Bharat Book Bureau Provides the Trending Market Research Report on “Beta-thalassemia (B-thal) - Pipeline Insight, 2020” under Life Sciences Category. The report offers a collection of superior market research, market analysis competitive intelligence and Market reports.


The “Beta thalassemia – Pipeline Insight Market 2020,” report provides comprehensive insights about 35+ companies and 35+ 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.

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.


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Geography Covered
• Global coverage
Beta thalassemia Understanding
Beta thalassemia: Overview

In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. 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.
Beta thalassemia is 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.


Symptoms
Children born with Beta thalassemia will have symptoms early in life that include:


• Pale skin
• Fussy
• Having a poor appetite
• Having many infections
• Over time more symptoms will appear, including:
• Slowed growth
• Belly (abdominal) swelling
• Yellowish skin (jaundice)


Diagnosis
Doctors diagnose thalassemias using blood tests, including a complete blood count (CBC) and special hemoglobin tests.
• A CBC measures the amount of hemoglobin and the different kinds of blood cells, such as red blood cells, in a sample of blood. People who have thalassemias have fewer healthy red blood cells and less hemoglobin than normal in their blood. People who have alpha or beta thalassemia trait may have red blood cells that are smaller than normal.
• Hemoglobin tests measure the types of hemoglobin in a blood sample. People who have thalassemias have problems with the alpha or beta globin protein chains of hemoglobin.
Treatment


The treatment involves folic acid supplementation in individuals with mild cases of anemia. Supplementation with folic acid, a B vitamin, boosts the production of red blood cells in certain individuals. Along with blood transfusions, folic acid may be given and medications to lower iron levels. In 2019, the Food and Drug Administration (FDA) approved Reblozyl (luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions. Individuals with beta thalassemia major require regular blood transfusions. A blood transfusion is a common procedure in which affected individuals receive donated blood in order to restore the levels of healthy, functioning hemoglobin to their blood.

Some individuals may be treated by the surgical removal of the spleen (splenectomy). An abnormally enlarged spleen (splenomegaly) can cause severe pain and contribute to anemia. Treatment of additional complications of beta thalassemia or iron overload is symptomatic and supportive.


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


• IMR-687: Imara
IMR-687 is a highly selective and potent small molecule inhibitor of PDE9. PDE9 selectively degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. Lower levels of cGMP are found in people with SCD and beta-thalassemia and are associated with reduced blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide mediated vasodilation. Blocking PDE9 acts to increase cyclic GMP levels, which is associated with reactivation of fetal hemoglobin, or HbF, a natural hemoglobin produced during fetal development.

Increased levels of HbF in RBCs have been demonstrated to improve symptomology and substantially lower disease burden in both patients with SCD and patients with β-thalassemia. It is currently in phase II stage of development.


• ST-400: Sangamo Therapeutics
ST-400 is being developed by Sangamo Therapeutics for the treatment of Beta Thalassemia. It is currnelty in phase I/II stage of development.
• SLN124: Silence Therapeutics
SLN124, which has been shown to lower serum iron levels and modulate tissue iron distribution in rodent models for β-Thalassemia and hereditary hemochromatosis,represents a promising therapeutic candidate for treating patients with iron overload disorders, including β-Thalassemia. It is currently in phase I stage of development.


• EDIT-301: Editas Medicine
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease and β-Thalassemia. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. It is in preclinical stage of development.


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. 35+ 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 II include Imara and others


• Phases
report covers around 35+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage products (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
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravenous
• Oral
• Parenteral
• Subcutaneous

• Molecule Type
Products have been categorized under various Molecule types such as
• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

• 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.

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.
• In June 2020 Imara announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia.


• In January 2019, Silence Therapeutics announces that its lead medicine candidate SLN124 has been granted Orphan Drug Designation by the Committee for Orphan Medicinal Products (COMP), the European Medicines Agency's (EMA).
• Sanofi and Sangamo are in partnership for developing gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease, using Sangamo’s zinc finger nuclease technology.
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
• Vifor
• Sangamo Therapeutics
• Acceleron Pharma
• Imara, Inc.
• Shanghai Bioray Laboratory Inc.
• bluebird bio
• EmeraMed
• Genzyme, a Sanofi Company
• La Jolla Pharmaceutical
• HemaQuest Pharmaceuticals
Key Products
• VIT-2763
• ST-400
• IMR-687
• ACE-536
• β-globin restored autologous HSC
• GT56-252
• LentiGlobin BB305 Drug Product
• LJPC-401
• HQK-1001

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https://www.bharatbook.com/marketreports/beta-thalassemia-b-thal-pipeline-insight-2020/2187333


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