001) before its acquisition by Astellas, the therapy utilizes a bicistronic vector to deliver genes encoding Brain-Derived Neurotrophic Factor (BDNF) and its cognate receptor, Tropomyosin Receptor Kinase B (TrkB/NTRK2), directly to retinal ganglion cells (RGCs). By overexpressing these neurotrophic factors, ASP2767 aims to enhance the survival and metabolic health of RGCs, which are the primary cells damaged by elevated intraocular pressure in glaucoma. This neuroprotective approach is intended to slow or halt the progression of vision loss independently of intraocular pressure lowering. The drug is administered as a single intravitreal injection and is currently being evaluated in Phase 1/2 clinical trials.