NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV)-BASED THERAPEUTICS REACH PIVOTAL PHASE 2 CLINICAL TRIALS

Abstract

This brief review summarizes the design of two phase 2 clinical trials of recombinant adeno-associated virus (rAAV)-based vector that are currently underway. These include a trial of a rAAV2-RPE65 vector for patients with Leber congenital amaurosis and a trial of a rAAV1-alpha-1-antitrypsin(AAT) vector delivered intramuscularly in patients with genetic emphysema due to AAT deficiency. In both cases, the current phase 2 trials were preceded by phase 1 trials indicating a good safety profile and persistent transgene expression.