UMass Chan researchers receive FDA authorization to advance gene therapy clinical trial ...

UMass Chan researchers have received FDA authorization to begin Phase I/II clinical trials of a second-generation AAV gene therapy for GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases), a rare fatal neurodegenerative disorder with no existing treatments. The therapy builds on over a decade of research and aims to improve cellular delivery at lower doses than previous approaches.

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