OTOV101C

OTOV101C is a gene therapy candidate developed by Otovia Therapeutics for the treatment of DFNB9, an autosomal recessive form of deafness caused by mutations in the *OTOF* gene. Due to the large size of the *OTOF* cDNA (encoding the protein otoferlin), which exceeds the ~4.7 kb packaging limit of standard adeno-associated virus (AAV) vectors, OTOV101 utilizes a dual-vector approach. OTOV101C is the AAV vector containing the C-terminal fragment of the *OTOF* gene. When co-injected into the cochlea with its partner vector, OTOV101N (containing the N-terminal fragment), the two components undergo concatemerization or trans-splicing within the target inner hair cells to express the full-length, functional otoferlin protein. Otoferlin is essential for calcium-mediated synaptic vesicle fusion and neurotransmitter release in the inner ear; its restoration is intended to recover auditory function in affected patients.