A-Z™ HD Drug Discovery Platform

After more than 10 years of dedicated research and development, Creative Biolabs has pioneered an agile biotechnology platform for preclinical drug discovery based on neurological disorders. They also provide a flexible platform for the creation of new therapeutic agents.

Overview

Huntington's disease (HD) is one of nine neurodegenerative polyglutamine disorders. HD is inherited in an autosomal dominant manner and can lead to sleep disorders, motor dysfunction, cognitive impairment, psychiatric abnormalities, and ultimately premature death. The cause of HD has been identified as a mutation in the gene encoding the protein huntingtin (Htt). So far, no effective treatment has been found to prevent the onset or progression of HD. Stem cell-based techniques represent a promising direction for HD treatment because of the ability of stem cells to differentiate into NPCs, replace damaged neurons, and prevent nerve cell death.

Models and Assays for HD Preclinical Drug Discovery

HD in vitro disease models and in vivo models are invaluable for understanding the pathogenesis of the disease, screening potential drugs, and determining the efficacy of lead molecules.

• In Vitro Models-iPSC-Based Modeling of HD

iPSC technology in neuroscience not only provides a possible method of cell therapy but also greatly facilitates the study of disease pathology. iPSC-derived HD models represent a much more suitable alternative for drug screening and toxicity testing than widely used animal models. Human iPSC-based models have the potential to serve as powerful tools for high-throughput drug screening, bioinformatics, and global gene expression analysis, accelerating the pace of drug discovery and reducing drug wastage rates.

Fig.1 The generation and application of iPSCs in HD research.

• In Vivo Models

Numerous transgenic mouse and rat and knock-in mouse models of HD are available for preclinical research. In vivo models of HD have been developed in many organisms, including Drosophila, the ringworm Caenorhabditis elegans, mice, and rats. The lower animal models can serve as simple platforms for initial drug screening but not for detailed analyses. The use of mouse models has been very fruitful, and genetically engineered rodent models have provided excellent insight into HD pathology, including estimation of the extent of gene expression changes, the discovery of neuronal intranuclear inclusions, and other aspects of HD such as cognitive dysfunction, sleep, and circadian rhythm disorders.

Creative Biolabs' visionary team has been at the forefront of preclinical drug discovery for more than a decade. Our cutting-edge science, coupled with strong drug evaluation capabilities, supports our unique drug discovery platform to meet customized solutions. If your project needs the help of our preclinical drug discovery platform, please do not hesitate to contact us for a detailed discussion with our team of experts.