GNE-Lipoplex

**GNE-Lipoplex** is a non-viral gene therapy consisting of a liposomal formulation delivering the wild-type human **GNE** gene (UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase) to address mutations causing **Hereditary Inclusion Body Myopathy (HIBM)**, also known as **GNE Myopathy**, a rare autosomal recessive disorder leading to progressive skeletal muscle weakness and atrophy due to hyposialylation. Developed initially by **Gradalis** (formerly Mary Crowley Cancer Research/Intrexon), it demonstrated proof-of-principle in a single-patient compassionate IND trial approved by the FDA in 2008, showing intramuscular and intravenous delivery led to transgene expression, increased sialic acid levels, stabilization of muscle strength decline, and acceptable safety with transient mild adverse events like fever and myalgia. Solve GNE partnered with Gradalis for next-generation improvements using bi-shRNAi for a planned Phase 1 trial in 2024 targeting less advanced patients.[1][2][3][5][10]