This “Immunologic Deficiency Syndrome - Pipeline Insight, 2021,” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Immunologic Deficiency 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.
Immunological deficiency syndromes are diseases or conditions in which there is a loss of or defect in a component of the immune system. Patients with these syndromes have an increased susceptibility to infections and to developing certain cancers. These syndromes include primary immune deficiency diseases, such as severe combined immunodeficiency, and acquired immunodeficiency syndrome (AIDS). There are two types of immunodeficiency disorders: Primary and Secondary. Primary disorders are usually present at birth and are genetic disorders that are usually hereditary. The secondary disorders generally develop later in life and often result from use of certain drugs or from another disorder, such as diabetes or human immunodeficiency virus (HIV) infection.
People with an immunodeficiency disorder tend to have one infection after another. Usually, respiratory infections (such as sinus and lung infections) develop first and recur often. Most people eventually develop severe bacterial infections that persist, recur, or lead to complications. Infections of the mouth, eyes, and digestive tract are common. People with certain immunodeficiency disorders may have many large, noticeable warts (caused by viruses). Many people have fevers and chills and lose their appetite and/or weight. Abdominal pain may develop, possibly because the liver or spleen is enlarged. Other symptoms vary depending on the severity and duration of the infections.
A diagnosis of Immunologic Deficiency Syndrome is made based upon a thorough clinical evaluation, a detailed patient history, and a variety of specialized tests. Laboratory tests are needed to confirm the diagnosis of immunodeficiency and to identify the type of immunodeficiency disorder. Blood tests, skin tests and biopsy are helpful in diagnosis. Genetic testing will help in identifying any gene mutation or mutations that cause many immunodeficiency disorders.
Treatment of immunodeficiency disorders usually involves preventing infections, treating infections when they occur, and replacing parts of the immune system that are missing when possible. Antimicrobials, such as antibiotics and antifungals, are medications that fight bacterial or fungal infections. Inability to produce adequate amounts or functional immunoglobulin or antibodies may benefit from replacement therapy with gamma globulins. Other treatments for some types of immune deficiencies are bone marrow transplants, hematopoietic cell transplantation, thymus transplantation, gene therapy, enzyme replacement and biologic cytokine inhibitors are examples of other treatments. Researchers in immunology are experimenting with gene therapy and other treatments that will be available in the future.
This segment of the Immunologic Deficiency 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.
Mavorixafor - Sanofi/X4 Pharmaceuticals
Mavorixafor is a first-in-class, oral, allosteric inhibitor of CXCR4 for the treatment of WHIM syndrome. WHIM syndrome is a rare genetic primary immunodeficiency that results from a “gain of function” mutation in the single gene that encodes for the CXCR4 receptor. In June 2019, European Commission has granted orphan drug designation (ODD) to Mavorixafor (X4P-001) for the treatment of WHIM syndrome. In October 2018, mavorixafor was granted ODD for the treatment of WHIM syndrome by the USFDA. The drug is currently in Phase 3 development for the treatment of WHIM syndrome.
Leniolisib - Novartis
Leniolisib (CDZ173) is a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. CDZ173 inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). The Dutch drugmaker, Pharming is developing leniolisib under license agreement from Novartis and is currently evaluating the treatment in a phase 2/3 trial. In October 2021, Leniolisib has received an Orphan Drug designation from the European Commission for the treatment of a rare, genetic immunodeficiency disease known as activated phosphoinositide 3-kinase delta syndrome (APDS).
There are approx. 40+ key companies which are developing the therapies for Immunologic Deficiency Syndrome. The companies which have their Immunologic Deficiency Syndrome drug candidates in the most advanced stage, i. e. phase III include, X4 Pharmaceuticals.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Immunologic Deficiency Syndrome therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Immunologic Deficiency Syndrome drugs.
Geography Covered
- Global coverage
Immunologic Deficiency Syndrome Understanding
Immunologic Deficiency Syndrome: Overview
Immunological deficiency syndromes are diseases or conditions in which there is a loss of or defect in a component of the immune system. Patients with these syndromes have an increased susceptibility to infections and to developing certain cancers. These syndromes include primary immune deficiency diseases, such as severe combined immunodeficiency, and acquired immunodeficiency syndrome (AIDS). There are two types of immunodeficiency disorders: Primary and Secondary. Primary disorders are usually present at birth and are genetic disorders that are usually hereditary. The secondary disorders generally develop later in life and often result from use of certain drugs or from another disorder, such as diabetes or human immunodeficiency virus (HIV) infection.
Symptoms
People with an immunodeficiency disorder tend to have one infection after another. Usually, respiratory infections (such as sinus and lung infections) develop first and recur often. Most people eventually develop severe bacterial infections that persist, recur, or lead to complications. Infections of the mouth, eyes, and digestive tract are common. People with certain immunodeficiency disorders may have many large, noticeable warts (caused by viruses). Many people have fevers and chills and lose their appetite and/or weight. Abdominal pain may develop, possibly because the liver or spleen is enlarged. Other symptoms vary depending on the severity and duration of the infections.
Diagnosis
A diagnosis of Immunologic Deficiency Syndrome is made based upon a thorough clinical evaluation, a detailed patient history, and a variety of specialized tests. Laboratory tests are needed to confirm the diagnosis of immunodeficiency and to identify the type of immunodeficiency disorder. Blood tests, skin tests and biopsy are helpful in diagnosis. Genetic testing will help in identifying any gene mutation or mutations that cause many immunodeficiency disorders.
Treatment
Treatment of immunodeficiency disorders usually involves preventing infections, treating infections when they occur, and replacing parts of the immune system that are missing when possible. Antimicrobials, such as antibiotics and antifungals, are medications that fight bacterial or fungal infections. Inability to produce adequate amounts or functional immunoglobulin or antibodies may benefit from replacement therapy with gamma globulins. Other treatments for some types of immune deficiencies are bone marrow transplants, hematopoietic cell transplantation, thymus transplantation, gene therapy, enzyme replacement and biologic cytokine inhibitors are examples of other treatments. Researchers in immunology are experimenting with gene therapy and other treatments that will be available in the future.
Immunologic Deficiency Syndrome Emerging Drugs Chapters
This segment of the Immunologic Deficiency 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.
Immunologic Deficiency Syndrome Emerging Drugs
Mavorixafor - Sanofi/X4 Pharmaceuticals
Mavorixafor is a first-in-class, oral, allosteric inhibitor of CXCR4 for the treatment of WHIM syndrome. WHIM syndrome is a rare genetic primary immunodeficiency that results from a “gain of function” mutation in the single gene that encodes for the CXCR4 receptor. In June 2019, European Commission has granted orphan drug designation (ODD) to Mavorixafor (X4P-001) for the treatment of WHIM syndrome. In October 2018, mavorixafor was granted ODD for the treatment of WHIM syndrome by the USFDA. The drug is currently in Phase 3 development for the treatment of WHIM syndrome.
Leniolisib - Novartis
Leniolisib (CDZ173) is a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially anti-neoplastic activities. CDZ173 inhibits the production of phosphatidylinositol-3-4-5-trisphosphate (PIP3). The Dutch drugmaker, Pharming is developing leniolisib under license agreement from Novartis and is currently evaluating the treatment in a phase 2/3 trial. In October 2021, Leniolisib has received an Orphan Drug designation from the European Commission for the treatment of a rare, genetic immunodeficiency disease known as activated phosphoinositide 3-kinase delta syndrome (APDS).
Immunologic Deficiency Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Immunologic Deficiency Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Immunologic Deficiency Syndrome
There are approx. 40+ key companies which are developing the therapies for Immunologic Deficiency Syndrome. The companies which have their Immunologic Deficiency Syndrome drug candidates in the most advanced stage, i. e. phase III include, X4 Pharmaceuticals.
Phases
This report covers around 40+ products under different phases of clinical development like
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Immunologic Deficiency 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
- Oral
- Parenteral
- intravitreal
- Subretinal
- Topical.
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Immunologic Deficiency Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Immunologic Deficiency 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 Immunologic Deficiency Syndrome drugs.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Immunologic Deficiency Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Immunologic Deficiency Syndrome.
- In August 2019, Pharming Group has entered into a development collaboration and license agreement with Novartis to develop and commercialize CDZ173, a small molecule phosphoinositide 3-kinase delta (PI3K?) inhibitor being developed by Novartis to treat patients with Activated Phosphoinositide 3-kinase Delta Syndrome (“APDS”).
Immunologic Deficiency Syndrome Report Insights
- Immunologic Deficiency Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Immunologic Deficiency Syndrome Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Immunologic Deficiency Syndrome drugs?
- How many Immunologic Deficiency Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Immunologic Deficiency Syndrome?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Immunologic Deficiency 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 Immunologic Deficiency Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Sanofi
- X4 Pharmaceuticals
- Novartis
- Rocket Pharmaceuticals
- Mustang Bio
- Bellicum Pharmaceuticals
- Lactiga
- GC Pharma
- Zynerba Pharmaceuticals
Key Products
- Mavorixafor
- Leniolisib
- RP L201
- MB-107
- Rivogenlecleucel
- LCTG-001
- GC-5101B
- Cannabidiol
Table of Contents
IntroductionExecutive Summary
Immunologic Deficiency Syndrome: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Immunologic Deficiency Syndrome - Analytical Perspective
In-depth Commercial Assessment
- Immunologic Deficiency Syndrome companies’ collaborations, Licensing, Acquisition - Deal Value Trends
Immunologic Deficiency Syndrome Collaboration Deals
- Company-Company Collaborations (Licensing / Partnering) Analysis
- Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
Mavorixafor: Sanofi/X4 Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II/III)
- Comparative Analysis
Leniolisib: Novartis
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I/II)
- Comparative Analysis
RP L201: Rocket Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Pre-clinical and Discovery Stage Products
- Comparative Analysis
LCTG-001: Lactiga
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Immunologic Deficiency Syndrome Key Companies
Immunologic Deficiency Syndrome Key Products
Immunologic Deficiency Syndrome- Unmet Needs
Immunologic Deficiency Syndrome- Market Drivers and Barriers
Immunologic Deficiency Syndrome- Future Perspectives and Conclusion
Immunologic Deficiency Syndrome Analyst Views
Immunologic Deficiency Syndrome Key Companies
AppendixList of Tables
Table 1 Total Products for Immunologic Deficiency Syndrome
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Immunologic Deficiency Syndrome
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Companies Mentioned
A selection of companies mentioned in this report includes:
- Sanofi
- X4 Pharmaceuticals
- Novartis
- Rocket Pharmaceuticals
- Mustang Bio
- Bellicum Pharmaceuticals
- Lactiga
- GC Pharma
- Zynerba Pharmaceuticals
