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The Usher syndrome market was valued at USD 1.79 Billion in 2025 and is expected to grow at a CAGR of 3.30% reaching USD 2.48 Billion by 2035. The market growth is driven by the rising genetic screening programs and increasing awareness about inherited retinal diseases across the 8 major markets.
Base Year
Historical Period
Forecast Period
The rising prevalence of Usher syndrome, with over 721,000 patients worldwide, is driving demand for advanced diagnostics, genetic testing, and targeted therapies.
Breakthroughs in gene and RNA therapies, such as TIGEM’s AAV success and Sepul Bio’s LUNA trial, are reshaping treatment pipelines for Usher syndrome.
Dual-AAV innovations targeting PCDH15 and USH2A genes are accelerating the shift toward precision medicine in rare genetic disorders.
Compound Annual Growth Rate
3.3%
Value in USD Billion
2026-2035
*this image is indicative*
The market is evolving steadily, driven by advancements in genetic research and rising awareness of rare inherited disorders. Usher syndrome, characterized by combined hearing loss and progressive vision impairment, represents a significant unmet medical need. The increasing prevalence, alongside improved diagnostic capabilities through genetic testing, has heightened focus on early intervention. Furthermore, ongoing clinical trials exploring gene therapy and novel treatment approaches are expanding potential therapeutic options. Collectively, these factors are shaping the market growth trajectory. The market is expected to grow at a CAGR of 3.30% during the forecast period of 2026-2035 and attain a market value of USD 2.48 Billion by 2035.
Rising Global Prevalence of Usher Syndrome to Drive Market Expansion
The increasing prevalence of Usher syndrome is a significant factor fueling market growth. A comprehensive genomic analysis in February 2025 reported a global prevalence of 1 in 29,000 individuals, amounting to around 721,769 affected patients worldwide, with 12,090 newborns diagnosed annually. Additionally, 1% of the global population carries a pathogenic USH variant, with USH2A being the most common gene, showing a 1 in 150 carrier rate. This growing patient pool highlights the urgent need for advanced diagnostic tools, genetic testing, and innovative therapies, accelerating market opportunities worldwide.
Major market trends include emerging genetic and RNA-based therapies with transformative innovations.
Gene Therapy Breakthroughs Driving Market Growth
The market is witnessing robust momentum with recent breakthroughs in gene therapy. For instance, in July 2025, researchers at TIGEM reported early clinical success using a dual-vector AAV therapy for Usher syndrome type 1B. The therapy significantly improved vision in a treated patient after 12 months, with no severe adverse events. Such advancements demonstrate the market’s shift toward innovative genetic treatments for inherited retinal diseases, fueling investment opportunities and expanding the therapeutic pipeline for rare sensory-neurodegenerative disorders.
RNA-Based Therapies Transforming Usher Syndrome Pipeline
The development of RNA therapies is reshaping the Usher syndrome treatment landscape. In December 2024, Sepul Bio initiated the Phase 2b LUNA trial of ultevursen, an RNA-based therapy designed for exon 13 mutations in the USH2A gene. This milestone trial represents one of the most advanced targeted approaches for Usher syndrome type 2, signaling the rise of precision medicine in rare genetic disorders. Growing clinical success in RNA-based interventions is expected to attract significant partnerships and investment, driving accelerated market growth.
Dual-AAV Therapies Expanding Prospects in Usher Syndrome Treatment
Dual-AAV gene therapy research is creating new opportunities in the Usher syndrome market. In November 2024, Harvard Medical School scientists demonstrated promising outcomes for Usher syndrome type 1F by targeting the PCDH15 gene with a dual-vector approach. Preclinical results showed restoration of auditory and visual functions in animal and organoid models, underscoring the therapy’s potential to address dual sensory impairment. This advancement highlights a growing focus on complex gene therapy solutions, positioning the market for rapid expansion in innovative treatments.
Cochlear Implants to Lead the Market by Treatment
Among treatment options, cochlear implants are expected to hold the largest market share due to their effectiveness in addressing severe hearing loss, a core symptom of Usher syndrome. Unlike hearing aids or assistive listening devices, cochlear implants directly stimulate the auditory nerve, offering significant improvements in communication and quality of life. Increasing adoption is further supported by technological advancements, favorable reimbursement policies, and expanding surgical accessibility. This positions cochlear implants as the dominant treatment choice, driving substantial market growth.
In the United States, the market benefits from advanced genomic testing, robust funding for rare disease research, and strong adoption of hearing technologies like cochlear implants. The growing clinical trials in gene therapy are further strengthening market potential. In Japan, rapid advancements in precision diagnostics, coupled with strong governmental support for innovative therapies, drive significant opportunities. The country’s high adoption of assistive devices and investments in vision and hearing restoration research make Japan a pivotal growth hub for Usher syndrome treatment innovation.
The market report offers a detailed analysis of the market based on the following segments:
Market Breakup by Types
Market Breakup by Treatment
Market Breakup by End User
Market Breakup by Distribution Channel
Market Breakup by Region
The key features of the market report comprise clinical trials analysis, patent analysis, funding and investment analysis, and strategic initiatives by the leading players. The major companies in the market are as follows:
Headquartered in New Brunswick, New Jersey, and formally established as Johnson & Johnson MedTech in 2022, J&J originates from the 1886-founded medical devices branch. The MedTech division offers diverse emergency and surgical solutions, including advanced wound care and trauma devices. Leading the medical device sector, it reported over USD 31 billion in annual sales as of 2023. Its vast R&D investments and recent brand unification drive global innovation in injury management and first aid capabilities.
Founded around 2016 and headquartered in both London and New York, MeiraGTx is a clinical-stage genetic medicines company focusing on ocular, neurodegenerative, and salivary gland disorders. Its pipeline includes advanced gene therapies addressing inherited retinal diseases. In January 2025, the FDA granted Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for Leber congenital amaurosis, signaling near-term innovation in vision restoration, impactful for future sensory-impairment interventions.
Established in June 2003 and headquartered in Tokyo, Japan, Nobelpharma specializes in developing pharmaceuticals and medical devices for rare and unmet medical needs. Its strategic model emphasizes late-stage development and licensing therapies for orphan diseases. Nobelpharma America, its U.S. subsidiary based in Bethesda, Maryland, was certified a Great Place to Work® in 2023, highlighting organizational strength in rare-disease innovation.
Headquartered at Macquarie University, Sydney, Australia, Cochlear Ltd. was founded in 1981 and spun off as a public entity in 1995. As the global leader in implantable hearing solutions, its portfolio includes cochlear implants, bone-conduction implants, and accessory systems. It has delivered over 600,000 implantable devices globally and invests more than AUD 180 million annually in R&D, ensuring continuous innovation and expansion across over 180 countries.
*Please note that this is only a partial list; the complete list of key players is available in the full report. Additionally, the list of key players can be customized to better suit your needs.*
Other key players in the market include Advanced Bionics AG, Amgen Inc., ProQR Therapeutics NV, Zounds Hearing and Sivantos Pte. Ltd., Audina Hearing Instruments Inc., and Century Hearing Aids.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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| REPORT FEATURES | DETAILS |
| Base Year | 2025 |
| Historical Period | 2019-2025 |
| Forecast Period | 2026-2035 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Types |
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| Breakup by Treatment |
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| Breakup by End User |
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| Breakup by Distribution Channel |
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| Breakup by Region |
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| Market Dynamics |
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| Supplier Landscape |
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| Companies Covered |
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