Transforming CNS delivery for gene therapy
We are developing a non-viral delivery platform using exosomes for central nervous system (CNS) therapies, with a focus on intranasal delivery to enable broad brain distribution. Recent data in ischemic stroke, Alzheimer’s disease, and Parkinson’s disease supports the potential of this approach for effective CNS targeting.
Vybion has demonstrated proof of concept in vitro and in animal models for its therapeutic candidate targeting Huntington’s disease, along with Orphan Disease designation and prior engagement with the FDA. The platform is designed to deliver targeted genetic material to address underlying disease mechanisms associated with neurodegeneration.
We believe exosome-based delivery represents a promising next-generation approach for CNS therapies, with potential advantages in safety, repeat dosing, and accessibility.
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