Journal: Molecular Therapy Advances

Article Title: Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness

doi: 10.1016/j.omta.2026.201762

Figure Lengend Snippet: Intracochlear injection of SENS-501 in NHPs results in a mild humoral and an undetectable cellular response to the capsid (A) AAV8 anti-drug antibody (ADA) titers measured in serum at pre-dose and at 16, 29, and 92 days post-vector administration in NHPs. (B) Anti-AAV8 neutralizing antibodies (NAb) titers measured in serum at pre-dose and at 16, 29, and 92 days post-vector administration in NHPs. (C and D) IFN-γ spot forming units (SFUs) measured by ELISpot assay at 29 (C; left) and 92 (D; right) days post-injection. Peripheral blood mononuclear cells (PBMCs) from the indicated groups were stimulated with three different AAV8 peptide pools and a positive control (PMA/ionomycin). The dotted line represents the assay-specific positivity threshold. Each dot represents one animal. Bars represent the mean ± SEM.

Article Snippet: Briefly, AAV8-CMV-luciferase (ViroVek) was pre-incubated with either a dilution of NHP serum, a neutralization control consisting of diluted anti-AAV8 neutralizing antibodies (Progen), or vehicle (non-neutralizing condition).

Techniques: Injection, Plasmid Preparation, Enzyme-linked Immunospot, Positive Control