Beta-thalassemia - Pipeline Insight, 2024

This “Beta-thalassaemia- Pipeline Insight, 2024” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in Beta-thalassaemia 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

Beta-thalassaemia: Understanding

Beta-thalassaemia: Overview

Beta thalassemia is an inherited blood disorder that limits your body’s ability to make beta-globin. Beta-globin is an important protein needed to make hemoglobin and red blood cells. Beta thalassemia can cause the patient to experience anemia symptoms.

There are several types of thalassemia. The types include beta thalassemia major, beta thalassemia intermedia and beta thalassemia minor. The signs and symptoms you have depend on the type and severity of your condition. Thalassemia signs and symptoms may include fatigue, weakness, pale or yellowish skin, facial bone deformities, slow growth, abdominal swelling, and dark urine.

Thalassemia is caused by mutations in the DNA of cells that make hemoglobin - the substance in red blood cells that carries oxygen throughout your 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. In alpha-thalassemia, the severity of thalassemia you have depends on the number of gene mutations you inherit from your parents. The more mutated genes, the more severe your thalassemia and in beta-thalassemia, the severity of thalassemia you have depends on which part of the hemoglobin molecule is affected.

Beta thalassemia is often diagnosed in childhood. Beta thalassemia major, the most severe form, is diagnosed early in childhood, by age 2. The Healthcare professional diagnose beta thalassemia based on your symptoms and blood test results. The diagnosis may include the following tests.

A complete blood count (CBC): A CBC provides information about blood cells, including red blood cells. It can show whether the red blood cells are less than normal count, oddly shaped or pale (light red). These characteristics may be signs of thalassemia.- Reticulocyte count: Immature red blood cells are called reticulocytes. A low reticulocyte count indicates that the body isn’t producing enough red blood cells. Severe thalassemia is usually accompanied by an elevated reticulocyte count. This is because the body is trying to make more red cells to make up for the destruction of the red cells containing the abnormal hemoglobin.

Molecular genetic testing: Molecular genetic testing allows doctors to study hemoglobin closely and identify the mutation associated with beta thalassemia.- Hemoglobin electrophoresis: A hemoglobin electrophoresis test measures different types of hemoglobin proteins in blood. Certain types of hemoglobin proteins are increased with beta thalassemia, while other types are decreased.

The treatments options for beta- thalassemia may include:
Blood transfusions: Frequent blood transfusions (as much as every two weeks) with beta thalassemia major. During the procedure, blood is received from a donor. The influx of blood from a transfusion supplies red blood cells needed to carry oxygen to tissues throughout your body.- Iron chelation therapy: Iron is an important part of the hemoglobin protein allowing it to carry oxygen. Too much iron, however, can be harmful. Iron chelation therapy can help prevent iron overload.

Folic acid supplements: Folic acid can help boost your body’s ability to make red blood cells. Doctor may recommend supplements if the patient has beta thalassemia minor or folic acid in addition to receiving regular blood transfusions if the condition is more severe.- Luspatercept: In case of severe thalassemia, an injection (shot) of luspatercept is received every three weeks to help the body make more red blood cells. Luspatercept improves anemia in people diagnosed with beta thalassemia who are receiving blood transfusions.

Bone marrow and stem cell transplant: Replacement of bone marrow stem cells with the stem cells of a healthy donor can cure beta thalassemia. Unfortunately, finding a compatible donor can be challenging. Also, this type of transplant is considered a high-risk procedure.Beta-thalassaemia- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Beta-thalassaemia pipeline landscape is provided which includes the disease overview and Beta-thalassaemia treatment guidelines. The assessment part of the report embraces, in depth Beta-thalassaemia 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, Beta-thalassaemia 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 Beta-thalassaemia R&D. The therapies under development are focused on novel approaches to treat/improve Beta-thalassaemia.

Beta-thalassaemia Emerging Drugs Chapters

This segment of the Beta-thalassaemia 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.

Beta-thalassaemia Emerging Drugs

CTX001: CRISPR Therapeutics CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from ß-thalassemia and sickle cell disease. The drug is in Phase I/II clinical evaluation for the treatment of ß-thalassemia. CTX001 has been designated an orphan drug in the U.S. and Europe, and given fast track, rare pediatric disease, and regenerative medicine advanced therapy designations in the U.S. for SCD and transfusion-dependent Beta thalassemia (B-thal) (TDT).

VIT-2763: CSL Vifor VIT-2763 is an orally administered small molecule developed by Vifor Pharma. Intended for daily administration, VIT-2763 has the potential for treating diseases with impaired iron metabolism. Ferroportin is an iron transporter that plays a key role in regulating iron uptake and distribution in the body and thus in controlling iron levels in the blood. At the molecular level, VIT-2763 binds to ferroportin and blocks it to prevent excessive iron release into the blood. Pre-clinical evidence serving as the basis for the clinical development of VIT-2763 revolves around its efficacy for reducing elevated blood and tissue iron levels and for restricting iron uptake in patients suffering from conditions in which iron metabolism isaltered.

Vamifeport is currently in phase II development for beta-thalassemia, an inherited rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body and potentially cause anaemia.

Emeramide: EmeraMed Emeramide is an antioxidant heavy metal chelator. It prevents methylmercury-induced glutathione (GSH) loss, and cytotoxicity to, isolated mouse aortic endothelial cells when used at a concentration of 50 µM. Emeramide is a lipophilic, di-thiol antioxidant, anti-viral, and metal chelator. Orphan Drug Designations were received for the treatment of mercury toxicity in the EU and US. Pre-clinical safety studies, a Phase I trial, and four Phase II trials have been performed targeting multiple indications. Currently the drug is in phase II for the treatment ofbeta-thalassaemia.

Beta-thalassaemia: Therapeutic Assessment

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

Major Players in Beta-thalassaemia

There are approx. 22+ key companies which are developing the therapies for Beta-thalassaemia. The companies which have their Beta-thalassaemia drug candidates in the most advanced stage, i.e. pre-registration include, CRISPR Therapeutics.

Phases

This report covers around 22+ 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

Beta-thalassaemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Beta-thalassaemia: Pipeline Development Activities

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

Beta-thalassaemia Report Insights

• Beta-thalassaemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• CRISPR Therapeutics
• CSL Vifor
• Beam Therapeutics
• EmeraMed
• Fulcrum Therapeutics
• Editas Medicine
• Edi GeneInc
• Silence Therapeutics
• Phoenicia Biosciences
• Shanghai BDgene
• Disc Medicine

Key Products

• Exa-cel
• VIT-2763
• BEAM 101
• Emeramide
• FTX 6058
• EDIT 301
• ET-01
• SLN124
• PB-04
• BD 211
• DISC a

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