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Thalassemia Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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    Report

  • 156 Pages
  • April 2022
  • Region: Global
  • Global Markets Direct
  • ID: 5589911
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The latest Pharmaceutical and Healthcare disease pipeline guide Thalassemia - Drugs In Development, 2022, provides an overview of the Thalassemia (Hematological Disorders) pipeline landscape.

Thalassemia is a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include fussiness, paleness, frequent infections, failure to thrive, poor appetite and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant.

Report Highlights


The Pharmaceutical and Healthcare latest pipeline guide Thalassemia - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Thalassemia (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Thalassemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Thalassemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Phase 0, IND/CTA Filed, Preclinical and Discovery stages are 2, 2, 15, 8, 1, 2, 22 and 10 respectively. Similarly, the Universities portfolio in Phase II, Phase 0, Preclinical and Discovery stages comprises 1, 1, 3 and 1 molecules, respectively.

Thalassemia (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope


  • The pipeline guide provides a snapshot of the global therapeutic landscape of Thalassemia (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Thalassemia (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Thalassemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Thalassemia (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Thalassemia (Hematological Disorders)

Reasons to Buy


  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Thalassemia (Hematological Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Thalassemia (Hematological Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

IntroductionReport CoverageThalassemia - Overview
Thalassemia - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
Thalassemia - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
Thalassemia - Companies Involved in Therapeutics DevelopmentThalassemia - Drug ProfilesThalassemia - Dormant ProjectsThalassemia - Discontinued ProductsThalassemia - Product Development Milestones
Featured News & Press Releases
  • Apr 12, 2022: San Rocco Therapeutics pushes clinical trial for a curative treatment for beta-thalassemia and sickle cell disease
  • Mar 25, 2022: Bristol Myers Squibb announces new Prescription Drug User Fee Act goal date for Reblozyl (luspatercept-aamt) supplemental biologics license application
  • Jan 25, 2022: Sickle cell disease and transfusion-dependent beta thalassemia: promising results of gene therapy treatment
  • Jan 18, 2022: bluebird provides update on FDA review timelines for Betibeglogene Autotemcel (beti-cel) for beta-thalassemia
  • Dec 20, 2021: Editas Medicine announces FDA clearance of investigational new drug (IND) application for EDIT-301 for the treatment of transfusion-dependent beta thalassemia
  • Dec 14, 2021: Imara presents preclinical data on IMR-261 at the American Society of Hematology (ASH) Annual Meeting 2021
  • Dec 13, 2021: Agios Presents mitapivat data highlighting long-term safety profile and durable improvement in hemoglobin and markers of hemolysis in non-transfusion-dependent a- and ß-thalassemia at 63rd ASH Annual Meeting and Exposition
  • Dec 12, 2021: New data from SLN124 healthy volunteer study reinforce broad therapeutic potential in hematological diseases
  • Dec 11, 2021: New data at ASH21, published in NEJM further demonstrate beti-cel as a potentially curative one-time gene therapy for ß-thalassemia patients who require regular transfusions through achievement of durable TI and normal or near-normal adult Hb levels
  • Dec 06, 2021: Fulcrum Therapeutics announces additional HBG mRNA induction from higher dose cohorts in phase 1 healthy adult volunteer trial of FTX-6058 for sickle cell disease and new preclinical mechanism data
  • Dec 03, 2021: U.S. Food and Drug Administration accepts for priority review supplemental biologics license application for Reblozyl (luspatercept-aamt) in adults with non-transfusion dependent (NTD) beta thalassemia
  • Nov 22, 2021: bluebird bio announces FDA priority review of biologics license application for beti-cel gene therapy for patients with ß-thalassemia who require regular red blood cell transfusions
  • Nov 04, 2021: Imara to present preclinical data at the American Society of Hematology (ASH) Annual Meeting 2021
  • Nov 04, 2021: EdiGene to present latest research on a novel surface marker and migration of hematopoietic stem cell (HSC) that could enhance HSC gene therapy and HSC transplantation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition
  • Nov 04, 2021: Editas Medicine to present data demonstrating progress towards transformative gene editing medicines for the treatment of hemoglobinopathies and cancer at the ASH Annual Meeting and Exposition
AppendixMethodologyCoverageSecondary ResearchPrimary ResearchExpert Panel ValidationContact the Analyst
List of Tables
  • Number of Products under Development for Thalassemia, 2022
  • Number of Products under Development by Companies, 2022
  • Number of Products under Development by Universities/Institutes, 2022
  • Products under Development by Companies, 2022
  • Products under Development by Universities/Institutes, 2022
  • Number of Products by Stage and Target, 2022
  • Number of Products by Stage and Mechanism of Action, 2022
  • Number of Products by Stage and Route of Administration, 2022
  • Number of Products by Stage and Molecule Type, 2022
  • Thalassemia - Pipeline by Abfero Pharmaceuticals Inc, 2022
  • Thalassemia - Pipeline by Acceleron Pharma Inc, 2022
  • Thalassemia - Pipeline by Agios Pharmaceuticals Inc, 2022
  • Thalassemia - Pipeline by Aruvant Sciences Inc, 2022
  • Thalassemia - Pipeline by ASC Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Ascentage Pharma Group Inc, 2022
  • Thalassemia - Pipeline by Beam Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Bellicum Pharmaceuticals Inc, 2022
  • Thalassemia - Pipeline by BGI Genomics Co Ltd, 2022
  • Thalassemia - Pipeline by Biomac Lifesciences Pvt Ltd, 2022
  • Thalassemia - Pipeline by bluebird bio Inc, 2022
  • Thalassemia - Pipeline by Cell Source Inc, 2022
  • Thalassemia - Pipeline by Cetya Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Complexa Inc, 2022
  • Thalassemia - Pipeline by CRISPR Therapeutics AG, 2022
  • Thalassemia - Pipeline by CSL Ltd, 2022
  • Thalassemia - Pipeline by Disc Medicine Inc, 2022
  • Thalassemia - Pipeline by DisperSol Technologies LLC, 2022
  • Thalassemia - Pipeline by EdiGene Inc, 2022
  • Thalassemia - Pipeline by Editas Medicine Inc, 2022
  • Thalassemia - Pipeline by Epidestiny Inc, 2022
  • Thalassemia - Pipeline by Forma Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Fulcrum Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Gamida Cell Ltd, 2022
  • Thalassemia - Pipeline by Garuda Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Gilead Sciences Inc, 2022
  • Thalassemia - Pipeline by Graphite Bio Inc, 2022
  • Thalassemia - Pipeline by Hangzhou Zede Pharmaceutical Technology Co Ltd, 2022
  • Thalassemia - Pipeline by Imago BioSciences Inc, 2022
  • Thalassemia - Pipeline by ImmuneCyte Inc, 2022
  • Thalassemia - Pipeline by Invenux LLC, 2022
  • Thalassemia - Pipeline by Ionis Pharmaceuticals Inc, 2022
  • Thalassemia - Pipeline by Jasper Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Jazz Pharmaceuticals Plc, 2022
  • Thalassemia - Pipeline by Kanglin Biotech Hangzhou Co Ltd, 2022
  • Thalassemia - Pipeline by Kymab Ltd, 2022
  • Thalassemia - Pipeline by Mabwell Shanghai Bioscience Co Ltd, 2022
  • Thalassemia - Pipeline by Merganser Biotech Inc, 2022
  • Thalassemia - Pipeline by Monte Rosa Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Nanomedic Inc, 2022
  • Thalassemia - Pipeline by Orchard Therapeutics Plc, 2022
  • Thalassemia - Pipeline by Phoenicia Biosciences Inc, 2022
  • Thalassemia - Pipeline by Rare Partners Srl, 2022
  • Thalassemia - Pipeline by Regenacy Pharmaceuticals LLC, 2022
  • Thalassemia - Pipeline by San Rocco Therapeutics LLC, 2022
  • Thalassemia - Pipeline by Sana Biotechnology Inc, 2022
  • Thalassemia - Pipeline by Sanquin Plasma Products BV, 2022
  • Thalassemia - Pipeline by Shanghai Bendao Gene Technology Co Ltd, 2022
  • Thalassemia - Pipeline by Shanghai Bioray Laboratory Inc, 2022
  • Thalassemia - Pipeline by Shanghai Pharmaceuticals Holding Co Ltd, 2022
  • Thalassemia - Pipeline by Silence Therapeutics Plc, 2022
  • Thalassemia - Pipeline by Suzhou GenAssist Therapeutics Co Ltd, 2022
  • Thalassemia - Pipeline by Syros Pharmaceuticals Inc, 2022
  • Thalassemia - Pipeline by TransThera Sciences (Nanjing) Inc, 2022
  • Thalassemia - Pipeline by Vera Therapeutics Inc, 2022
  • Thalassemia - Pipeline by Vertex Pharmaceuticals Inc, 2022
  • Thalassemia - Pipeline by Vifor Pharma Ltd, 2022
  • Thalassemia - Dormant Projects, 2022
  • Thalassemia - Discontinued Products, 2022

List of Figures
  • Number of Products under Development for Thalassemia, 2022
  • Number of Products under Development by Companies, 2022
  • Number of Products under Development by Universities/Institutes, 2022
  • Number of Products by Top 10 Targets, 2022
  • Number of Products by Stage and Top 10 Targets, 2022
  • Number of Products by Top 10 Mechanism of Actions, 2022
  • Number of Products by Stage and Top 10 Mechanism of Actions, 2022
  • Number of Products by Routes of Administration, 2022
  • Number of Products by Stage and Routes of Administration, 2022
  • Number of Products by Top 10 Molecule Types, 2022
  • Number of Products by Stage and Top 10 Molecule Types, 2022

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Abfero Pharmaceuticals Inc
  • Acceleron Pharma Inc
  • Agios Pharmaceuticals Inc
  • Aruvant Sciences Inc
  • ASC Therapeutics Inc
  • Ascentage Pharma Group Inc
  • Beam Therapeutics Inc
  • Bellicum Pharmaceuticals Inc
  • BGI Genomics Co Ltd
  • Biomac Lifesciences Pvt Ltd
  • bluebird bio Inc
  • Cell Source Inc
  • Cetya Therapeutics Inc
  • Complexa Inc
  • CRISPR Therapeutics AG
  • CSL Ltd
  • Disc Medicine Inc
  • DisperSol Technologies LLC
  • EdiGene Inc
  • Editas Medicine Inc
  • Epidestiny Inc
  • Forma Therapeutics Inc
  • Fulcrum Therapeutics Inc
  • Gamida Cell Ltd
  • Garuda Therapeutics Inc
  • Gilead Sciences Inc
  • Graphite Bio Inc
  • Hangzhou Zede Pharmaceutical Technology Co Ltd
  • Imago BioSciences Inc
  • ImmuneCyte Inc
  • Invenux LLC
  • Ionis Pharmaceuticals Inc
  • Jasper Therapeutics Inc
  • Jazz Pharmaceuticals Plc
  • Kanglin Biotech Hangzhou Co Ltd
  • Kymab Ltd
  • Mabwell Shanghai Bioscience Co Ltd
  • Merganser Biotech Inc
  • Monte Rosa Therapeutics Inc
  • Nanomedic Inc
  • Orchard Therapeutics Plc
  • Phoenicia Biosciences Inc
  • Rare Partners Srl
  • Regenacy Pharmaceuticals LLC
  • San Rocco Therapeutics LLC
  • Sana Biotechnology Inc
  • Sanquin Plasma Products BV
  • Shanghai Bendao Gene Technology Co Ltd
  • Shanghai Bioray Laboratory Inc
  • Shanghai Pharmaceuticals Holding Co Ltd
  • Silence Therapeutics Plc
  • Suzhou GenAssist Therapeutics Co Ltd
  • Syros Pharmaceuticals Inc
  • TransThera Sciences (Nanjing) Inc
  • Vera Therapeutics Inc
  • Vertex Pharmaceuticals Inc
  • Vifor Pharma Ltd