PTX-101

PTX-101 is an investigational ex-vivo gene-edited cell therapy being developed by Primera Therapeutics for the treatment of chronic kidney disease (CKD) in patients harboring the m.3243A>G mitochondrial DNA mutation. The therapy involves the extraction of patient-derived renal progenitor cells, which are then edited using a TALE-based (Transcription Activator-Like Effector) mitochondrial DNA editing platform to correct the pathogenic m.3243 mutation. This mutation, located in the MT-TL1 gene, impairs mitochondrial protein synthesis and is a significant driver of renal dysfunction in affected individuals. By correcting the mutation ex-vivo and re-administering the edited cells, PTX-101 aims to restore mitochondrial function and slow or reverse the progression of kidney disease. The program is currently in Phase 1 development.

Other names

TALE-based mtDNA gene editing systemm.A3243G gene editing programPrimera Therapeutics mitochondrial gene editing therapy