HitGen Inc. announced that it has entered into a research collaboration agreement with LoQus23 Therapeutics Ltd. a private biotechnology company discovering small molecule therapies that target aberrant DNA damage repair to treat Huntington's disease and other triplet repeat diseases (TRDs). HitGen will apply its DNA-encoded library (DEL) technology platform to discover compounds that bind to certain targets that are of interest to LoQus23. Triplet repeat diseases are genetic disorders that primarily affect the nervous system and are caused by abnormal trinucleotide repeat expansions.

Human genome-wide association studies on Huntington's disease and other TRDs have demonstrated that clinical disease measures, such as age of onset and rate of progression, are critically modified by genetic variants that increase mismatch repair pathway activity. Aberrant activity of this pathway leads to further expansions of trinucleotide repeats in the DNA sequence. That, in turn, translates into more toxicity in the neurons of vulnerable brain regions, thus accelerating the rate of neurodegeneration.

LoQus23's differentiated, structure-based approach targets DNA damage repair proteins with small molecule drugs to stop DNA instability and slow neurodegeneration in Huntington's disease, myotonic dystrophy type 1 and other TRDs. HitGen is a world leader in the development of the DEL technology and its applications to early-stage small molecule drug discovery. To explore therapies for the challenging TRDs, LoQus23 will provide novel targets, and HitGen will screen its expansive DELs of over 1.2 trillion small molecules against these targets.