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A Next-Generation, Non-Viral, Non-Lipid Gene Therapy Platform for Cystic Fibrosis & Beyond
A novel non‑viral, non‑lipid delivery system enables safe transfer of large genetic payloads, avoiding key limits of current gene therapies and improving uptake using targeted siRNA or drug-based enhancers.
Pseudotyped Lentiviral Vector for Duchenne Muscular Dystrophy (DMD)
Engineered lentiviral platforms leveraging Myomaker/Myomerger-mediated muscle targeting overcome key limitations, enabling delivery of large genetic payloads with the potential for reduced immunogenicity and repeat dosing in muscular dystrophies.
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