Usher Syndrome - Pipeline Insight, 2022

This Usher Syndrome - Pipeline Insight, 2022” report provides comprehensive insights about 5+ companies and 8+ 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), an autosomal recessive genetic disease, is characterized by deafness due to an impaired ability of the inner ear and auditory nerves to transmit sensory (sound) input to the brain (sensorineual hearing loss) accompanied by retinitis pigmentosa, a disorder that affects the retina and causes progressive loss of vision. Usher syndrome (USH) comprises a group of autosomal recessively inherited disorders characterized by a dual sensory impairment of the audiovestibular and visual systems. Three major clinical subtypes (USH type I, USH type II and USH type III) are distinguished on the basis of the severity of the hearing loss, the presence or absence of vestibular dysfunction and the age of onset of retinitis pigmentosa (RP). USH, first described by the Scottish ophthalmologist, Charles Usher, is the leading genetic cause of deaf-blindness with a prevalence in the range of 1-4 per 25,000 people.

Usher syndrome is characterized by deafness due to an impaired ability of the inner ear and auditory nerves to transmit sensory (sound) input to the brain (sensorineural hearing loss) as well as abnormal accumulation of colored (pigmented) material on the nerve-rich membrane (the retina) lining the eyes (retinitis pigmentosa or RP). RP eventually causes retinal degeneration leading to progressive loss of vision and legal blindness. Sensorineural nerve deafness may be profound or mild, and may be progressive. The vision loss caused by RP may begin during childhood or later during life, and often first presents with difficulty seeing at night or in low light.

"Usher Syndrome - Pipeline Insight, 2022" report by the publisher 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.

This segment of the Usher Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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 Drugs

Ultevursen: ProQR Therapeutics

Ultevursen (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. Ultevursen is intended to be administered through intravitreal injections in the eye. Currently the product is in Phase II/III stage of development for the treatment of Usher Syndrome.

Usher Syndrome: Therapeutic Assessment

This segment of the report provides insights about the 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 ProQR Therapeutics.

Phases

The report covers around 8+ 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

• Intranasal
• Intrathecal
• Intravenous
• Oral
• Oral/Intravenous
• Parenteral
• Subcutaneous
• Subcutaneous/Intramuscular
• Transdermal
• Molecule Type

Products have been categorized under various Molecule types such as

• Antisense oligonucleotide
• Gene therapy
• Hormones
• Neuropeptides
• Oligonucleotides
• Small Molecule
• Triglyceride
• 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 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 Answered

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

• Editas Medicine
• Atsena Therapeutics
• Nacuity Pharmceuticals
• ProQR Therapeutics

Key Products

• EDIT-102
• ATSN-301
• NPI-001
• Ultevursen