TwinFin

TwinFin is a multi-targeting gene therapy developed for the treatment of neurodegenerative disorders characterized by TDP-43 proteinopathy, such as Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). Developed by researchers at the University of California San Diego and Genentech, TwinFin utilizes a single self-complementary adeno-associated virus serotype 9 (scAAV9) vector to deliver engineered U7 small nuclear RNAs (snRNAs). These snRNAs are specifically designed to block the inclusion of cryptic exons in the STMN2 and UNC13A transcripts, a pathological process triggered by the nuclear depletion of TDP-43. By restoring normal pre-mRNA processing, TwinFin enables the production of functional stathmin-2 and UNC13A proteins, which are essential for axonal regeneration and synaptic signaling.