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news.REGENX BioSciences has received FDA orphan drug designation for treatment of familial hypercholesterolemia (HoFH) using NAV rAAV8 vectors.
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The REGENX program consists of a NAV rAAV8 vector expressing a normal copy of the human low-density lipoprotein receptor (hLDLR) gene.
The study, conducted at University of Pennsylvania in a mouse model of HoFH, has shown that delivery of functional hLDLR to the liver through NAV rAAV8-mediated gene delivery results in decreases in circulating LDL-C.
REGENX along with its academic collaborators is developing a clinical program intended to support the licensure of a product for the treatment of HoFH.
REGENX president and chief executive officer Ken Mills said: "This designation underscores our interests in developing novel therapies for rare, genetic diseases with serious unmet medical need."