Sickle Cell Disease (Hematology) – Drugs In Development, 2021

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Sickle Cell Disease – Drugs In Development, 2021, provides an overview of the Sickle Cell Disease (Hematological Disorders) pipeline landscape.

Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. Treatment includes antibiotics, pain relievers, blood transfusion and stem cell transplant.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Sickle Cell Disease – Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Sickle Cell Disease (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 Sickle Cell Disease (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Sickle Cell Disease 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, IND/CTA Filed, Preclinical and Discovery stages are 2, 4, 16, 16, 5, 32 and 13 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 7 and 1 molecules, respectively.

Sickle Cell Disease (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 Global Markets Direct’s 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 Sickle Cell Disease (Hematological Disorders).

– The pipeline guide reviews pipeline therapeutics for Sickle Cell Disease (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 Sickle Cell Disease (Hematological Disorders) therapeutics and enlists all their major and minor projects.

– The pipeline guide evaluates Sickle Cell Disease (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 Sickle Cell Disease (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 Sickle Cell Disease (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 Sickle Cell Disease (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.

Companies mentioned

Agios Pharmaceuticals Inc

Alfasigma SpA

Alkermes Plc

Angiocrine Bioscience Inc

Annexin Pharmaceuticals AB

Aruvant Sciences Inc

Aviceda Therapeutics

Axcella Health Inc

Beam Therapeutics Inc

Bellicum Pharmaceuticals Inc

bluebird bio Inc

Bristol Myers Squibb Co

Brooklyn ImmunoTherapeutics Inc

C4X Discovery Holdings Plc

Cannabis Science Inc

Cell Source Inc

Cetya Therapeutics Inc

CRISPR Therapeutics AG

CSL Ltd

Daiichi Sankyo Co Ltd

Editas Medicine Inc

Emmaus Life Sciences Inc

Epidestiny Inc

Epizyme Inc

ExCellThera Inc

Forma Therapeutics Inc

Fulcrum Therapeutics Inc

Functional Fluidics LLC

Gamida Cell Ltd

Gilead Sciences Inc

Global Blood Therapeutics Inc

Glycan Therapeutics Inc

Graphite Bio Inc

Hartis Pharma SA

Hillhurst Biopharmaceuticals Inc

Homology Medicines Inc

IllExcor Therapeutics LLC

Imago BioSciences Inc

Imara Inc

Intellia Therapeutics Inc

Invenux LLC

Jasper Therapeutics Inc

Jazz Pharmaceuticals Plc

KM Biologics Co Ltd

Medeor Therapeutics Inc

Merck & Co Inc

Micelle BioPharma Inc

MimeTech Srl

Modus Therapeutics AB

Molecules For Health Inc

Monte Rosa Therapeutics Inc

Nanjing Transthera Biosciences Co Ltd

Novartis AG

Oryzon Genomics SA

Pfizer Inc

PHD Biosciences

Phoenicia Biosciences Inc

Rare Partners Srl

Regenacy Pharmaceuticals LLC

ReNeuroGen LLC

San Rocco Therapeutics LLC

Sana Biotechnology Inc

Sangamo Therapeutics Inc

Secura Bio Inc

Syros Pharmaceuticals Inc

Tarus Therapeutics Inc

ThioLab LLC

Vanguard Therapeutics Inc

Vera Therapeutics Inc

Vertex Pharmaceuticals Inc

Vifor Pharma Ltd

Table of Contents

Table of Contents

Introduction

Sickle Cell Disease - Overview

Sickle Cell Disease - Therapeutics Development

Sickle Cell Disease - Therapeutics Assessment

Sickle Cell Disease - Companies Involved in Therapeutics Development

Sickle Cell Disease - Drug Profiles

Sickle Cell Disease - Dormant Projects

Sickle Cell Disease - Discontinued Products

Sickle Cell Disease - Product Development Milestones

Appendix

List of Tables

List of Tables

Number of Products under Development for Sickle Cell Disease, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Mechanism of Action, 2021 (Contd..1)

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Sickle Cell Disease – Pipeline by Agios Pharmaceuticals Inc, 2021

Sickle Cell Disease – Pipeline by Alfasigma SpA, 2021

Sickle Cell Disease – Pipeline by Alkermes Plc, 2021

Sickle Cell Disease – Pipeline by Angiocrine Bioscience Inc, 2021

Sickle Cell Disease – Pipeline by Annexin Pharmaceuticals AB, 2021

Sickle Cell Disease – Pipeline by Aruvant Sciences Inc, 2021

Sickle Cell Disease – Pipeline by Aviceda Therapeutics, 2021

Sickle Cell Disease – Pipeline by Axcella Health Inc, 2021

Sickle Cell Disease – Pipeline by Beam Therapeutics Inc, 2021

Sickle Cell Disease – Pipeline by Bellicum Pharmaceuticals Inc, 2021

Sickle Cell Disease – Pipeline by bluebird bio Inc, 2021

Sickle Cell Disease – Pipeline by Bristol Myers Squibb Co, 2021

Sickle Cell Disease – Pipeline by Brooklyn ImmunoTherapeutics Inc, 2021

Sickle Cell Disease – Pipeline by C4X Discovery Holdings Plc, 2021

Sickle Cell Disease – Pipeline by Cannabis Science Inc, 2021

Sickle Cell Disease – Pipeline by Cell Source Inc, 2021

Sickle Cell Disease – Pipeline by Cetya Therapeutics Inc, 2021

Sickle Cell Disease – Pipeline by CRISPR Therapeutics AG, 2021

Sickle Cell Disease – Pipeline by CSL Ltd, 2021

Sickle Cell Disease – Pipeline by Daiichi Sankyo Co Ltd, 2021

Sickle Cell Disease – Pipeline by Editas Medicine Inc, 2021

Sickle Cell Disease – Pipeline by Emmaus Life Sciences Inc, 2021

Sickle Cell Disease – Pipeline by Epidestiny Inc, 2021

Sickle Cell Disease – Pipeline by Epizyme Inc, 2021

Sickle Cell Disease – Pipeline by ExCellThera Inc, 2021

Sickle Cell Disease – Pipeline by Forma Therapeutics Inc, 2021

Sickle Cell Disease – Pipeline by Fulcrum Therapeutics Inc, 2021

Sickle Cell Disease – Dormant Projects, 2021

Sickle Cell Disease – Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development for Sickle Cell Disease, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Number of Products by Top 10 Targets, 2021

Number of Products by Stage and Top 10 Targets, 2021

Number of Products by Top 10 Mechanism of Actions, 2021

Number of Products by Stage and Top 10 Mechanism of Actions, 2021

Number of Products by Top 10 Routes of Administration, 2021

Number of Products by Stage and Top 10 Routes of Administration, 2021

Number of Products by Top 10 Molecule Types, 2021

Number of Products by Stage and Top 10 Molecule Types, 2021

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