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Thalassemia Pipeline Analysis 2018 - Focusing on Clinical Trials and Results, Drug Profiling, Patents, Collaborations, and Other Recent Developments

  • ID: 4542603
  • Report
  • 98 pages
  • Knowledge Sourcing Intelligence LLP
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  • Acceleron Pharma Inc.
  • Biogen
  • Calimmune Inc.
  • Errant Gene Therapeutics
  • GlaxoSmithKline plc.
  • Novartis AG
  • MORE
Thalassemia Pipeline Analysis report covers more than 24 drugs currently in different phases of development. Thalassemia is an inherited blood disorder disease in which body makes abnormal hemoglobin. Hemoglobin consist of two protein subunits alfa and beta, if there is any mutation in the genes of these protein subunits leads to thalassemia. The disorder result in large number RBC being destroyed which leads to anemia. There are two main types of thalassemia: Alfa thalassemia and Beta thalassemia. Alfa thalassemia occurs when there is mutation in alfa subunit gene and beta thalassemia occurs when similar mutational effects occurs on the beta subunit gene. Symptoms include: fatigue, weakness, pale or yellowish skin, facial bone deformities, slow growth, abdominal swelling and dark urine.

The report provides thalassemia treatment drugs by company, phases of development including products in early discovery stage and NDA filing, molecule type, route of administration and region. The report will help to evaluate the collaboration, in-licensing and out-licensing opportunities, formulating business development strategies and tracking the activities of the key market players. Epidemiology, major drivers, restraints, and opportunities have been covered to provide an exhaustive picture of the market. The analysis presents in-depth information regarding the development, trends, and industry policies and regulations implemented to offer stakeholders a better understanding of the key factors affecting the overall market environment.

Various databases (for patents and Clinical Trials), studies and data published by industry associations, analyst reports, investor presentations, press releases and journals among others have been taken into consideration while conducting the secondary research.

Market intelligence is presented in the form of analysis, charts, and graphics to help the clients in gaining faster and efficient understanding of the market.

Major industry players profiled as part of the report are Novartis AG, F. Hoffmann-La Roche Ltd., GlaxoSmithKline plc., Celgene Corporation, Acceleron Pharma Inc., Sanofi, Sangamo Therapeutics Inc., bluebird bio, Inc., Cadila Healthcare Ltd., La Jolla Pharmaceuticals Company, Ionis Pharmaceuticals Inc., Imara Inc., Gamida Cell, Alnylam Pharmaceuticals, Vertex - CRISPR Therapeutics AG, Biogen, Protagonist Therapeutics, Inc., Errant Gene Therapeutics, Agios Inc., Silence Therapeutics, Calimmune, Inc., Editas Medicine, Vifor Pharma and Kiadis Pharma.

  • By Company
  • By Phase
  • By Molecule Type
  • By Region
  • By Route of Administration
Note: Product cover images may vary from those shown
2 of 4


  • Acceleron Pharma Inc.
  • Biogen
  • Calimmune Inc.
  • Errant Gene Therapeutics
  • GlaxoSmithKline plc.
  • Novartis AG
  • MORE
1. Introduction
1.1. Research Methodology
1.2. Research Scope
1.2.1. Analysis by Company
1.2.2. Analysis by Phase
1.2.3. By Molecule Type
1.2.4. By region

2. Disease Overview
2.1.1. Introduction
2.1.2. Classification
2.1.3. Symptoms
2.1.4. Causes
2.1.5. Diagnoses
2.1.6. Treatment
2.1.7. Epidemiology

3. Executive Summary

4. Market Dynamics
4.1. Drivers
4.2. Restraints
4.3. Opportunities

5. Pipeline Analysis/Outlook
5.1. Analysis by Company
5.2. Analysis by Phase
5.2.1. Phase of Development Introduction Drug Profiling
  • Drug Name
  • Generic Name
  • Synonyms
  • Company
  • Collaborator
  • Route of administration
  • Target
  • Mechanism of Action
  • Technology
  • Molecule type
  • CAS Number
  • Weight
  • Chemical Formula
  • IUPAC name
  • ATC code Strategic Developments Clinical trials Clinical trial results Patents Technology
5.2.2. Comparative Analysis for Trials by Phase (Pie, Bar graph)
5.3. By Molecule type
5.4. Analysis by Region

6. Company profiling
6.1. Novartis AG
6.1.1. Introduction
6.1.2. Financials
6.1.3. Products and Services
6.1.4. SWOT
6.2. F. Hoffmann-La Roche Ltd.
6.2.1. Introduction
6.2.2. Financials
6.2.3. Products and Services
6.2.4. SWOT
6.3. GlaxoSmithKline plc.
6.3.1. Introduction
6.3.2. Financials
6.3.3. Products and Services
6.3.4. SWOT
6.4. Celgene Corporation.
6.4.1. Introduction
6.4.2. Financials
6.4.3. Products and Services
6.4.4. SWOT
6.5. Acceleron Pharma Inc.,
6.5.1. Introduction
6.5.2. Financials
6.5.3. Products and Services
6.5.4. SWOT
6.6. Sanofi
6.6.1. Introduction
6.6.2. Financials
6.6.3. Products and Services
6.6.4. SWOT
6.7. Sangamo Therapeutics, Inc.
6.7.1. Introduction
6.7.2. Financials
6.7.3. Products and Services
6.7.4. SWOT
6.8. bluebird bio, Inc.,
6.8.1. Introduction
6.8.2. Financials
6.8.3. Products and Services
6.8.4. SWOT
6.9. Cadila Healthcare Ltd.
6.9.1. Introduction
6.9.2. Financials
6.9.3. Products and Services
6.9.4. SWOT
6.10. La Jolla Pharmaceuticals Company
6.10.1 Introduction
6.10.2 Financials
6.10.3 Product and Services
6.10.4 SWOT
6.11. Ionis Pharmaceuticals Inc.,
6.11.1 Introduction
6.11.2 Financials
6.11.3 Product and Services
6.11.4 SWOT
6.12 Imara Inc.
6.12.1 Introduction
6.12.2 Financials
6.12.3 Product and Services
6.12.4 SWOT
6.13 Gamida Cell
6.13.1 Introduction
6.13.2 Financials
6.13.3 Product and Services
6.13.4 SWOT
6.14 Alnylam Pharmaceuticals
6.14.1 Introduction
6.14.2 Financials
6.14.3 Product and Services
6.14.4 SWOT
6.15 Vertex - CRISPR Therapeutics AG
6.15.1 Introduction
6.15.2 Financials
6.15.3 Product and Services
6.15.4 SWOT
6.16 Biogen
6.16.1 Introduction
6.16.2 Financials
6.16.3 Product and Services
6.16.4 SWOT
6.17 Protagonist Therapeutics Inc.
6.17.1 Introduction
6.17.2 Financials
6.17.3 Product and Services
6.17.4 SWOT
6.18 Errant Gene Therapeutics
6.18.1 Introduction
6.18.2 Financials
6.18.3 Product and Services
6.18.4 SWOT
6.19 Agios Inc.
6.19.1 Introduction
6.19.2 Financials
6.19.3 Product and Services
6.19.4 SWOT
6.20 Silence Therapeutics
6.20.1 Introduction
6.20.2 Financials
6.20.3 Product and Services
6.20.4 SWOT
6.21 Calimmune Inc.
6.21.1 Introduction
6.21.2 Financials
6.21.3 Product and Services
6.21.4 SWOT
6.22 Editas Medicine
6.22.1 Introduction
6.22.2 Financials
6.22.3 Product and Services
6.22.4 SWOT
6.23 Vifor Pharma
6.23.1 Introduction
6.23.2 Financials
6.23.3 Product and Services
6.23.4 SWOT
6.24 Kiadis Pharma.
6.24.1 Introduction
6.24.2 Financials
6.24.3 Product and Services
6.24.4 SWOT

List of Tables
List of Figures
Note: Product cover images may vary from those shown
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4 of 4
  • Novartis AG
  • F. Hoffmann-La Roche Ltd.
  • GlaxoSmithKline plc.
  • Celgene Corporation
  • Acceleron Pharma Inc.
  • Sanofi
  • Sangamo Therapeutics, Inc.
  • bluebird bio, Inc.,
  • Cadila Healthcare Ltd.
  • La Jolla Pharmaceuticals Company
  • Ionis Pharmaceuticals Inc.
  • Imara Inc.
  • Gamida Cell
  • Alnylam Pharmaceuticals
  • Vertex - CRISPR Therapeutics AG
  • Biogen
  • Protagonist Therapeutics Inc.
  • Errant Gene Therapeutics
  • Agios Inc.
  • Silence Therapeutics
  • Calimmune Inc.
  • Editas Medicine
  • Vifor Pharma
  • Kiadis Pharma
Note: Product cover images may vary from those shown
5 of 4

The research study is based on the publisher's proprietary research model. The process involves sourcing of information from various industry experts, key professionals, decision-makers and various other stakeholders along with the validation of the responses received from the vast in-house database and premium paid databases. Publicly available sources were also taken into consideration for the final market estimates and forecast for the segments and sub-segments.

The first phase of the research process is the identification of key industry leaders, experts, decision-makers and C-level executives and then conducting interviews and surveys to gain vital insights about the market. Simultaneously, extensive secondary research is done, and key secondary data is collected as part of this process. This secondary data is collected from various databases, industry journals, whitepapers, company annual reports, research publications, and other paid data sources and relevant literature. Company profiles which include strategies, new product developments, recent investments, major products and services, financials and business plans are sourced from the annual reports, SEC filings, industry associations to preserve the quality of the information being displayed. The findings from both the primary and the secondary research process combined go under further processing for market breakdown and forecasting.

The last phase is providing intelligence in the form of presentation, charts, graphics and other different formats helping the clients in a faster and efficient understanding of the market. Under this phase complete market engineering is involved which includes analyzing the collected data from different sources and existing proprietary datasets while using various data triangulation methods for market breakdown and forecasting. The publisher has developed their market forecasting methodology that, in addition to the main market values, also takes into account other macroeconomic factors (global or local) that might have some bearing on the market under study. Taking all the necessary data, a robust forecast is developed using an algorithm that involves both static (multivariate) as well as dynamic (time-series) regression analysis. Special care is taken to make sure their algorithm passes the necessary statistical tests, and the mean prediction for future years obtained is reliable with a very high confidence level. Following data validation, the analysts begin to write the report presenting insights with the help of charts, graphs, and tables.