Thalassemia pipeline Research Monitor, 2020- Drugs, Companies, Clinical Trials, R&D pipeline updates, status and outlook

July 2020 | 145 pages | ID: TF883644DA51EN
VPAResearch

Abstracts

2020 Thalassemia PIPELINE HIGHLIGHTS
Thalassemia is one of the widely researched conditions during 2020 with 37 companies actively focusing on realizing pipeline’s potential. Development of Thalassemia medicines is identified as integral to the strategy of the majority of companies operating in the industry.
Global Thalassemia market presents promising new product pipeline with NME Projects, pivotal trials, and rapidly phase-advancing therapeutic candidates. Increasing number of companies are assessing the feasibility of developing treatment options for Thalassemia.

Good progress is anticipated during 2020 and 2021 with Thalassemia pipeline molecules advancing from pre-clinical investigation to completion of advanced Phase clinical trials. Thalassemia pipeline continues to expand and progress with novel mechanisms and diverse routes of administration being tested by companies.
Thalassemia DRUG DEVELOPMENT PIPELINE OVERVIEW
The “Thalassemia pipeline Research Monitor, 2020” report is an analytical research study on the progress achieved by pipeline companies during the year along with its historical development, current status, and outlook.
This Thalassemia pipeline review explores high-potential early to late-stage pipeline projects with a continued focus on new insights, accelerated processes, and pipeline progression.

The competitive intelligence report on Thalassemia presents detailed insights into therapeutic drug pipeline development, industry news, deals, and analysis across the length and breadth of the Thalassemia pipeline. Information on R&D pipeline updates, results of key clinical trials are also included in the report.
Thalassemia DRUG PROFILES
Thalassemia development pipeline including projects in early- and late-stage development are detailed in the report. Details of clinical trial data and submissions to regulatory authorities are also provided. For the drug candidates included in the report, the following information is provided-

• Current Status of Development including phase advancements, regulatory approvals of phases, acquisitions, licensing and technology transfers, product launches in various markets, and others.
• Phase of development
• Mechanism of Action
• Route of Administration
• Companies involved including originator, licensing companies, developer, investors, and others
• New molecular entity details
• Orphan drug designation and other special status provided by regulators

Thalassemia COMPANY PROFILES

Both small size and large size pharmaceutical companies are investing their resources in Thalassemia drug development operations. Further, financial institutions are extending support to small pharmaceutical companies, universities, and other researchers for the development of treatment of Thalassemia. Partnerships and acquisitions are also increasingly observed in the pipeline.

This research report presents an analysis of 37 Thalassemia companies including company overview, key snapshot, contact information, and their strategies on accelerating Thalassemia pipeline development. Mid-stage and early portfolios of these companies are analyzed in detail in the report.

Companies analyzed in the report include- Acceleron Pharma Inc, Agios Pharmaceuticals Inc, Allife Medical Science and Technology Co Ltd, Aruvant Sciences, ASC Therapeutics Inc, Beam Therapeutics Inc, bluebird bio Inc, Cetya Therapeutics Inc, CSL Behring, Disc Medicine Inc, EdiGene Inc, Editas Medicine Inc, EmeraMed Ltd, Errant Gene Therapeutics LLC, ExCellThera Inc, Fulcrum Therapeutics Inc, Gamida Cell Ltd, Gilead Sciences Inc, Imara Inc, Invenux LLC, Ionis Pharmaceuticals Inc, Kymab Group Ltd, Merganser Biotech Inc , Nanomedic Inc, Orchard Therapeutics Ltd, Phoenicia Biosciences Inc, Protagonist Therapeutics Inc, Rare Partners Srl, Regenacy Pharmaceuticals LLC, Sangamo Therapeutics Inc, Sanquin Plasma Products BV, Shanghai Bioray Laboratory Inc, Silence Therapeutics Plc, Syros Pharmaceuticals Inc, Trucode Gene Repair Inc, Vertex Pharmaceuticals Inc, Vifor Pharma AG

REASONS TO BUY

• The current market conditions are forcing companies and investors worldwide to prioritize their investments and accelerate pipeline candidates only through careful quantitative assessment.
• This research work assists decision makers in pharmaceutical companies towards successful pipeline development through critically evaluated pipeline data
• Buyers can identify most promising drug candidates for treatment of Thalassemia
• It allows users to strengthen their pipeline through acquisitions, licensing and collaborations
• Users can estimate possible delays in the delivery of pipeline or launch of new products
• Stay ahead of competition through understanding their pipeline progression, strategies and outlook
• The report details all the pipeline candidates under investigation in various stages of development from Phase I through to Phase III to enable business development managers to understand the impact of new launches.
• Optimize your licensing and technology transfer strategies through identification of prospect partners

Contents

1. INTRODUCTION TO THALASSEMIA

1.1 Thalassemia- Disease overview
1.2 Thalassemia- Market Size
1.3 Thalassemia- Companies Involved

2. THALASSEMIA PIPELINE SNAPSHOT- 2020

2.1 Thalassemia Pipeline by Phase
2.2 Thalassemia Pipeline by Mechanism of Action
2.3 Thalassemia Pipeline by Route of Administration
2.4 Thalassemia Pipeline- New Molecular Entities
2.5 Thalassemia Pipeline- Orphan Drug Designation/ Special Designation

3. THALASSEMIA DRUG PROFILES

3.1 Current Status
3.2 Thalassemia Drug Snapshot
3.3 Thalassemia Therapeutic Candidates- Originator/Licensor
3.4 Route of Administration
3.5 Mechanism of Action
3.6 NME
4.1 Acceleron Pharma Inc Thalassemia Pipeline Insights and Clinical Trials
4.2 Agios Pharmaceuticals Inc Thalassemia Pipeline Insights and Clinical Trials
4.3 Allife Medical Science and Technology Co Ltd Thalassemia Pipeline Insights and Clinical Trials
4.4 Aruvant Sciences Thalassemia Pipeline Insights and Clinical Trials
4.5 ASC Therapeutics Inc Thalassemia Pipeline Insights and Clinical Trials
4.6 Beam Therapeutics Inc Thalassemia Pipeline Insights and Clinical Trials
4.7 bluebird bio Inc Thalassemia Pipeline Insights and Clinical Trials
4.8 Cetya Therapeutics Inc Thalassemia Pipeline Insights and Clinical Trials
4.9 CSL Behring Thalassemia Pipeline Insights and Clinical Trials
4.10 Disc Medicine Inc Thalassemia Pipeline Insights and Clinical Trials
4.11 EdiGene Inc Thalassemia Pipeline Insights and Clinical Trials
4.12 Editas Medicine Inc Thalassemia Pipeline Insights and Clinical Trials
4.13 EmeraMed Ltd Thalassemia Pipeline Insights and Clinical Trials
4.14 Errant Gene Therapeutics LLC Thalassemia Pipeline Insights and Clinical Trials
4.15 ExCellThera Inc Thalassemia Pipeline Insights and Clinical Trials
4.16 Fulcrum Therapeutics Inc Thalassemia Pipeline Insights and Clinical Trials
4.17 Gamida Cell Ltd Thalassemia Pipeline Insights and Clinical Trials
4.18 Gilead Sciences Inc Thalassemia Pipeline Insights and Clinical Trials
4.19 Imara Inc Thalassemia Pipeline Insights and Clinical Trials
4.20 Invenux LLC Thalassemia Pipeline Insights and Clinical Trials
4.21 Ionis Pharmaceuticals Inc Thalassemia Pipeline Insights and Clinical Trials
4.22 Kymab Group Ltd Thalassemia Pipeline Insights and Clinical Trials
4.23 Merganser Biotech Inc Thalassemia Pipeline Insights and Clinical Trials
4.24 Nanomedic Inc Thalassemia Pipeline Insights and Clinical Trials
4.25 Orchard Therapeutics Ltd Thalassemia Pipeline Insights and Clinical Trials
4.26 Phoenicia Biosciences Inc Thalassemia Pipeline Insights and Clinical Trials
4.27 Protagonist Therapeutics Inc Thalassemia Pipeline Insights and Clinical Trials
4.28 Rare Partners Srl Thalassemia Pipeline Insights and Clinical Trials
4.29 Regenacy Pharmaceuticals LLC Thalassemia Pipeline Insights and Clinical Trials
4.30 Sangamo Therapeutics Inc Thalassemia Pipeline Insights and Clinical Trials
4.31 Sanquin Plasma Products BV Thalassemia Pipeline Insights and Clinical Trials
4.32 Shanghai Bioray Laboratory Inc Thalassemia Pipeline Insights and Clinical Trials
4.33 Silence Therapeutics Plc Thalassemia Pipeline Insights and Clinical Trials
4.34 Syros Pharmaceuticals Inc Thalassemia Pipeline Insights and Clinical Trials
4.35 Trucode Gene Repair Inc Thalassemia Pipeline Insights and Clinical Trials
4.36 Vertex Pharmaceuticals Inc Thalassemia Pipeline Insights and Clinical Trials
4.37 Vifor Pharma AG Thalassemia Pipeline Insights and Clinical Trials

5. THALASSEMIA MARKET NEWS AND UPDATES

6. APPENDIX

6.1 Sources and Methodology
6.2 Contact Information