Beta thalassemia - Pipeline Insight, 2021

Beta thalassemia - Pipeline Insight, 2021

  • March 2021 •
  • 60 pages •
  • Report ID: 6042283 •
  • Format: PDF
“Beta thalassemia - Pipeline Insight, 2021,” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in Beta 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

Beta thalassemia Understanding

Beta thalassemia: Overview
Beta thalassemia is an inherited blood disorder that reduces the production of hemoglobin. Affected individuals also have a shortage of red blood cells (anemia), which can cause pale skin, weakness, fatigue, and more serious complications. People with beta thalassemia are at an increased risk of developing abnormal blood clots. The characteristic finding of beta thalassemia is anemia, which is caused because red blood cells are abnormally small (microcytic), are not produced at the normal amounts, and do not contain enough functional hemoglobin. Beta thalassemia can be classified into two types depending on the severity of symptoms: thalassemia major (also known as Cooley’s anemia) and thalassemia intermedia. Of the two types, thalassemia major is more severe. Blood tests for diagnosis of Beta thalassemia can reveal the number of red blood cells and abnormalities in size, shape or color. Blood tests can also be used for DNA analysis to look for mutated genes. Mild forms of thalassemia trait don’t need treatment. For moderate to severe thalassemia, treatments might include: frequent blood transfusions, chelation therapy, and stem cell transplant.

"Beta thalassemia - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Beta thalassemia pipeline landscape is provided which includes the disease overview and Beta thalassemia treatment guidelines. The assessment part of the report embraces, in depth Beta 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, Beta 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 Beta thalassemia R&D. The therapies under development are focused on novel approaches to treat/improve Beta thalassemia.

Beta thalassemia Emerging Drugs Chapters
This segment of the Beta thalassemia 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 thalassemia Emerging Drugs
• CTX001: CRISPR Therapeutics
CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from ?-thalassemia and sickle cell disease. The drug is in Phase I/II clinical evaluation for the treatment of ?-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 (TDT).

• IMR-687: Imara
IMR-687, which is an oral, once-a-day, potentially disease-modifying treatment for sickle cell disease (SCD) and ?-thalassemia. IMR-687 is a highly selective, potent small molecule inhibitor of PDE9 (Phosphodiesterase 9A). Imara has evaluating the drug in Phase 2b clinical trial for patients with beta-thalassemia. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation, Rare Pediatric Disease designation, and Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia.
Further product details are provided in the report……..

Beta thalassemia: Therapeutic Assessment
This segment of the report provides insights about the different Beta thalassemia drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Beta thalassemia
There are approx. 22+ key companies which are developing the therapies for Beta thalassemia. The companies which have their Beta thalassemia drug candidates in the most advanced stage, i.e. Phase IIb include, Imara.
• 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 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
• 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.

Beta thalassemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Beta 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 Beta thalassemia drugs.

Beta thalassemia Report Insights
• Beta thalassemia Pipeline Analysis

• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players
• CRISPR Therapeutics
• Imara
• Silence Therapeutics
• Acceleron Pharma
• Celgene Corporation
• Kiadis Pharma
• Fulcrum Therapeutics
• Aruvant Sciences
• Editas Medicine
• EdiGene
• EpiDestiny
• Novo Nordisk

Key Products
• CTX001
• IMR-687
• SLN-124
• Sotatercept
• ATIR-101
• FTX-6058
• ARU-1801
• EDIT-301
• ET 01
• EPI 01