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Table 2 Adverse immune response against AAV in haemophilia B clinical trials

From: Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering

Vector No of subjects Vector dose Adverse Effect Immune response Reference
AAV2 2 2X10^12 (high dose) Liver toxicity based on elevated AST/ALT levels beginning 4 weeks post vector infusion with concomitant decline of circulating h.FIX to the baseline (<0.1%) by 8 weeks CD8+ T cell response against AAV capsid as well as preexisting neutralizing antibody against AAV2 capsid prevented long term expression [11]
AAV2 4 4X10^11 (low dose) No increase in circulating hF.IX from the baseline (<0.1%), increased transaminases only in one subject having the lowest pretreatment NAb CD8+ T cell response against AAV capsid as well as prexisting neutralizing antibody against AAV2 capsid prevented hF.IX expression [11]
AAV8 1 2X10^12 (high dose) Liver toxicity based on elevated AST/ALT levels beginning 8 weeks post vector infusion with concomitant decline of circulating hF.IX levels CD8+ T cell response against AAV capsid leading to destruction of the transduced hepatocytes [16]