For AAV and LV vectors it has been shown that such replication-defective vectors are non-immunogenic in hosts that are naïve to the cognate virus and induce strong immune tolerance responses to the transgene proteins. Viral vector-mediated expression of a primary self-antigen (pSAg) of an autoimmune disease such as diabetes mellitus type 1, rheumatoid arthritis and multiple sclerosis, induces immune tolerance to the pSAg and both protects and cures the treated animal from developing the disease. Viral vector-mediated tolerization e.g. reverse vaccination has an enormous potential to effectively treat autoimmune/degenerative diseases for which the pSAgs have been identified. Since LV vectors can only be used in ex vivo gene therapies and AAV vectors are immunogenic in humans, our SVec platform is the only vector system suitable for use as reverse vaccines to treat human autoimmune diseases such as neurodegenerative & psychiatric diseases, atherosclerotic cardiovascular disease, obesity, diabetes mellitus, arthritis, chronic obstructive pulmonary disease, inflammatory bowel diseases and many more.
For our reverse vaccine project AMA002, mouse experiments have shown that SVac-mediated expression of the pSAg (proinsulin) of an autoimmune/degenerative disease (diabetes mellitus type 1) protects the reverse vaccinated animals from developing the disease. This proof-of-principle in an animal model of an autoimmune/degenerative disease opens the way to develop a whole new generation of effective reverse vaccines for the major diseases of our time.