DelveInsight’s “Wiskott-Aldrich Syndrome Pipeline Insight 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in the Wiskott-Aldrich Syndrome pipeline landscape. It covers the Wiskott-Aldrich Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Wiskott-Aldrich Syndrome therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Wiskott-Aldrich Syndrome Pipeline Report
- On December 10, 2025 — Fondazione Telethon announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy developed for patients with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening immunodeficiency.
- DelveInsight’s Wiskott-Aldrich Syndrome pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Wiskott-Aldrich Syndrome treatment.
- The leading Wiskott-Aldrich Syndrome Companies such as Orchard Therapeutics PLC, Genethon and others.
- Promising Wiskott-Aldrich Syndrome Therapies such as Dextrose, 5% in Water, Flebogamma 5% DIF, Cyclosporine and others.
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The Wiskott-Aldrich Syndrome Pipeline Report provides a disease overview, pipeline scenario, and therapeutic assessment of the key pipeline therapies in this domain. The Wiskott-Aldrich Syndrome Pipeline Report also highlights the unmet needs with respect to the Wiskott-Aldrich Syndrome.
Wiskott-Aldrich Syndrome Overview
Wiskott-Aldrich Syndrome (WAS) is a rare, inherited primary immunodeficiency disorder characterized by a triad of eczema, recurrent infections, and low platelet counts (thrombocytopenia). It is caused by mutations in the WAS gene, which affects the function of immune cells and platelets, leading to immune system dysfunction and abnormal bleeding. Because the immune system is compromised, individuals with WAS are more vulnerable to bacterial, viral, and fungal infections. The condition can also increase the risk of autoimmune diseases and certain blood cancers. WAS is typically diagnosed in early childhood and primarily affects males, as the gene is located on the X chromosome. Treatment may include supportive care, immunoglobulin replacement, platelet transfusions, and in severe cases, hematopoietic stem cell transplantation or gene therapy.
Wiskott-Aldrich Syndrome Emerging Drugs Profile
- 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-Aldrich Syndrome.
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The Wiskott-Aldrich Syndrome Pipeline Report Provides Insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Wiskott-Aldrich Syndrome with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Wiskott-Aldrich Syndrome Treatment.
- Wiskott-Aldrich Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Wiskott-Aldrich Syndrome Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Wiskott-Aldrich Syndrome market
Wiskott-Aldrich Syndrome Companies
Orchard Therapeutics PLC, Genethon and others.
The 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
Wiskott-Aldrich Syndrome Products have been categorized under various Molecule types such as,
- Small molecules
- Natural metabolites
- Monoclonal antibodies
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Scope of the Wiskott-Aldrich Syndrome Pipeline Report
- Coverage- Global
- Wiskott-Aldrich Syndrome Companies- Orchard Therapeutics PLC, Genethon and others.
- Wiskott-Aldrich Syndrome Therapies- Dextrose, 5% in Water, Flebogamma 5% DIF, Cyclosporine and others.
- Wiskott-Aldrich Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Wiskott-Aldrich Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Table of Contents
- Introduction
- Executive Summary
- Wiskott-Aldrich Syndrome: Overview
- Pipeline Therapeutics
- Therapeutics Assessment
- Wiskott-Aldrich Syndrome – DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Wiskott-Aldrich Syndrome Collaboration Deals
- Late Stage Products (Phase III)
- Otl-103: Orchard Therapeutics Plc
- Pre-clinical and Discovery Stage Products
- Inactive Products
- Wiskott-Aldrich Syndrome Key Companies
- Wiskott-Aldrich Syndrome Key Products
- Wiskott-Aldrich Syndrome- Unmet Needs
- Wiskott-Aldrich Syndrome- Market Drivers and Barriers
- Wiskott-Aldrich Syndrome- Future Perspectives and Conclusion
- Wiskott-Aldrich Syndrome Analyst Views
- Wiskott-Aldrich Syndrome Key Companies
- 20. Appendix
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