uniQure's AMT-191 Gene Therapy For Fabry Disease Advances In Clinical Trial

UniQure N.V., a pioneering gene therapy organisation, announced the completion of enrollment of patients in the first Phase I/IIa clinical cohort to evaluate their experimental gene therapy AMT-191 for the treatment of Fabry disease. The Independent Data Monitoring Committee reviewed two initial patients without identifying substantial safety concerns, thus authorizing enrollment of the second cohort.  

Walid Abi-Saab, M.D., chief medical officer of uniQure, said in a statement, “Fabry is a debilitating disease that continues to represent a significant unmet medical need. We are encouraged by the initial pharmacodynamics, biomarkers, and safety profile observed to date for AMT-191, as well as the positive outcome of the IDMC review. This strengthens our confidence in the potential of AMT-191 to make a meaningful difference in the lives of patients with Fabry disease. We look forward to advancing to the second cohort in this important clinical program.”

AMT-191 represents an investigational gene therapy that uses the AAV5 vector to deliver galactosidase alpha (GLA) transgene through a special, highly powerful promoter. Through this method, the GLA protein is targeted to develop within the liver in order to resolve the enzyme deficiency from Fabry disease, which stems from GLA gene mutations. A harmful accumulation of lipids in multiple organs, including the heart and kidneys, because of this condition forms a multi-system disorder. AMT-191 offers a potential one-time intravenous treatment option for patients.