Beta-Thalassemia Disease Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018

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Beta-Thalassemia Disease Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018



Beta-Thalassemia is an inherited blood disorder characterized by decreased levels of functional hemoglobin, which is the iron containing protein present in red blood cells that carries oxygen to the cells throughout the body. People with Beta-Thalassemia will be suffering from lack of oxygen supply in body and also shortage of red blood cells and will be at the increased risk of developing abnormal blood clots.

Symptoms of Beta-Thalassemia includes pale skin, jaundice, enlarged spleen, liver and heart, delayed puberty. Beta-Thalassemia can be diagnosed by molecular and genetic tests, blood tests etc. Treatment of Beta-Thalassemia includes frequent blood transfusions, bone marrow transplant, surgery to remove spleen or gallbladder and medications.


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By Trial Phase, Beta-Thalassemiapipeline drugs are segmented as:

·         Preclinical Trials

·         Phase 1

·         Phase 2

·         Phase 3

·         Phase 4

By Company, Beta-Thalassemiapipeline drugs are segmented as:

·         Celgene Pharmaceuticals

·         bluebird bio.Inc

·         GlaxoSmithKline

·         Acceleron Pharma, Inc.

·         Sanofi

·         Novartis

·         HemaQuest Pharmaceuticals Inc.

·         Hoffmann-La Roche

·         FerroKinBioSciences, Inc.

·         Others

By Drugs, Beta-Thalassemia pipeline drugs are segmented as:

·         LentiGlobin

·         Deferitrin

·         Desferoxamine

·         Bitopertin

·         Deferasirox

·         Luspatercept

·         Zoledronic acid

·         Ambrisentan

·         Others

By Type of Condition, Beta-Thalassemia pipeline drugs are segmented as:

·         Thalassemia Major (Cooley’s anemia)

·         Thalassemia Intermedia

By Route of Administration, Beta-Thalassemiapipeline drugs are segmented as:

·         Oral

·         Parenteral


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Space Analysis:

·         In October 2017, Sangamo Therapeutics and Bioverativreceived U.S.FDA IND acceptance to begin human clinical studies of ST-400, a gene-edited cell therapy candidate for people with transfusion-dependent beta-thalassemia.

·         In September 2016, bluebird bio, Inc. commenced a phase III global multi center study of its HGB-207  in patients with transfusion-dependent beta-thalassemia with non-β00genotypes

·         In May 2015, Luspatercept of Celgene Corporation and Acceleron Pharma Inc. was granted Fast track designations by U.S. FDA for two separate indications, for the treatment of patients with transfusion dependent beta-thalassemia and patients with non-transfusion dependent beta-thalassemia. Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members of the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation


Report Description:

Beta-ThalassemiaDisease Pipeline Drugs Assessment report studies the various therapeutics under clinical development for Beta-Thalassemia treatment along with targets for various drug candidate. The report provides plethora of information pertaining to trail phases, companies involved in the Beta-Thalassemia disease pipeline drugs development. This report studies the dynamics of the Beta-Thalassemia Disease Pipeline Drugs i.e. drivers, challenges and opportunities which are significantly impacting the product development. The report provides various information pertaining the clinical trials such as designation, grants, patents, and technology among others. Moreover, the report on Beta-Thalassemia disease pipeline drugs assessment comprehensively presents the geographic location, trial status information along with key players involved in the therapeutics development.


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Key Features of the Report:

·         Provides the information related to universities and research institutes working in the therapeutics development

·         Report comprehensively covers the all active and discontinued studies

·         Studies the entire pipeline with special emphasis on companies actively involved in the therapeutics development

·         Presents the prominent targets for drug development in each stage of clinical trial

·         Provides the in-depth analysis on the each drug candidates in the clinical trial phases


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