CRISPRAlz

CRISPRAlz is a pre-clinical stage biotechnology company focused on developing one-time, disease-modifying gene-editing therapies for neurodegenerative disorders, with a primary focus on Alzheimer's disease. Co-founded by Dr. Subhojit Roy and Dr. Prasad Sunkara, the company leverages CRISPR-Cas9 technology delivered via adeno-associated virus (AAV) vectors to edit the amyloid precursor protein (APP) gene. Specifically, their therapeutic approach targets and deletes the endocytic YENPTY motif at the C-terminus of APP, which prevents its internalization into endosomes and shifts processing away from the amyloidogenic pathway. This strategy reduces toxic beta-cleavage fragments, including amyloid-beta (Aβ), while upregulating neuroprotective alpha-cleavage products, offering a potential universal treatment for both familial and sporadic forms of Alzheimer's disease.