DT003

DT003 is an adeno-associated virus (AAV) vector-based gene therapy candidate developed by Diorasis Therapeutics for the treatment of open-angle glaucoma (OAG). The therapy is designed to deliver a gene encoding an enzymatically inactive variant of tissue plasminogen activator (EI-tPA) directly to the cells of the trabecular meshwork via a single intracameral injection. Once expressed, the EI-tPA acts as a master regulator of matrix metalloproteinases (MMPs), which are enzymes responsible for remodeling the extracellular matrix (ECM) of the eye's outflow tissues. By modulating ECM turnover, DT003 aims to restore the eye's natural drainage system, thereby improving aqueous humor outflow facility and providing a long-term, sustained reduction in intraocular pressure (IOP) for both juvenile and adult glaucoma patients.

Other names

AAV-engineered tPAAAV-engineered tPA (EI-tPA)tPA gene therapy