Author Archives: Patricia Inacio PhD

FDA Places Gene Therapy AMT-130 on Fast Track to Speed Development

The U.S. Food and Drug Administration (FDA) has given fast track designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. The designation helps to speed the development, testing, and review of therapies for serious diseases with high unmet medical need. “Achieving Fast Track Designation from the…

Biomarker Platform That May Track Huntington’s Decades Before Symptom Onset Receives FDA Letter of Support

The U.S. Food and Drug Administration (FDA) has issued a letter of support encouraging sRNAlytics to further develop its sRNA-FIND platform for discovering and monitoring variants of small RNA molecules that may work as biomarkers for tracking progression of Huntington’s disease decades before symptom onset. In addition, these biomarkers may…

Fast Screening Strategy Identifies Potential New Inhibitors for Huntington’s Disease

Several small molecules have been identified by the Alborada Drug Discovery Institute as potential inhibitors to help halt the underlying mechanisms that drive the development of neurodegenerative diseases such as Huntington’s and Parkinson’s disease. Silencing phosphatidylinositol 5-phosphate 4 (PI5P4) kinases has been shown to enhance the natural cleaning system of cells, called autophagy,…