This year’s Hearing Therapeutics Summit brought together leading voices from research, industry, and patient communities to explore the breakthroughs shaping the future of the hearing therapeutics landscape.
From gene therapy to investment strategies, the event highlighted both the promise and the challenges of this rapidly evolving field.
A new era for hearing therapeutics
Hearing loss and tinnitus affect millions worldwide, yet treatment options remain limited. The Summit underscored a growing consensus: hearing therapeutics is entering a transformative phase. Advances in genetics, regenerative medicine, and drug development are paving the way for interventions that could prevent, restore, or even reverse hearing loss.
Gene therapy takes centre stage
Several sessions focused on the potential of gene therapy to tackle inherited forms of hearing loss:
- Norrie Disease: Researchers presented compelling pre-clinical evidence that delivering a corrected NDP gene can prevent hearing loss in mouse models. With orphan drug designation secured, clinical trial applications are on the horizon.
- GJB2 mutations: Sensorion’s novel gene therapy aims to restore hearing in patients with connexin-26 mutations—responsible for nearly half of congenital deafness cases. Animal studies show promising results, and regulatory submissions to enable clinical trials are expected in early 2026.
- Otoferlin-related hearing loss: The CHORD trial is testing intracochlear gene therapy in children with profound hearing loss, marking a milestone for rare genetic conditions.
These developments signal a shift from scientific concept to clinical application, offering hope for families affected by genetic hearing disorders.
Beyond genes: expanding the therapeutic toolkit
Innovation isn’t limited to genetic approaches. Researchers shared exciting progress in other areas:
- Transcriptomics for drug discovery: RNID-funded research identified candidate drugs, that may slow progressive hearing loss by targeting dysregulated genes.
- Stress granules and hair cell survival: researchers revealed how stress granules—molecular structures that regulate protein production—could protect hair cells under stress, opening new avenues for ototoxicity treatments.
- Regenerative cell therapy: Rinri Therapeutics is advancing Rincell-1, a cell therapy designed to restore auditory neurons, addressing conditions where cochlear implants fall short.
These approaches highlight the diversity of strategies needed to tackle the complexity of hearing loss.
Investment and market access
Despite vast unmet need, hearing therapeutics still struggles to attract investment. Key challenges include:
- Lack of standardised diagnostics and outcome measures
- Unclear regulatory pathways
- Limited success stories to build confidence
Yet great opportunity exists. Gene therapy and rare disease indications are gaining traction, and age-related hearing loss represents a multi-billion-dollar market. As one panellist noted:
We need one breakthrough to push the field forward.”
Tinnitus: an unmet need
Lived experience voices at the summit emphasized the profound impact of tinnitus. Current treatments, including sound therapy and CBT, offer limited relief. Promising developments include implanted stimulators and novel drug therapies, but the field remains underfunded and poorly understood. Researchers called for greater investment and collaboration to accelerate progress.
Building confidence through collaboration
The summit reinforced the importance of cross-sector partnerships. Priorities include:
- Core outcome measures: Standardising endpoints to enable meaningful comparisons and big data analysis.
- Patient-reported outcomes: Incorporating patient perspectives into regulatory processes to ensure treatments address real-world needs.
- Data sharing and ecosystem building: Creating repositories to accelerate research and clinical translation.
As one expert put it:
We need pharma companies on board as well as investors—hearing loss must become strategically important.”
Looking ahead
The momentum is undeniable. From pioneering gene therapies to novel drug and cell-based approaches, the hearing therapeutics field is entering a new phase of opportunity. But success will depend on collaboration, investment, and a shared commitment to making hearing loss and tinnitus treatable conditions.
Thanks to our sponsors and supporters
Shionogi – Gold Sponsor
Acousia Therapeutics – Silver Sponsor
Sensorion – Evening Reception Sponsor
Turner Scientific – Digital Agenda Sponsor
Norgine – Grant Supporter*
*Norgine provided an unrestricted grant as funding and had no input into the content or organisation of this event.
