Leader in the development of human gene therapies, uniQure, have announced the release of new preclinical data that supports its gene therapy program for Huntinton’s disease.
uniQure’s program, AMT-130, is delivered as a one-time dose to the CNS to silence the Huntington gene (HTT), delivered via an AAV5 vector. The study describing the preclinical study is found published in Molecular Therapy-Nucleic Acids.
The paper describes several approaches that were used to silence the HTT gene using expression cassette-optimized artificial microRNAs (miHTTs). Using uniQure’s baculovirus-based manufacturing platform, several miHTTs constructs were incorporated into an AAV5 vector before being administered to a humanized mouse model. Results displayed a strong silencing of mutant HTT and complete silencing of HTT in vitro and in vivo.
“We are excited by the results of this study, and believe this degree of knock-down of mutant Huntingtin protein, if duplicated in our ongoing non-human primate safety toxicology studies and future human clinical trials, could significantly alter the course of the disease,” said Charles W. Richard, Senior Vice President, Research and Development, Neuroscience at uniQure.
Source: uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington’s Disease; Press Release