Immunologic Deficiency Syndrome - Pipeline Insight, 2024

This “Immunologic Deficiency Syndrome - Pipeline Insight, 2024,” 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.

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

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

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

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