FAQ about NAV®
Q. What is NAV® Technology?
A. NAV Technology is based on NAV vectors, the next generation of recombinant adeno-associated virus (rAAV) vectors used for in vivo therapeutic gene delivery and exhibit much improved characteristics over the existing vectors. There are over 100 NAV vectors. The most common NAV vectors include NAV rAAV8, NAV rAAV9 and NAV rAAVrh.10.
Q. What is a NAV vector made of?
A. Therapeutic candidates based on NAV vectors consist of only a protein coat that contains a single-stranded DNA genome that includes a therapeutic gene expression cassette. When used in vivo for gene transfer, these NAV vectors give stable, long-term gene expression, at therapeutic levels.
Q. How do NAV vectors work?
A. NAV vectors are able to enter cells and deliver their DNA genome to the nucleus, but are designed to be unable to replicate. When the genome from a NAV vector reaches the nucleus gene expression will occur. Genomes delivered by NAV vectors do not integrate into chromosomal DNA and gene expression may persist for years.
Q. What are NAV vectors’ benefits?
A. NAV vectors convey the safety advantages and the stability of expression of traditional rAAV vectors (like rAAV2), but deliver much higher levels of expression.
Q. Have NAV vectors been studied in humans?
A. The first human clinical study using a NAV vector was initiated in 2010 for treating Hemophilia, using NAV rAAV8. A second study was initiated later in 2010 for treating Batten disease (an inherited severe neurodegenerative disorder), using NAV rAAVrh.10. In 2011, a third study was initiated using NAV rAAVrh.10 to treat MPS IIIA, an inherited severe neurodegenerative disorder in the mucopolysaccharidosis family of diseases. REGENX Biosciences and its partners are developing novel personalized therapies based on NAV technology.
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