Using its proprietary assay, Reata has discovered small molecule chaperones that correct misfolding of several important biological targets

Discovery

Reata is focused on discovering small molecule “chaperones” that can stabilize protein targets in the proper conformation. The key to our discovery efforts is a proprietary, best-in-class assay that has a number of advantages over competing technologies:

	Specificity for the protein of interest
	Conducted in mammalian cells, which allows protein folding to occur in a much more appropriate cellular context than in yeast-based assays
	Deployment in high-throughput format
	Flexibility in application to different types of protein-folding diseases (diseases of protein aggregation and diseases caused by loss of protein function)
	Does not require knowledge of a target’s normal function (unclear for a variety of misfolded protein targets)
	Relies on the proven approach of small molecule ligand binding

	Disease		Protein Target
	Cancer		p53 (mutated in 50% of all cancers)
	Amyotrophic lateral sclerosis (ALS )		SOD1
	Cystic fibrosis		CFTR
	Alzheimer’s disease		Tau, Aβ, others
	Parkinson’s disease		α-synuclein
	Huntington’s disease		Huntingtin

Using this platform, Reata has identified small molecule drugs that
have corrected the folding of SOD-1 and have demonstrated activity
in validated animal models of ALS. The lead agent in this program, RTA 801, has been advanced to preclinical development.