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Kinea Bio, Inc. secured a $796,218 NIH SBIR/STTR award: Development of novel genetic medicine approaches to treat orphan diseases
Amount Raised
$796,000
Round Type
Grant
SeattleBiotechnologyHealth CareInvestors
Description
SUMMARY: Adeno-associated viral (AAV) vectors hold great promise for treating genetic disorders. AAVs efficiently transduce tissues, including skeletal and cardiac muscles, which is attractive for treating multisystemic muscular diseases. The primary shortcoming of using AAV vectors is their limited cargo capacity (~5 kb), which excludes many disorders, such as dystro- or dysferlinopathies, from using these vectors as gene transporters. Due to the large coding sequences of the defective genes, r
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