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.
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.
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.
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.
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Report Coverage
The Wiskott-Aldrich Syndrome Drug Pipeline Insight Report by the publisher 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 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 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-103Sponsored 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.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?
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Table of Contents
1 Preface
3 Overview of Wiskott-Aldrich Syndrome
4 Patient Profile: Wiskott-Aldrich Syndrome
5 Wiskott-Aldrich Syndrome: Epidemiology Snapshot
6 Wiskott-Aldrich Syndrome: Market Dynamics
7 Wiskott-Aldrich Syndrome: Key Facts Covered
8 Wiskott-Aldrich Syndrome, Drug Pipeline Assessment
9 Drug Pipeline Comparative Analysis
10 Wiskott-Aldrich Syndrome Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
11 Wiskott-Aldrich Syndrome Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
12 Wiskott-Aldrich Syndrome Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
13 Wiskott-Aldrich Syndrome Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
14 Wiskott-Aldrich Syndrome, Key Drug Pipeline Companies
