Drugging the undruggable
Diseases are caused by multifactorial changes in the cell. Transcription factors (TFs) are a class of proteins that regulate dozens or even hundreds of genes. Research in the past several decades have elucidated misregulated expression of TF networks across a wide range of therapeutic areas. Although many disease-causing TFs have been genetically validated to drive diseases, they are hard to target due to the lack of small molecule binding sites. At Altay, we have developed a scalable method of identifying novel small molecule binding domains resulting in direct nM binding to target TF.
Our disease agnostic platform enables discovery of small molecule binding sites within intrinsically disordered domains. Using our platform, we have developed three first-in-class inhibitors targeting previously undruggable TFs for the treatment of fibrosis (IPF & NASH), Ewing’s Sarcoma/Prostate Cancer, and Facioscapulohumeral muscular dystrophy (FSHD). Based on the knowledge gained from our platform, we are now able to drug disease-causing TFs to develop more specific and safer therapies to improve the quality of life for an even greater patient population.
In vitro genetic assays determine residues critical for protein function.