Neutralizing Antibodies (NAb’s)

What are NAb’s and why are they important?

Neutralizing antibodies (NAbs) are the body’s defense mechanism against foreign pathogens (viruses). AAV is a type of virus used to deliver therapeutics – whether for gene replacement or gene editing. Individuals can have pre-existing NAb’s to the AAV virus as a function of everyday life. Certain levels of NAb’s can cause the body to attack the AAV virus instead of accepting it as a helpful therapeutic.

What are we doing about it?

CRD has partnered with the University of Massachusetts Medical School to provide Cure Rare Disease families the opportunity to gain knowledge about NAb levels in efforts to help themselves or their loved ones make the best decision for therapeutic options.

Academic Publications and More Information:

Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery

Relationship Between Neutralizing Antibodies Against Adeno-Associated Virus in the Vitreous and Serum: Effects on Retinal Gene Therapy

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

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