Opal Sandy, a 3-year-old girl from the United Kingdom, has achieved a remarkable milestone after undergoing a groundbreaking gene therapy treatment aimed at addressing profound hearing loss. The therapy was the initial cohort of a clinical trial of DB-OTO gene therapy. Most of all, it has returned the power of sound for the very first time in her life to Opal!
Opal was born with an inherited condition, due to mutations of the OTOF gene, that had left her profoundly deaf. Her condition was extremely important. Her older sister, Nora, had faced the exact same set of obstacles due to the same exacting genetic mutation. Ojai, California-based Opal began her journey with restoration at just 11 months old. This milestone made her the youngest patient in the world to receive the gene therapy injection.
The clinical trial featured participants across four continents, including the United Kingdom, United States, Spain and Germany. We were super grateful that, during her procedure, Opal was able to receive a cochlear implant in her left ear along with gene therapy. The whole procedure lasted just 15 minutes and was done under general anesthesia. The therapy used a non-replicating virus to deliver a working copy of the OTOF gene directly into Opal’s right cochlea.
Two years after the surgery, Opal’s progress has been remarkable. This time, she was able to hear without her cochlear implant at all. Shelby’s parents are shocked by how far she’s come. She called the transformation to her hearing experience “mind-blowing.”
This pioneering new treatment of state-of-the-art gene therapy is a game changing breakthrough in medical science. The success of Opal’s treatment shines a light on what’s possible for many other children afflicted by similar devastating genetic disorders. As research continues, these therapies carry the potential to be game-changing. In addition, they could present new possibilities for curing hearing loss due to genetic mutations.
