Wilson Disease - Pipeline Insight, 2024

This “Wilson’s Disease - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Wilson’s Disease 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

Wilson ’s disease Understanding

Wilson ’s disease: Overview

Wilson disease is a rare genetic disorder characterized by excess copper stored in various body tissues, particularly the liver, brain, and corneas of the eyes. The disease is progressive and, if left untreated, it may cause liver (hepatic) disease, central nervous system dysfunction, and death. Common signs of associated liver disease include a yellow discoloration (jaundice) of the skin, mucous membranes and the membranes (sclera) that line the eye, swelling (edema) of the legs and abdomen (ascites). Wilson disease is inherited as an autosomal recessive trait. Wilson disease is caused by disruption or changes (mutations) of the ATP7B gene, which plays an important role in the movement of excess copper from the liver to the bile to eventually be excreted from the body through the intestines. Treatment for Wilson disease is life-long and aimed at lowering copper levels to nontoxic levels, and at preventing the progression of the disease and trying to reverse any signs and symptoms that have appeared because of copper accumulation in the body.

Wilson’s Disease - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Wilson’s Disease pipeline landscape is provided which includes the disease overview and Wilson’s Disease treatment guidelines. The assessment part of the report embraces, in depth Wilson’s disease 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, Wilson’s Disease 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 Wilson’s Disease R&D. The therapies under development are focused on novel approaches to treat/improve Wilson’s Disease.

Wilson’s Disease Emerging Drugs Chapters

This segment of the Wilson’s Disease 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.

Wilson’s Disease Emerging Drugs

ALXN1840: Alexion PharmaceuticalsALXN1840 (bis-choline tetrathiomolybdate) is a novel oral copper-protein binding agent with a unique mechanism of action, under investigation for Wilson disease, a rare, chronic, genetic, and potentially life-threatening liver disorder of impaired copper transport. Enrollment of participants have been completed in a Phase 3 study of ALXN1840 in Wilson disease. The study results are expected in the first half of 2021.

TETA 4HCL: OrphalanTriethylenetatramine (TETA) is a highly selective divalent Cu(II) chelator and a drug that revereses copper overload in tissues. Trientine is a copper chelator used in the treatment of Wilson's disease as an alternative to D-penicillamine. Clinical trials for TETA 4HCL are being carried out in phase III stage of development for the treatment of Wilsons disease.

Wilson’s Disease: Therapeutic Assessment

This segment of the report provides insights about the different Wilson’s Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Wilson’s Disease

There are approx. 5+ key companies which are developing the therapies for Wilson’s Disease. The companies which have their Wilson’s Disease drug candidates in the most advanced stage, i.e. phase III include, Alexion Pharmaceuticals.

Phases

This report covers around 5+ 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

Wilson’s Disease 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
• Subcutaneous
• Intravenous
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Bispecific Antibody
• Peptides
• Small molecule
• Gene therapy
• Product Type

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

Wilson’s Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Wilson’s Disease 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 Wilson’s Disease drugs.

Wilson’s Disease Report Insights

• Wilson’s Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Wilson’s Disease 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 Wilson’s Disease drugs?
• How many Wilson’s Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Wilson’s Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Wilson’s Disease 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 Wilson’s Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

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