SynoGene

SynoGene is a clinical-stage biotechnology company based in Beijing, China, specializing in the development of adeno-associated virus (AAV) mediated gene therapies. The company focuses on treating rare genetic disorders, particularly in ophthalmology and hematology, where current treatment options are limited. Its lead candidate, SN-101, is an investigational gene therapy for Leber Hereditary Optic Neuropathy (LHON) caused by ND4 mutations, while SN-201 targets Hemophilia B. SynoGene utilizes proprietary vector engineering and optimized manufacturing processes to enhance the safety and efficacy of its genetic medicines. The firm is backed by leading venture capital investors and aims to deliver transformative, one-time treatments for patients globally.