Pseudotyped Lentiviral Vector for Duchenne Muscular Dystrophy (DMD)
Summary
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.
Overview
Engineered lentiviral platforms using Myomaker/Myomerger-mediated muscle targeting enable delivery of large genetic payloads with potential for reduced immunogenicity and repeat dosing in muscular dystrophies. One to two million patients have rare genetic muscle diseases with no satisfactory treatment, and AAVs are not suitable due to liver toxicity at high doses and pre-existing immunity limiting re-dosing. Cincinnati Children’s pseudotyped lentivirus system is muscle-specific, allows large payloads, and is non-immunogenic for applications such as Duchenne Muscular Dystrophy.
Applications
Duchenne Muscular Dystrophy
Value Proposition
Cincinnati Children’s pseudotyped lentivirus delivery system with MyoMaker and MyoMerger is a muscle-specific delivery system that allows large payloads and is non-immunogenic.
Market Overview
TAM: $4B (driven by DMD and LGMD); SAM: $700M; SOM: $400M
Investigator
Douglas Millay, PhD and Sajedah Hindi, PhD
