Drug intelligence / Profile preview

AAV.RTN202

Development stage
Preclinical
Lead developer
Westlake Genetech
Modality
Gene Therapies
Administration
Ophthalmic
01

Overview

AAV.RTN202 is an AI-engineered adeno-associated virus (AAV) vector designed for robust pan-retinal gene delivery via intravitreal (IVT) injection. Developed by Westlake Genetech using an AI-evolution platform, it is a variant of AAV2 specifically optimized to overcome the inner limiting membrane (ILM) barrier, which typically restricts the penetration of viral vectors into the retina. In preclinical studies involving cynomolgus macaques, AAV.RTN202 demonstrated significantly higher transduction efficiency (5.3-fold) and a faster onset of expression compared to the benchmark vector AAV2.7m8. It also showed a superior safety profile, avoiding structural complications like iris synechiae, and exhibited improved manufacturing characteristics such as higher yields and lower empty capsid percentages.

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