MDL-104

MDL-104 is an investigational epigenome editing therapy developed by Modalis Therapeutics for the treatment of tauopathies, a group of neurodegenerative disorders characterized by the pathological aggregation of tau protein, including Alzheimer's disease. The therapy utilizes Modalis's proprietary CRISPR-GNDM (Guide Nucleotide-Directed Modulation) technology, which employs a catalytically inactive Cas9 (dCas9) protein fused to a transcriptional repressor to modulate gene expression without inducing double-strand DNA breaks. Delivered via an adeno-associated virus (AAV) vector, MDL-104 is designed to specifically target and suppress the transcription of the MAPT gene, thereby reducing the production of tau protein at the source and potentially halting the progression of tau-related pathology in the central nervous system.