Drug intelligence / Profile preview

BN-1001

Development stage
Unknown
Lead developer
Bioheng Therapeutics
Modality
Gene Therapies
Administration
Intravitreal
01

Overview

BN-1001 is an investigational adeno-associated virus (AAV)-based gene therapy developed by West China Hospital of Sichuan University for the treatment of neovascular (wet) age-related macular degeneration (nAMD). The therapy is designed to deliver a genetic sequence encoding an anti-VEGF (vascular endothelial growth factor) protein directly to ocular tissues via a single intravitreal injection. By enabling the endogenous and sustained production of VEGF inhibitors, BN-1001 aims to suppress the pathological angiogenesis and vascular leakage characteristic of nAMD, potentially reducing the treatment burden associated with frequent injections of recombinant anti-VEGF proteins. It is currently undergoing evaluation in a Phase 1 clinical trial in China to assess its safety, tolerability, and preliminary ophthalmic outcomes in patients previously treated with standard-of-care anti-VEGF agents.

02

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