Usher Syndrome Pipeline Insight

DelveInsight’s, “Usher syndrome - Pipeline Insight, 2025” report provides comprehensive insights about 5+ companies and 6+ pipeline drugs in Usher syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Geography Covered

• Global coverage

 

Usher syndrome: Understanding

Usher syndrome: Overview

Usher syndrome is a rare, inherited disorder causing combined hearing and vision loss, and sometimes balance issues. It is the leading genetic cause of deaf-blindness, with three clinical types (USH1, USH2, USH3) varying in symptom onset and severity. USH1 is the most severe, involving congenital hearing loss, early vision decline, and vestibular dysfunction. Mutations in at least 11 genes, including MYO7A and USH2A, are known to impair sensory cells in the ear and retina. At least nine genes are confirmed to be involved, with different genes linked to each of the three clinical types. USH1 genes such as MYO7A and CDH23 cause severe congenital hearing loss, early-onset vision loss, and balance issues. USH2 genes like USH2A and ADGRV1 lead to moderate-to-severe hearing loss with later-onset vision loss. USH3, caused by CLRN1 mutations, presents with variable onset and progressive hearing and vision decline. The affected proteins are crucial for the proper function of hair cells and photoreceptors, and mutations impair these systems. Symptoms include congenital or progressive sensorineural hearing loss, progressive vision loss due to retinitis pigmentosa, and vestibular dysfunction. The severity and onset of symptoms vary based on the genetic subtype and individual mutations.

The diagnosis of Usher syndrome requires both clinical evaluation and molecular genetic testing due to its genetic and phenotypic complexity. Clinically, diagnosis is based on identifying sensorineural hearing loss, retinitis pigmentosa, and, in some cases, vestibular dysfunction. Audiometric, ophthalmologic, and vestibular assessments help classify the syndrome into USH1, USH2, or USH3. Molecular diagnosis has advanced significantly with the use of targeted next-generation sequencing (NGS), which allows efficient detection of mutations across all known Usher-related genes. Additional techniques like MLPA and minigene assays help identify large deletions or confirm splicing defects missed by sequencing. Early genetic diagnosis enhances counseling, clinical management, and access to emerging gene-specific therapies.

Treatment research for Usher syndrome is rapidly advancing, with a primary focus on gene therapy, RNA-based therapies, and pharmacological interventions. Gene therapies using dual AAV vectors are being developed to deliver large genes like MYO7A and PCDH15, showing promising results in animal models and early clinical trials. RNA-based treatments, such as antisense oligonucleotides, are being explored to correct splicing errors, particularly in USH2A mutations. Antioxidants like N-acetylcysteine amide are in trials aiming to slow retinal degeneration by reducing oxidative stress. Preclinical models using stem cells and organoids are enabling more precise testing of therapies.

 

"Usher syndrome- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Usher syndrome pipeline landscape is provided which includes the disease overview and Usher syndrome treatment guidelines. The assessment part of the report embraces, in depth Usher syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Usher syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Usher syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Usher syndrome.

 

Usher syndrome Emerging Drugs Chapters

This segment of the Usher syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

Usher syndrome Emerging Drugs

• Ultevursen: Laboratoires Thea

Ultevursen (formerly QR-421a) is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. Ultevursen is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. It is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA. Currently, the drug is being evaluated in Phase II stage of its clinical trial.

 Further product details are provided in the report……..

 

Usher syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Usher syndrome drugs segregated based on following parameters that define the scope of the report, such as:

• Major  Players in Usher syndrome
• There are approx. 5+ key companies which are developing the therapies for Usher syndrome. The companies which have their Usher syndrome drug candidates in the most advanced stage, i.e. Phase II include, Laboratoires Thea.

 

Phases

DelveInsight’s report covers around 6+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

 

Route of Administration

Usher syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

 

Usher syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Usher syndrome therapeutic drugs key players involved in developing key drugs.

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Usher syndrome drugs.

 

Usher syndrome Report Insights

• Usher syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

 

Usher syndrome Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

 

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Usher syndrome drugs?
• How many Usher syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Usher syndrome?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Usher syndrome therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Usher syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?