Wiskott-Aldrich Syndrome - Pipeline Insight, 2024

This “Wiskott-Aldrich Syndrome - Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Wiskott-Aldrich 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

Wiskott-Aldrich Syndrome Understanding

Wiskott-Aldrich Syndrome: Overview

Wiskott-Aldrich Syndrome (WAS), an X-linked syndrome is characterized by the medical triad of micro thrombocytopenia, eczema, and recurrent infections. The disorder is caused by the mutation in WAS gene located on the short arm of X-chromosome (Xp11.4-p11.21). WAS disorder being rare has incidence rate of less than 1 in 100,000 population. This disorder most exclusively affects males.

Symptoms
Owing to abnormal functioning of immune system and reduced ability to form clots, individuals with WAS can exhibit various symptoms which include purpura, petechiae, eczema, skin diseases (cellulitis, impetigo), bloody diarrhea, mucosal bleeding, bleeding inside the brain that can be fatal.

Diagnosis
Diagnosis for Wiskott-Aldrich Syndrome is based on family history, physical examination and laboratory investigations to look for severe thrombocytopenia with reduced platelet size with a usually normal number of megakaryocytes, as well as altered antibody production. Absent or decreased WAS protein levels and genetic testing confirm the diagnosis.

Treatment
Currently, hematopoietic stem cell transplantation is the only curative treatment available for Wiskott-Aldrich Syndrome. However, symptoms such as infection, and severe eczema can be treated with the administration of immunoglobin replacement therapy or oral antibiotics, and topical, or short-term systemic steroids respectively. Gene therapy is still in clinical trials, and may be a promising treatment option in future.

Wiskott-Aldrich Syndrome Emerging Drugs Chapters

This segment of the Wiskott-Aldrich 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.

Wiskott-Aldrich Syndrome Emerging Drugs

OTL-103: Orchard Therapeutics plc Orchard’s OLT-103 is an ex vivo autologous gene therapy being investigated for the treatment of WAS. It uses a modified virus to insert a working copy of the WAS gene into a patient’s cells. The therapy is in its phase III clinical trials and has been designated with orphan drug status for Wiskott-AldrichSyndrome.

Wiskott-Aldrich Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Wiskott-Aldrich Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Wiskott-Aldrich Syndrome

There are approx. 5+ key companies which are developing the therapies for Wiskott-Aldrich Syndrome. The companies which have their Wiskott-Aldrich Syndrome drug candidates in the mid to advanced stage, i.e. phase III include, Orchard Therapeutics plc and others.

Phases

This report covers around 5+ products under different phases of clinical development like

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

Route of Administration

Wiskott-Aldrich 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

• Subcutaneous
• Intravenous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecules
• Natural metabolites
• Monoclonal antibodies

Product Type

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

Wiskott-Aldrich Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Wiskott-Aldrich 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 Wiskott-Aldrich Syndrome drugs.

Report Highlights

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

Wiskott-Aldrich Syndrome Report Insights

• Wiskott-Aldrich Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Orchard Therapeutics plc
• Genethon

Key Products

• OTL-103
• WAS gene therapy