Usher Syndrome - Pipeline Insight, 2024

This “Usher Syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 7+ 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 (USH) is the most common deaf-blind syndrome, with 50% of deaf-blindness in persons younger than 65 years of age. It is a genetic condition that includes hearing loss, retinopathy (retinitis pigmentosa), and vestibular areflexia with different entities and onset. It is an autosomal recessive disorder - passing from parents to children - that impacts three major senses in the body: vision, hearing and balance. Balance disorders and bilateral vestibular areflexia are also observed in some cases. USH has been grouped into three main clinical types: 1, 2, and 3, which are caused by mutations in different genes and are further divided into different subtypes.

Diagnosis
Diagnosis of Usher Syndrome involves pertinent questions regarding the person’s medical history and testing of hearing, balance, and vision. A hearing (audiologic) exam measures the frequency and loudness of sounds that a person can hear. An electroretinogram measures the electrical response to the light-sensitive cells in the retina of the eyes. A retinal exam is done to observe the retina and other structures in the back of the eye. Vestibular (balance) function can be assessed by a variety of tests that evaluate different parts of the balance system. Genetic testing is clinically available for most of the genes associated with Usher Syndrome.

Treatment
Presently, there is no cure for Usher Syndrome. Treatment involves managing hearing, vision, and balance problems. Some researchers have shown that taking a specific daily dose of vitamin A may slow the progression of retinal degeneration in some people with typical RP and Usher Syndrome type 2. Typically, treatment for hearing management includes hearing aids, assistive listening devices, cochlear implants, or other communication methods. Although some ophthalmologists believe that a high dose of vitamin A palmitate may slow, but not halt the progression of retinitis pigmentosa. Other treatments such as gene therapy, drug based therapy, artificial organ replacement are currently leading the market. Ongoing research on the Usher proteins function in auditory and visual cells will be important in determining the optimal target for gene replacement therapy.

Usher Syndrome - Pipeline Insight, 2024 report 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 ThéaUltevursen (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 Usher Syndrome due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon-skipping approach with the aim of stopping or reversing vision loss in patients. Ultevursen 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, Ultevursen is being investigated in a Phase II/III trial for the treatment of Usher Syndrome.

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/III include, Laboratoires Théa.

Phases

This report covers around 7+ 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
• Parenteral
• intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• 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?

Key Players

• Laboratoires Théa
• Nacuity Pharmaceuticals
• Eli Lilly and Company
• Odylia Therapeutics

Key Products

• AAV- USH1C
• NPI-001
• Ultevursen
• AK-CLRN1

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