EH002

EH002 is an investigational gene therapy designed to treat congenital sensorineural hearing loss (specifically DFNB9) caused by mutations in the otoferlin (**OTOF**) gene. Developed by **EnhanX Biopharmaceuticals**, the therapy utilizes an adeno-associated virus (AAV) vector system to deliver a functional copy of the OTOF gene directly into the inner hair cells of the cochlea. Because the OTOF gene is too large for a single standard AAV vector, EH002 typically employs a dual-vector approach where the gene is split into two halves that recombine within the target cells. By restoring the production of the otoferlin protein, which is essential for calcium-triggered neurotransmitter release at the auditory synapse, EH002 aims to restore hearing in patients who are otherwise profoundly deaf from birth.