Committed to discovering and developing new, advanced treatments for devastating rare diseases.
Aquracy Therapeutics is a biotechnology start-up based in southern Portugal, at the University of the Algarve. Our main focus is on pre-clinical research and innovation, with the aim of developing new treatments for rare and devastating genetic disorders.
Our main preclinical research activities focus on key stages leading to clinical translation, which include:
– Target discovery using bioinformatics tools to identify new therapeutic candidates.
– The generation and validation of potential targets using gene therapy techniques.
– Testing of candidate molecules in cellular models using cell culture and molecular analysis.
– Evaluation of the candidate’s efficacy and toxicity in animal models.
Aquracy Therapeutics is currently strongly focused on PolyQ diseases, a group of rare neurodegenerative disorders that includes spinocerebellar ataxias (SCA) types 1, 2, 3, 6 and 17, Huntington’s disease (HD), dentatorubral-pallidoluysian atrophy (DRPLA) and spinal and bulbar muscular atrophy (SBMA). Through a gene-enhancement therapeutic strategy, we have obtained proof of concept and preliminary safety data for a genetic therapeutic target, both in vitro and in vivo, and we are now taking the necessary steps towards submitting an Investigational New Drug (IND) application. We have recently added preclinical research into Cockayne syndrome, a paediatric neurodegenerative disorder, to our pipeline, and are currently investigating disease-modifying molecular targets in vitro.
Aquracy Therapeutics’ ultimate goal is to translate discovery and innovation into meaningful real-world impact, as well as to seek out new therapies that change lives.
