Thalassemia - Pipeline Insight, 2024

This “Thalassemia- Pipeline Insight, 2024,”report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in Thalassemia 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

Thalassemia Understanding

Thalassemia: Overview

Thalassemia is a group of inherited blood disorders that affect the body’s ability to produce hemoglobin and red blood cells when the body doesn’t make enough of a protein called hemoglobin, an important part of red blood cells. Thalassemia is caused by mutations in the DNA of cells that make hemoglobin - the substance in red blood cells that carries oxygen throughout the body. The mutations associated with thalassemia are passed from parents to children.

Hemoglobin molecules are made of chains called alpha and beta chains that can be affected by mutations. In thalassemia, the production of either the alpha or beta chains are reduced, resulting in either alpha-thalassemia or beta-thalassemia. Symptoms include fussiness, paleness, frequent infections, failure to thrive, poor appetite, and jaundice. Treatment includes blood transfusions and bone marrow transplants.

The symptoms of this disease usually appears in the childhood the disease can be diagnosed depending upon the severity range of symptoms i.e. by complete blood count that includes measures of hemoglobin and size of red blood cells, a reticulocyte count that may indicate that bone marrow is not producing enough red blood cells, and genetic testing to diagnose the other types of thalassemia.

The treatment options available for this disease includes frequent blood transfusions on weekly basis that will blood transfusions cause a buildup of iron in bloodstream, which can further damage the heart, liver and other organs secondly is the chelation therapy this treatment is done to remove excess iron from the blood. Iron can build up as a result of regular transfusions some people suffering with thalassemia who don't have regular transfusions can also develop excess iron removing the excess iron is vital for health other treatment option is stem cell transplant also called a bone marrow transplant, a stem cell transplant might be an option in some cases. For children with severe thalassemia, it can eliminate the need for lifelong blood transfusions and drugs to control iron overload. This procedure involves receiving infusions of stem cells from a compatible donor, usually a sibling.

Thalassemia- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Thalassemia pipeline landscape is provided which includes the disease overview and Thalassemia treatment guidelines. The assessment part of the report embraces, in depth Thalassemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Thalassemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Thalassemia R&D. The therapies under development are focused on novel approaches to treat/improve Thalassemia.

Thalassemia Emerging Drugs Chapters

This segment of the Thalassemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Thalassemia Emerging Drugs

Mitapivat: Agios Pharmaceuticals, Inc Mitapivat is the potential drug candidate which is a quinolone sulfonamide administered through oral route as a capsule and tablet. It targets pyruvate kinase receptor (PKR).Currently it is in Phase III stage of clinical trial evaluation to treatThalassemia.

VIT-2763: Vifor Pharma Group VIT-2763 is an orally administered small molecule developed by Vifor Pharma. An oral metal-transporting-protein-1-inhibitors ferroportin by inhibiting hepcidin binding to ferroportin and blocks iron efflux. Currently the drug is being evaluated in Phase II for the treatment ofThalassemia.

BRL-101: BRL MEDICINEBLR-101 is a type of hematopoietic stem cell therapy based on HBB gene stimulants (Hemoglobin beta chain gene stimulants) mechanism of action that targets
ß-globin (Hemoglobin beta chain) developed for the treatment of patients suffering with thalassemia. This drug candidate is currently in Phase I stage of clinical studies.

Thalassemia: Therapeutic Assessment

This segment of the report provides insights about the different Thalassemia drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Thalassemia

There are approx. 30+ key companies which are developing the therapies for Thalassemia. The companies which have their Thalassemia drug candidates in the most advanced stage, i.e. phase III include, Agios Pharmaceuticals, Inc

Phases

This report covers around 30+ products under different phases of clinical development like

• Late stage products (Phase II)
• 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

Thalassemia 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
• intravenous
• Subcutaneous
• 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.

Thalassemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III II, I, preclinical and discovery stage. It also analyses Thalassemia 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 Thalassemia drugs.

Thalassemia Report Insights

• Thalassemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Thalassemia 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 Thalassemia drugs?
• How many Thalassemia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Thalassemia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Thalassemia 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 Thalassemia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Agios Pharmaceuticals, Inc
• CRISPR Therapeutics
• Vifor Pharma Group
• BRL MEDICINE
• Sangamo Therapeutics
• Vifor (International) Inc
• Celgene
• Pharmacosmos A/S
• Bioray Laboratories
• DisperSol Technologies, LLC

Key Products

• Mitapivat
• CTX001
• Vamifeport
• BRL-101
• ST-400
• VIT-2763
• Luspatercept
• SP-420
• BRL-101
• DST-0509

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