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Lilly's AK-OTOF gene therapy restored hearing in an 11-year-old born profoundly deaf within 30 days
The first child treated in the AK-OTOF-101 trial, an 11-year-old deaf since birth from OTOF mutations, regained hearing across all tested frequencies within 30 days. Some frequencies reached the normal range. Lilly said the therapy and its surgical delivery were well tolerated with no serious adverse events.
- Hearing thresholds of 65 to 20 dB HL at day 30, from profound deafness.
- Single injection into one cochlea with a dual-AAV vector carrying the otoferlin gene.
- Trial sites included the Children's Hospital of Philadelphia; target of 14 patients aged 2 to 17 at the time.
- AK-OTOF holds FDA Orphan Drug and Rare Pediatric Disease designations.