SMDG-HD11

SMDG-HD11 is a small molecule therapeutic candidate currently in the lead optimization phase for the treatment of Huntington's Disease. Developed by SM Discovery Group (SMDG), the program utilizes the company's platform for discovering orally bioavailable small molecules that target disease-relevant RNA or protein pathways. In the context of Huntington's Disease, SMDG-HD11 is designed to address the underlying genetic cause of the disorder, which is characterized by an expanded CAG repeat in the HTT gene leading to the production of toxic mutant Huntingtin (mHTT) protein. The therapeutic objective of SMDG-HD11 is to lower the levels of mHTT, potentially by modulating HTT mRNA splicing or stability, thereby slowing or halting neurodegeneration.