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Dyno-3hv

Development stage
Preclinical
Lead developer
Dyno Therapeutics
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Recombinant Proteins and Enzymes, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

Dyno-3hv is an engineered adeno-associated virus (AAV) capsid developed by Dyno Therapeutics for gene therapy applications targeting neuromuscular disorders. It is the first AAV capsid to demonstrate highly efficient and enhanced delivery to multiple tissues affected by neuromuscular diseases, including heart, skeletal muscle, and brain, using a single drug product. In non-human primate studies, intravenous administration of Dyno-3hv resulted in transduction of 53–90% of skeletal myofibers as well as multiple regions of the heart and central nervous system at clinically relevant doses. The capsid features liver detargeting properties—minimizing off-target effects—and utilizes a novel receptor mechanism for crossing the blood-brain barrier that is conserved between non-human primates and humans. This supports its translational potential for human therapies. The vector is designed for manufacturability at large scale and compatibility with existing AAV9-based production processes[1][2].

02

Targets

MUSK (Muscle skeletal receptor tyrosine-protein kinase MuSK)

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