VGN-R13

VGN-R13 is an adeno-associated virus (AAV)-based gene therapy under early clinical development for the treatment of amyotrophic lateral sclerosis (ALS). It is being developed by Tongji Hospital Tongji Medical College of HUST. The therapy is delivered intrathecally and aims to address neurodegenerative processes in ALS, a fatal central nervous system disease with limited effective treatments. While the precise transgene or molecular target encoded by this AAV vector has not been disclosed in public sources, preclinical studies on related compounds such as R13—a small-molecule prodrug of 7,8-dihydroxyflavone that activates tropomyosin receptor kinase B (TrkB)—suggest a focus on neurotrophic signaling pathways relevant to motor neuron survival and function[1][2][3]. However, it should be noted that VGN-R13 itself is described specifically as an AAV-based gene therapy rather than a small molecule.

Brand names

VGN-R13VGN-R-13VGN-R 13

Other names

VGN-R13VGN-R-13VGN-R 13