Success Story

Gene therapy pioneer invents new therapy to improve delivery of gene therapies to treat ocular diseases

Jean Bennett, MD, PhD, F.M. Kirby Professor of Ophthalmology at Penn Medicine, is listed as lead inventor on an issued patent that describes mRNA trans-splicing as a gene therapy for treatment of ocular diseases caused by defects in large genes. The technology offers the ability to use smaller constructs to replace only a portion of a target gene, overcoming prior challenges caused by the transgene packaging limits of standard AAV-based gene therapy vectors.  This technology has been licensed to Ascidian Therapeutics (formerly Limelight Bio). 

Jean Bennett
Jean Bennett, MD, PhD
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