Latest Techniques in Hearing Restoration

Gene therapy restores hearing, even in a 24-year-old who was born deaf (which is a big deal, not just for them but also the science): 🧬A single shot of gene therapy restored hearing in 10 patients aged 1 to 24 with congenital deafness caused by OTOF gene mutations 🧬 While this is not the first Gene therapy for deafness, the surprise was the 14- and 24-year-old participants saw dramatic improvements, something researchers didn’t expect based on previous studies 🧬 Average hearing improved from 106 decibels (profound deafness) to 52 (functional range) within six months 🧬 Hearing gains were often rapid, with most patients showing improvement within the first month 🧬 One 7-year-old recovered enough to hold daily conversations and hear rain for the first time, despite having a cochlear implant in the other ear 🧬 The therapy uses an adeno-associated virus to deliver a working copy of the OTOF gene through a single injection to the inner ear #digitalhealth

Gene therapy just gave a 7-year-old her hearing back. Not partial. Not assistive. Actual restored hearing, after a single injection into her inner ear. Researchers used an AAV vector to deliver a working copy of the OTOF gene. Within four months, she could engage in daily conversation. Her hearing threshold improved from 106 decibels to 52. This wasn’t a mouse study. It worked in children and adults - ages 1 to 24. And it’s starting to feel like we see a new success story from CGT almost every day. Why this matters: → One of the first gene therapies to reverse sensory loss → Real human results, not just biomarkers → A future where single-dose cures aren’t sci-fi—they’re clinical reality Hearing loss affects over 430 million people worldwide. This trial just cracked open the door to something bigger.

One injection. A lifetime of sound. A clinical trial just proved #gene therapy can restore hearing in children and adults with OTOF-related congenital #deafness. One 16-minute inner-ear injection delivered a working OTOF gene and reconnected toddlers to voices, teens to music, adults to life. 🔹 5 of 6 regained conversational hearing 🔹 Both ears treated, sound localization restored 🔹 No lifelong device just one shot, one cure Opal, 18 months old, heard her parents for the first time weeks later. Six months in, her soft-sound hearing is nearly normal. And this isn’t a moonshot. With trials underway in the US, UK, China, and Regeneron’s DB-OTO showing promise, we may be nearing global access. Insight: Nearly 200,000 people are born with genetic deafness each year—most still rely on implants. Imagine a future where: • Children speak, connect, and thrive without delay • Adults reclaim voices they’ve never heard • AAV-based therapies treat dozens of rare hearing loss types What do you think: Should we fast-track gene therapies with high functional outcomes? Harvey Castro, MD, MBA. #DrGPT #GeneTherapy #RegenerativeMedicine #CongenitalDeafness #BiotechBreakthrough

Eleven-year-old Aissam Dam, born deaf in Morocco, recently became the first person in the US to undergo #genetherapy for #congenitaldeafness at the Children's Hospital of Philadelphia. Aissam lived in a silent world, but following this remarkable treatment can now hear, telling interviewers “There’s no sound I don’t like”. The trial aims to replace the mutated otoferlin gene in the inner ear with a functional one. Although challenges exist, including societal views within the Deaf community, patient enrollment timelines, and the availability of cochlear implants, Aissam's remarkable results indicate the potential of gene therapy in treating congenital deafness. Researchers, led by companies like Eli Lilly and Company and Akouos see this as a pioneering step that may lead to therapies for various forms of congenital deafness. An incredible milestone for both patients suffering from #rarecongenitaldisorders and the implications of gene therapy as a whole!