openPR Logo
Press release

Wiskott-Aldrich Syndrome Clinical Trial Pipeline Expands as 5+ Companies Driving Innovation in the Therapeutics | DelveInsight

12-12-2025 12:28 AM CET | Business, Economy, Finances, Banking & Insurance

Press release from: ABNewswire

Wiskott-Aldrich Syndrome Clinical Trial Pipeline Expands as 5+

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.

Discover the latest drugs and treatment options in the Wiskott-Aldrich Syndrome Pipeline. Dive into DelveInsight's comprehensive report today! @ Wiskott-Aldrich Syndrome Pipeline Outlook [https://www.delveinsight.com/sample-request/wiskott-aldrich-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

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.

Stay ahead with the most recent pipeline outlook for Wiskott-Aldrich Syndrome. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Wiskott-Aldrich Syndrome Treatment Drugs [https://www.delveinsight.com/sample-request/wiskott-aldrich-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

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.

Explore groundbreaking therapies and clinical trials in the Wiskott-Aldrich Syndrome Pipeline. Access DelveInsight's detailed report now! @ New Wiskott-Aldrich Syndrome Drugs [https://www.delveinsight.com/sample-request/wiskott-aldrich-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

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

Unveil the future of Wiskott-Aldrich Syndrome Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Wiskott-Aldrich Syndrome Market Drivers and Barriers [https://www.delveinsight.com/sample-request/wiskott-aldrich-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

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

Get the latest on Wiskott-Aldrich Syndrome Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Wiskott-Aldrich Syndrome Companies, Key Products and Unmet Needs [https://www.delveinsight.com/sample-request/wiskott-aldrich-syndrome-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

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

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=wiskottaldrich-syndrome-clinical-trial-pipeline-expands-as-5-companies-driving-innovation-in-the-therapeutics-delveinsight]
Phone: 09650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/report-store/wiskott-aldrich-syndrome-pipeline-insight

Legal Disclaimer: Information contained on this page is provided by an independent third-party content provider. ABNewswire makes no warranties or responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you are affiliated with this article or have any complaints or copyright issues related to this article and would like it to be removed, please contact retract@swscontact.com



This release was published on openPR.

Permanent link to this press release:

Copy
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.

You can edit or delete your press release Wiskott-Aldrich Syndrome Clinical Trial Pipeline Expands as 5+ Companies Driving Innovation in the Therapeutics | DelveInsight here

News-ID: 4312302 • Views:

More Releases from ABNewswire

Dilated Cardiomyopathy Treatment Pipeline Shows Strong Momentum as 8+ Pharma Companies in the Race | DelveInsight
Dilated Cardiomyopathy Treatment Pipeline Shows Strong Momentum as 8+ Pharma Com …
DelveInsight's "Dilated Cardiomyopathy Pipeline Insight 2025" report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in the Dilated Cardiomyopathy pipeline landscape. It covers the Dilated Cardiomyopathy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Dilated Cardiomyopathy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Discover the latest drugs and treatment
Facioscapulohumeral Muscular Dystrophy Clinical Trial Pipeline Shows Potential with Active Contributions from 10+ Key Companies | DelveInsight
Facioscapulohumeral Muscular Dystrophy Clinical Trial Pipeline Shows Potential w …
DelveInsight's "Facioscapulohumeral Muscular Dystrophy Pipeline Insight 2025" report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in the Facioscapulohumeral Muscular Dystrophy pipeline landscape. It covers the Facioscapulohumeral Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Facioscapulohumeral Muscular Dystrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Discover the
Small Cell Lung Cancer Clinical Trial Pipeline Accelerates as 100+ Pharma Companies Rigorously Develop Drugs for Market Entry | DelveInsight
Small Cell Lung Cancer Clinical Trial Pipeline Accelerates as 100+ Pharma Compan …
DelveInsight's, "Small Cell Lung Cancer Pipeline Insight 2025" report provides comprehensive insights about 100+ companies and 100+ pipeline drugs in Small Cell Lung Cancer pipeline landscape. It covers the Small Cell Lung Cancer pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Small Cell Lung Cancer pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products
Patrick Huston PA, Realtor Solidifies Position as Premier Realtor Cape Coral Residents Trust for Exceptional Real Estate Service and Market-Leading Results
Patrick Huston PA, Realtor Solidifies Position as Premier Realtor Cape Coral Res …
Realtor in Cape Coral - Patrick Huston PA, Realtor. Patrick Huston PA, recognized as Cape Coral's premier realtor, announces continued market leadership through exceptional customer service and proven results. Operating from 1715 Cape Coral Pkwy W #14, this trusted real estate expert delivers personalized strategies for buyers and sellers. Contact (239) 222-9676 or visit https://patrickmyrealtor.com for superior real estate guidance. CAPE CORAL, FL - December 11, 2024 - Patrick Huston PA,

All 5 Releases


More Releases for Syndrome

"Understanding Rett Syndrome: Trends and Advances in the Rett Syndrome Market"
The Rett Syndrome Market, valued at USD 225.02 million in 2022, is anticipated to surge to USD 515.06 million by 2029, reflecting a significant Compound Annual Growth Rate (CAGR) of 10.23% from 2023 to 2029. Overview of the Rett Syndrome Market: Rett syndrome, a rare genetic neurological and developmental disorder primarily impacting females, hinders brain development and causes progressive loss of motor skills and language. The market report underscores technological advancements
MISME Syndrome Market - Navigating Challenges, Embracing Abilities: MISME Syndro …
Newark, New Castle, USA: The "MISME Syndrome Market" provides a value chain analysis of revenue for the anticipated period from 2023 to 2031. The report will include a full and comprehensive analysis of the business operations of all market leaders in this industry, as well as their in-depth market research, historical market development, and information about their market competitors. MISME Syndrome Market: https://www.growthplusreports.com/report/misme-syndrome-market/8883 This latest report researches the industry structure, sales, revenue,
Felty Syndrome Market - A Dedicated Approach to Felty Syndrome Care and Empowerm …
Newark, New Castle, USA: The "Felty Syndrome Market" provides a value chain analysis of revenue for the anticipated period from 2023 to 2031. The report will include a full and comprehensive analysis of the business operations of all market leaders in this industry, as well as their in-depth market research, historical market development, and information about their market competitors. Felty Syndrome Market: https://www.growthplusreports.com/report/felty-syndrome-market/8440 This latest report researches the industry structure, sales, revenue,
MISME Syndrome Market - Transforming Lives: Empowering Recovery from MISME Syndr …
Newark, New Castle, USA - new report, titled MISME Syndrome Market The report has been put together using primary and secondary research methodologies, which offer an accurate and precise understanding of the MISME Syndrome market. Analysts have used a top-down and bottom-up approach to evaluate the segments and provide a fair assessment of their impact on the global MISME Syndrome market. The report offers an overview of the market, which
Hepatorenal Syndrome Market - Reimagining Wellness, Redefining Survival: Hepator …
Newark, New Castle, USA - new report, titled Hepatorenal Syndrome Market The report has been put together using primary and secondary research methodologies, which offer an accurate and precise understanding of the Hepatorenal Syndrome market. Analysts have used a top-down and bottom-up approach to evaluate the segments and provide a fair assessment of their impact on the global Hepatorenal Syndrome market. The report offers an overview of the market, which
Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syn …
Global Markets Directs, Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) - Pipeline Review, H2 2016, provides an overview of the Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome) pipeline landscape. The report provides comprehensive information on the therapeutics under development for Atypical Hemolytic Uremic Syndrome (Nondiarrhea- Associated Hemolytic Uremic Syndrome), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and