Explore Our Diverse Range Of Offerings
From detailed reports to experts services offered in 15+ Industry Domains
Report
Press Release
Blogs
Industry Statistics
Add 2 More Reports For 20% off

Report Overview

Wiskott-Aldrich syndrome is a rare X-linked recessive disease that causes immunological deficiency and reduced ability to form blood clots. The disease accounts for around 3% of all primary immunodeficiency disorders and has an estimated incidence (classic Wiskott-Aldrich syndrome phenotype) of 1 to 10 in 1 million cases per live birth. There is a high unmet clinical need for better therapies to treat Wiskott-Aldrich syndrome as current treatment options, such as bone marrow transplants, are limited by donor availability along with carrying significant complications. Further, the growing focus on targeted therapies, including gene and biologic-based treatments, are likely to support pipeline growth in the coming years.

  • Major companies involved in the Wiskott-Aldrich syndrome pipeline drugs market include Novartis Pharmaceuticals and GlaxoSmithKline, among others.

  • Leading drugs currently under the pipeline include OTL-103 and autologous CD34+ cells transduced with WASP lentiviral vector (haematopoietic stem cell gene therapy), among others.

  • The rising advancements in gene therapy and the robust support from regulatory bodies such as the U.S. Food and Drug Administration (FDA) are significantly contributing to the expansion of the Wiskott-Aldrich syndrome drug pipeline.

Report Coverage

The Wiskott-Aldrich Syndrome Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into Wiskott-Aldrich syndrome drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Wiskott-Aldrich syndrome. The report includes the analysis of over 100 pipeline drugs and 50+ companies. The Wiskott-Aldrich syndrome pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from Wiskott-Aldrich syndrome.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to Wiskott-Aldrich syndrome.

Wiskott-Aldrich Syndrome Drug Pipeline Outlook

Wiskott-Aldrich syndrome is a rare genetic disorder caused by a mutation in the gene encoding the Wiskott-Aldrich syndrome protein (WASP). The disease primarily affects boys, although their mother or sisters may carry one copy of the mutated gene. This X-linked recessive disorder leads to abnormal bleeding, frequent infections, rashes like eczema, and autoimmune issues. The severity of Wiskott-Aldrich syndrome ranges from classic to milder forms, with the classic form associated with autoimmunity, severe eczema, and bacterial and viral infections, among others.

Genetic testing is commonly used to identify gene mutations and diagnose the affected patient. Treatment options include prophylactic antibiotics, splenectomy, and immunoglobulin replacement therapy. To improve the survival rates of Wiskott-Aldrich syndrome patients, hematopoietic stem cell transplant is considered the gold standard. However, there remains a need for safer as well as more accessible therapies to effectively address the complications (autoimmune diseases and cancers) for patients worldwide.

Wiskott-Aldrich Syndrome – Drug Pipeline Therapeutic Assessment

This section of the report covers the analysis of Wiskott-Aldrich syndrome drugs based on several segmentations including:

By Phase

  • Late-Stage Products (Phase 3 and Phase 4)
  • Mid-Stage Products (Phase 2)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

By Drug Class

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies

By Route of Administration

  • Oral
  • Parenteral
  • Others

Wiskott-Aldrich Syndrome – Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II covers a major share of the total clinical trials for Wiskott-Aldrich syndrome.

Wiskott-Aldrich Syndrome – Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the Wiskott-Aldrich syndrome pipeline analysis include small molecules, monoclonal antibodies, and gene therapies. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Wiskott-Aldrich syndrome.

Wiskott-Aldrich Syndrome Clinical Trials Therapeutic Assessment – Competitive Dynamics

The EMR report for the Wiskott-Aldrich syndrome drug pipeline covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in Wiskott-Aldrich syndrome clinical trials:

  • Novartis Pharmaceuticals
  • GlaxoSmithKline
  • Grifols Therapeutics LLC
  • Genethon

Wiskott-Aldrich Syndrome – Emerging Drugs Profile

Major drugs currently in the drug pipeline are as follows:

Genetic: OTL-103

Sponsored by Fondazione Telethon, the objective of this Phase 3, open-label, single-arm study is to examine the efficacy of the cryopreserved formulation of OTL-103 gene therapy containing autologous CD34+ hematopoietic stem cell for the treatment of Wiskott-Aldrich syndrome. The study is expected to be completed by September 2027 and has an estimated 10 participants.

Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector

Genethon is conducting a Phase I/II clinical trial aimed at evaluating the haematopoietic stem cell gene therapy consisting of autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector for the treatment of Wiskott-Aldrich syndrome. The interventional study has enrolled an estimated 10 subjects and is expected to be completed by October 2032.

*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*

Reasons To Buy This Report

The Wiskott-Aldrich Syndrome Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for Wiskott-Aldrich syndrome. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within Wiskott-Aldrich syndrome pipeline insights.

Key Questions Answered in the Wiskott-Aldrich Syndrome – Pipeline Insight Report

  • What is the current landscape of Wiskott-Aldrich syndrome pipeline drugs?
  • How many companies are developing Wiskott-Aldrich syndrome drugs?
  • How many phase III and phase IV drugs are currently present in Wiskott-Aldrich syndrome pipeline drugs?
  • Which companies/institutions are leading the Wiskott-Aldrich syndrome drug development?
  • What is the efficacy and safety profile of Wiskott-Aldrich syndrome pipeline drugs?
  • What are the opportunities and challenges present in the Wiskott-Aldrich syndrome drug pipeline landscape?
  • Which company is conducting major trials for Wiskott-Aldrich syndrome drugs?
  • What geographies are covered for Wiskott-Aldrich syndrome clinical trials?
  • What are emerging trends in Wiskott-Aldrich syndrome clinical trials?

Related Reports

Immune Thrombocytopenia Market

Global Clinical Trials Market

*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*

Looking for specific insights?

Get in touch with us for a customized solution tailored to your unique requirements and save upto 35%!

Report Summary

Explore our key highlights of the report and gain a concise overview of key findings, trends, and actionable insights that will empower your strategic decisions.

Key Highlights of the Report

Please note that the figures mentioned in the description serve as estimates and may vary from the actual figures presented in the final report.

Scope of the Report

Details

Drug Pipeline by Clinical Trial Phase

  • Late-Stage Products (Phase III and Phase IV)
  • Mid-Stage Products (Phase II)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products

Route of Administration

  • Oral
  • Parenteral
  • Others

Drug Classes

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies

Leading Sponsors Covered

  • Novartis Pharmaceuticals
  • GlaxoSmithKline
  • Grifols Therapeutics LLC
  • Genethon

Geographies Covered

  • North America
  • Europe
  • Asia Pacific
  • Others

Mini Report

10 % Off

USD

1,999

1,799

Single User License

10 % Off

USD

3,099

2,789

Five User License

15 % Off

USD

4,599

3,909

Corporate License

15 % Off

USD

5,999

5,099

  • Purchase Advantages
  • Report Delivery
  • Access Duration
  • Analyst Support
  • Discount on Future Updates
  • Customisation Options
  • Customisation Requests
  • 1 Print Allowed
  • Upto 3 Sections
  • Bulk Purchase Discounts
  • Multi-Device Access
  • PDF Format
  • Online Access
  • Excel Data Sheet
  • PPT/Word Format
  • Unlimited Access
  • One-Time Purchase
  • 3-Month Trade Data Access
  • Consultation Hours
  • Email Support
  • Phone Support
  • Up to 15 Analyst Hours
  • Up to 30 Analyst Hours
  • 5% Discount
  • 10% Discount
  • 15% Discount
  • 20% Discount
  • Basic (Up to 10%)
  • Advanced (Up to 50%)
  • Dedicated Analyst Access
  • Data Visualization Access
  • Custom Data Queries
  • Custom Report Layouts
  • Tailored Custom Summaries
  • Dashboard
  • Purchase Advantages
  • Report Delivery
  • Access Duration
  • Analyst Support
  • Discount on Future Updates
  • Customisation Options
  • Customisation Requests
  • 1 Print Allowed
  • Full Report Access
  • Bulk Purchase Discounts
  • Multi-Device Access
  • PDF Format
  • Online Access
  • Excel Data Sheet
  • PPT/Word Format
  • Unlimited Access
  • One-Time Purchase
  • 3-Month Trade Data Access
  • Consultation Hours
  • Email Support
  • Phone Support
  • Up to 15 Analyst Hours
  • Up to 30 Analyst Hours
  • 5% Discount
  • 10% Discount
  • 15% Discount
  • 20% Discount
  • Basic (Up to 10%)
  • Advanced (Up to 50%)
  • Dedicated Analyst Access
  • Data Visualization Access
  • Custom Data Queries
  • Custom Report Layouts
  • Tailored Custom Summaries
  • Dashboard
  • Purchase Advantages
  • Analyst Support
  • Report Delivery
  • Access Duration
  • Customisation Options
  • Discount on Future Updates
  • Customisation Requests
  • 5 Prints Allowed
  • Full Report Access
  • Bulk Purchase Discounts
  • Limited Device Access
  • Email Support
  • Phone Support
  • Up to 15 Analyst Hours
  • Up to 30 Analyst Hours
  • PDF Format
  • Online Access
  • Excel Data Sheet
  • PPT/Word Format
  • Unlimited Access
  • One-Time Purchase
  • 3-Month Trade Data Access
  • Consultation Hours
  • Basic (Up to 10%)
  • Advanced (Up to 50%)
  • Dedicated Analyst Access
  • Data Visualization Access
  • 5% Discount
  • 10% Discount
  • 15% Discount
  • 20% Discount
  • Custom Data Queries
  • Custom Report Layouts
  • Tailored Custom Summaries
  • Dashboard
  • Report Delivery
  • Purchase Advantages
  • Customisation Options
  • Customisation Requests
  • Analyst Support
  • Access Duration
  • Discount on Future Updates
  • PDF Format
  • Excel Data Sheet
  • PPT/Word Format
  • Online Access
  • Unlimited Prints Available
  • Full Report Access
  • Bulk Purchase Discounts
  • Multi-Device Access
  • Basic (Up to 10%)
  • Advanced (Up to 50%)
  • Dedicated Analyst Access
  • Data Visualization Access
  • Custom Data Queries
  • Custom Report Layouts
  • Tailored Custom Summaries
  • Dashboard
  • Email Support
  • Phone Support
  • Up to 15 Analyst Hours
  • Up to 30 Analyst Hours
  • Unlimited Access
  • One-Time Purchase
  • 3-Month Trade Data Access
  • Consultation Hours
  • 5% Discount
  • 10% Discount
  • 15% Discount
  • 20% Discount

Mini Report

One User

USD 1,999

USD 1,799

tax inclusive*

  • Selected Segments
  • Printing Restrictions
  • Excel Spreadsheet Delivered via Email
  • Full Report
  • Periodic Updates
  • Post Sales Analysts Support
  • Unlimited Prints

Single User License

One User

USD 3,099

USD 2,789

tax inclusive*

  • All Segments
  • Printing Restrictions
  • PDF Delivered via Email
  • Custom Report Layout
  • Post Sales Analysts Support
  • Periodic Updates
  • Unlimited Prints

Five User License

Five User

USD 4,599

USD 3,909

tax inclusive*

  • All Segments
  • Five Prints Available
  • PDF Delivered via Email
  • Limited Free Customization
  • Post Sales Analyst Support
  • Custom Report Layout
  • Periodic Updates
  • Unlimited Prints

Corporate License

Unlimited Users

USD 5,999

USD 5,099

tax inclusive*

  • All Segments
  • Unlimited Prints Available
  • PDF & Excel Delivery via Email
  • Limited Free Customization
  • Post Sales Analysts Support
  • Discount On Next Update
  • Custom Report Layout

*Please note that the prices mentioned below are starting prices for each bundle type. Kindly contact our team for further details.*

Bundle Type

Flash Bundle

20% OFF Number of Reports: 3

Small Business Bundle

25% OFF Number of Reports: 5

Growth Bundle

30% OFF Number of Reports: 8

Enterprise Bundle

35% OFF Number of Reports: 10
Overview
  • Life Time Access
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours
  • Complimentary Free 1 Month Subscription to Trade Data Base
  • Complimentary One Month Subscription to Price Database (Chemicals only)
  • Complementary PPT Version of the Report
  • Complimentary License Upgrade
  • Complimentary Power BI Dashboards
  • Life Time Access
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours - 50 Hours
  • Complimentary Free 1 Month Subscription to Trade Data Base
  • Complimentary One Month Subscription to Price Database (Chemicals only)
  • Complementary PPT Version of the Report
  • Complimentary License Upgrade
  • Complimentary Power BI Dashboards
  • Life Time Access
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours - 80 Hours
  • Complimentary Free 1 Month Subscription to Trade Data Base
  • Complimentary One Month Subscription to Price Database (Chemicals only)
  • Complementary PPT Version of the Report
  • Complimentary License Upgrade
  • Complimentary Power BI Dashboards
  • Life Time Access
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours - 100 Hours
  • Complimentary Free 1 Month Subscription to Trade Data Base
  • Complimentary One Month Subscription to Price Database (Chemicals only)
  • Complementary PPT Version of the Report
  • Complimentary License Upgrade
  • Complimentary Power BI Dashboards

*Please note that the prices mentioned below are starting prices for each bundle type. Kindly contact our team for further details.*

Flash Bundle

Number of Reports: 3

20%

tax inclusive*

  • 3 Reports Included
  • Life Time Acess
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Free 1 Month Subscription to Trade Data Base
  • 1 Month Subscription to Price Database (Chemicals only)
  • Complimentary Excel Data Set
  • PPT Version of the Report
  • Power BI Dashboards
  • License Upgrade
  • Free Analyst Hours

Small Business Bundle

Number of Reports: 5

25%

tax inclusive*

  • 5 Reports Included
  • Life Time Acess
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours - 50 Hours
  • Free 1 Month Subscription to Trade Data Base
  • 1 Month Subscription to Price Database (Chemicals only)
  • Complimentary Excel Data Set
  • PPT Version of the Report
  • Power BI Dashboards
  • License Upgrade

Growth Bundle

Number of Reports: 8

30%

tax inclusive*

  • 8 Reports Included
  • Life Time Acess
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours - 50 Hours
  • Free 1 Month Subscription to Trade Data Base
  • 1 Month Subscription to Price Database (Chemicals only)
  • License Upgrade
  • Free Analyst Hours - 80 Hours
  • Power BI Dashboards

Enterprise Bundle

Number of Reports: 10

35%

tax inclusive*

  • 10 Reports Included
  • Life Time Acess
  • Analyst Support Related to Report
  • PDF Version of the Report
  • Complimentary Excel Data Set
  • Free Analyst Hours - 50 Hours
  • Free 1 Month Subscription to Trade Data Base
  • 1 Month Subscription to Price Database (Chemicals only)
  • License Upgrade
  • Power BI Dashboards
  • Free Analyst Hours - 100 Hours

How To Order

Our step-by-step guide will help you select, purchase, and access your reports swiftly, ensuring you get the information that drives your decisions, right when you need it.

Select License Type

Choose the right license for your needs and access rights.

Click on ‘Buy Now’

Add the report to your cart with one click and proceed to register.

Select Mode of Payment

Choose a payment option for a secure checkout. You will be redirected accordingly.

Strategic Solutions for Informed Decision-Making

Connect For More Information

Our expert team of analysts will offer full support and resolve any queries regarding the report, before and after the purchase.

Our expert team of analysts will offer full support and resolve any queries regarding the report, before and after the purchase.

We employ meticulous research methods, blending advanced analytics and expert insights to deliver accurate, actionable industry intelligence, staying ahead of competitors.

Our skilled analysts offer unparalleled competitive advantage with detailed insights on current and emerging markets, ensuring your strategic edge.

We offer an in-depth yet simplified presentation of industry insights and analysis to meet your specific requirements effectively.

We’re here to help answer any questions about our products and services.

Contact us

Our Offices


Australia

63 Fiona Drive, Tamworth, NSW

+61-448-061-727

India

C130 Sector 2 Noida, Uttar Pradesh 201301

+91-723-689-1189

Philippines

40th Floor, PBCom Tower, 6795 Ayala Avenue Cor V.A Rufino St. Makati City,1226.

+63-287-899-028, +63-967-048-3306

United Kingdom

6 Gardner Place, Becketts Close, Feltham TW14 0BX, Greater London

+44-753-713-2163

United States

30 North Gould Street, Sheridan, WY 82801

+1-415-325-5166

Vietnam

193/26/4 St.no.6, Ward Binh Hung Hoa, Binh Tan District, Ho Chi Minh City

+84-865-399-124

30 North Gould Street, Sheridan, WY 82801

+1-415-325-5166

63 Fiona Drive, Tamworth, NSW

+61-448-061-727

C130 Sector 2 Noida, Uttar Pradesh 201301

+91-723-689-1189

40th Floor, PBCom Tower, 6795 Ayala Avenue Cor V.A Rufino St. Makati City, 1226.

+63-287-899-028, +63-967-048-3306

6 Gardner Place, Becketts Close, Feltham TW14 0BX, Greater London

+44-753-713-2163

193/26/4 St.no.6, Ward Binh Hung Hoa, Binh Tan District, Ho Chi Minh City

+84-865-399-124