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

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Published: April 29, 2022 Report Code: GMDGDHC22124IDB-ST

Report Overview
Key Players
Table of Contents
Table
Figures
FAQs

Thalassemia Pipeline Drugs Market Overview

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. Treatment includes blood transfusions and bone marrow transplants.

The Thalassemia Drugs in Development market research report provides an overview of the Thalassemia pipeline landscape. The report provides comprehensive information on the therapeutics under development for Thalassemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Thalassemia and features dormant and discontinued projects.

Thalassemia Pipeline Drugs Market Targets

Some of the targets of the Thalassemia pipeline drugs market are Hemoglobin Subunit Beta, Transmembrane Protease Serine 6, Growth/Differentiation Factor 11, Hemoglobin Subunit Gamma 1, Iron, Growth/Differentiation Factor 8, Pyruvate Kinase PKLR, Aromatic L Amino Acid Decarboxylase, B Cell Lymphoma/Leukemia 11A, and Solute Carrier Family 40 Member 1.

Thalassemia pipeline drugs market, by targets

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Mechanisms of Action of Thalassemia Pipeline Drugs Market

Some of the mechanisms of action of the Thalassemia pipeline drugs market are Hemoglobin Subunit Beta Activator, Transmembrane Protease Serine 6 Inhibitor, Growth/Differentiation Factor 11 Inhibitor, Hemoglobin Subunit Gamma 1 Activator, Iron Chelator, Growth/Differentiation Factor 8 Inhibitor, Pyruvate Kinase PKLR Activator, Aromatic L Amino Acid Decarboxylase Inhibitor, Solute Carrier Family 40 Member 1 Inhibitor, and B Cell Lymphoma/Leukemia 11A Inhibitor.

Thalassemia pipeline drugs market, by mechanisms of action

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Routes of Administration in Thalassemia Pipeline Drugs Market

The routes of administration in the Thalassemia pipeline drugs market are oral, intravenous, subcutaneous, parenteral, intramedullary, and topical.

Thalassemia pipeline drugs market, by routes of administration

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Molecule Types in Thalassemia Pipeline Drugs Market

The molecule types in the Thalassemia pipeline drugs market are small molecule, gene-modified cell therapy, gene therapy, cell therapy, Fusion Protein, monoclonal antibody, antisense oligonucleotide, oligonucleotide, antisense RNAi oligonucleotide, recombinant protein, and synthetic peptide.

Thalassemia pipeline drugs market, by molecule types

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Key Thalassemia Pipeline Drugs Market Companies

Some of the key companies in the Thalassemia pipeline drugs market are Acceleron Pharma Inc, Shanghai Bioray Laboratory Inc, Agios Pharmaceuticals Inc, Ionis Pharmaceuticals Inc, Phoenicia Biosciences Inc, Vertex Pharmaceuticals Inc, Abfero Pharmaceuticals Inc, Aruvant Sciences Inc, ASC Therapeutics Inc, and Ascentage Pharma Group Inc.

Thalassemia pipeline drugs market, by key companies

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Market report overview

Targets	Hemoglobin Subunit Beta, Transmembrane Protease Serine 6, Growth/Differentiation Factor 11, Hemoglobin Subunit Gamma 1, Iron, Growth/Differentiation Factor 8, Pyruvate Kinase PKLR, Aromatic L Amino Acid Decarboxylase, B Cell Lymphoma/Leukemia 11A, and Solute Carrier Family 40 Member 1
Mechanisms of Action	Hemoglobin Subunit Beta Activator, Transmembrane Protease Serine 6 Inhibitor, Growth/Differentiation Factor 11 Inhibitor, Hemoglobin Subunit Gamma 1 Activator, Iron Chelator, Growth/Differentiation Factor 8 Inhibitor, Pyruvate Kinase PKLR Activator, Aromatic L Amino Acid Decarboxylase Inhibitor, Solute Carrier Family 40 Member 1 Inhibitor, and B Cell Lymphoma/Leukemia 11A Inhibitor
Routes of Administration	Oral, Intravenous, Subcutaneous, Parenteral, Intramedullary, and Topical
Molecule Types	Small Molecule, Gene-Modified Cell Therapy, Gene Therapy, Cell Therapy, Fusion Protein, Monoclonal Antibody, Antisense Oligonucleotide, Oligonucleotide, Antisense RNAi Oligonucleotide, Recombinant Protein, and Synthetic Peptide
Key Companies	Acceleron Pharma Inc, Shanghai Bioray Laboratory Inc, Agios Pharmaceuticals Inc, Ionis Pharmaceuticals Inc, Phoenicia Biosciences Inc, Vertex Pharmaceuticals Inc, Abfero Pharmaceuticals Inc, Aruvant Sciences Inc, ASC Therapeutics Inc, and Ascentage Pharma Group Inc

Scope

This report provides:

A snapshot of the global therapeutic landscape of Thalassemia (Hematological Disorders).
Reviews of pipeline therapeutics for Thalassemia (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
Pipeline products based on several stages of development ranging from pre-registration to discovery and undisclosed stages.
Descriptive drug profiles for the pipeline products which include product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities.
Reviews of key companies involved in Thalassemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
Evaluation of Thalassemia (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
All the dormant and discontinued pipeline projects.
Reviews of the latest news and deals 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.

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

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Thalassemia – 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 Development

Thalassemia – Drug Profiles

Thalassemia – Dormant Projects

Thalassemia – Discontinued Products

Thalassemia – Product Development Milestones

Table

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 Companies, 2022 (Contd..1)

Number of Products under Development by Companies, 2022 (Contd..2)

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Companies, 2022 (Contd..1)

Products under Development by Companies, 2022 (Contd..2)

Products under Development by Companies, 2022 (Contd..3)

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 – Dormant Projects, 2022 (Contd..1)

Thalassemia – Discontinued Products, 2022

Figures

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

Frequently asked questions

What are the targets of the Thalassemia pipeline drugs market?

Some of the targets of the Thalassemia pipeline drugs market are Hemoglobin Subunit Beta, Transmembrane Protease Serine 6, Growth/Differentiation Factor 11, Hemoglobin Subunit Gamma 1, Iron, Growth/Differentiation Factor 8, Pyruvate Kinase PKLR, Aromatic L Amino Acid Decarboxylase, B Cell Lymphoma/Leukemia 11A, and Solute Carrier Family 40 Member 1.
What are the mechanisms of action of the Thalassemia pipeline drugs market?

Some of the mechanisms of action of the Thalassemia pipeline drugs market are Hemoglobin Subunit Beta Activator, Transmembrane Protease Serine 6 Inhibitor, Growth/Differentiation Factor 11 Inhibitor, Hemoglobin Subunit Gamma 1 Activator, Iron Chelator, Growth/Differentiation Factor 8 Inhibitor, Pyruvate Kinase PKLR Activator, Aromatic L Amino Acid Decarboxylase Inhibitor, Solute Carrier Family 40 Member 1 Inhibitor, and B Cell Lymphoma/Leukemia 11A Inhibitor.
What are the routes of administration in the Thalassemia pipeline drugs market?

The routes of administration in the Thalassemia pipeline drugs market are oral, intravenous, subcutaneous, parenteral, intramedullary, and topical.
What are the molecule types in the Thalassemia pipeline drugs market?

The molecule types in the Thalassemia pipeline drugs market are small molecule, gene-modified cell therapy, gene therapy, cell therapy, Fusion Protein, monoclonal antibody, antisense oligonucleotide, oligonucleotide, antisense RNAi oligonucleotide, recombinant protein, and synthetic peptide.
Which are the key companies in the Thalassemia pipeline drugs market?

Some of the key companies in the Thalassemia pipeline drugs market are Acceleron Pharma Inc, Shanghai Bioray Laboratory Inc, Agios Pharmaceuticals Inc, Ionis Pharmaceuticals Inc, Phoenicia Biosciences Inc, Vertex Pharmaceuticals Inc, Abfero Pharmaceuticals Inc, Aruvant Sciences Inc, ASC Therapeutics Inc, and Ascentage Pharma Group Inc.

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

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

Thalassemia Pipeline Drugs Market Overview

Thalassemia Pipeline Drugs Market Targets

Mechanisms of Action of Thalassemia Pipeline Drugs Market

Routes of Administration in Thalassemia Pipeline Drugs Market

Molecule Types in Thalassemia Pipeline Drugs Market

Key Thalassemia Pipeline Drugs Market Companies

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