XM001

XM001 is an investigational adeno-associated virus (AAV) vector-based gene therapy developed by Hefei Xingmou Biotechnology (also known as StarryGene) for the treatment of neovascular (wet) age-related macular degeneration (nAMD). The therapy is designed to deliver a genetic sequence encoding an anti-VEGF protein directly to retinal cells, enabling the continuous intraocular production of the therapeutic agent. This approach aims to provide a long-term alternative to the frequent intravitreal injections required by current standard-of-care anti-VEGF biologics, potentially improving patient compliance and long-term visual outcomes. As of early 2026, XM001 has progressed into Phase 3 clinical development following successful investigator-initiated trials (IIT) in China that demonstrated preliminary safety and efficacy.