Committed to finding and developing novel advanced therapies for devastating rare diseases.
Aquracy Therapeutics is a biotechnology startup originated in the south of Portugal, at the University of Algarve. Our primary focus is preclinical research and innovation aimed at developing new therapies for devastating rare genetic diseases.
Our main preclinical research activities are centered on essential steps for clinical translation, which include:
– Target discovery using bioinformatics tools to identify new therapeutic candidates.
– The production and validation of potential targets using gene therapy techniques.
– Testing candidate molecules in cellular models through cell culture and molecular analysis.
– Evaluating the candidate’s efficacy and toxicity in animal models.
Currently, Aquracy Therapeutics is highly focused on PolyQ diseases, a group of rare neurodegenerative diseases, which include Spinocerebellar ataxias (SCAs) type 1, 2, 3, 6, and 17, Huntington’s disease (HD), Dentatorubral pallidoluysian atrophy (DRPLA), and Spinal and bulbar muscular atrophy (SBMA). Through a gene augmentation therapeutic strategy, we obtained the proof-of-concept and preliminary safety data for a genetic therapeutic target, both in vitro and in vivo, and are now pursuing the steps for an Investigational New Drug (IND) application. We recently added to our pipeline preclinical research in Cockayne Syndrome, a pediatric neurodegenerative disease, in which we are investigating disease-modifying molecular targets in vitro.
Aquracy Therapeutics’ ultimate goal is to translate discovery and innovation into valuable real-life impact, as well as the pursuit of novel life-changing therapies.
