uniQure announced the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure’s AAV5 vector in a large animal model. uniQure has exclusive, worldwide rights to AAV5 for use in therapeutic products delivered to the brain or liver.
The method of injection used was found to result in very controlled and accurate administration with no adverse events observed in the non-human primates. Varying doses of AAV5 achieved predictable transduction of connected areas of the brain. The data demonstrate that AAV5 is a very effective vector for the central nervous system and has potential for the treatment of a wide range of neurological pathologies. These data will help guide the clinical development of uniQure’s gene therapy product candidate AMT-130, which consists of an AAV5 vector carrying an artificial micro-RNA that silences the huntingtin gene for the treatment of Huntington’s disease.
In the publication entitled “MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in nonhuman primate brain,” uniQure researchers in collaboration with experts from the University of California, San Francisco, describe the predictable and dose-dependent axonal transport of AAV5 after MRI-guided, convection-enhanced, parenchymal infusion. The results indicate that AAV5 can effectively achieve wide distribution throughout the central nervous system, which is crucial for the treatment of many CNS disorders, such as Huntington’s disease.
“This study further supports our belief that AAV5 is an optimal vector for CNS-delivery of gene therapy, and is an important step forward in defining the parameters for direct injection of AAV5 into the brain for future clinical applications in humans,” stated Harald Petry, uniQure’s chief scientific officer. “In particular, these data will help guide the design and development of the planned clinical trials of AMT-130. We look forward to making several preclinical data presentations at scientific meetings this year and to file an Investigational New Drug (IND) application for AMT-130 in 2018.”