AAV2.NN-TRPM1-NCR273

AAV2.NN-TRPM1-NCR273 is a next-generation optogenetic gene therapy designed for the treatment of severe retinal degeneration. Developed by Zhongmou Therapeutics, the therapy utilizes a novel engineered AAV2.NN capsid with improved inner retinal tropism to deliver a genetic payload via intravitreal injection. The expression cassette features a TRPM1 promoter, which enables selective expression of the enhanced photosensitive protein NCR273 specifically within ON-bipolar cells. By targeting these surviving downstream neurons, the therapy aims to restore light sensitivity and visual function in patients who have lost photoreceptor cells. Preclinical studies in rd10 mice models have demonstrated durable vision restoration, selective expression, and safety for up to 12 months, showing significant recovery of visually guided navigation and cortical signaling.