NG201

NG201 is an investigational adeno-associated virus (AAV) gene therapy being developed by Elisigen for the treatment of Amyotrophic Lateral Sclerosis (ALS). The therapy utilizes Elisigen's proprietary AAV vector engineering platform, which is designed to enhance delivery efficiency and transgene expression within the central nervous system. NG201 is engineered to deliver a therapeutic payload—likely an intracellular antibody (intrabody) or a gene-modifying construct—targeting pathological protein aggregates such as TAR DNA-binding protein 43 (TDP-43), which are a hallmark of neurodegeneration in the majority of ALS cases. By neutralizing these toxic aggregates or preventing their formation, NG201 aims to preserve motor neuron function and slow the progression of the disease. The program is currently in the preclinical or IND-enabling stage of development.