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

July 2022 | 277 pages | ID: SD8EDB82C585EN
Global Markets Direct

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

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Sickle Cell Disease - Drugs In Development, 2022, 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, 2022, 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, 7, 18, 16, 5, 45 and 19 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 2, 8 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.
Introduction
Global Markets Direct Report Coverage
Sickle Cell Disease - Overview
Sickle Cell Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Sickle Cell Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
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
Featured News & Press Releases
Jun 18, 2022: Forma Therapeutics announces presentations at upcoming Hematology Conferences
Jun 11, 2022: Vertex and CRISPR Therapeutics present new data on more patients with longer follow-Up treated with exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress
Jun 11, 2022: Vertex and CRISPR Therapeutics announce acceptance of late-breaking abstract for CTX001 at the 2022 Annual European Hematology Association (EHA) Congress
Jun 10, 2022: GBT presents positive new real-world evidence data at EHA2022 Congress further supporting clinical use of Oxbryta (voxelotor) in sickle cell disease
Jun 10, 2022: Fulcrum Therapeutics announces Proof-of-Concept for FTX-6058 in sickle cell disease based on initial data from the ongoing phase 1b trial
Jun 06, 2022: GBT’s inclacumab receives U.S. FDA orphan drug and rare pediatric disease designations for the treatment of sickle cell disease
May 12, 2022: Editas Medicine receives FDA Orphan Drug Designation for EDIT-301 for the treatment of beta thalassemia
May 12, 2022: Fulcrum Therapeutics to present initial data from phase 1b trial of FTX-6058 in adults living with sickle cell disease at the European Hematology Association (EHA) Hybrid Congress in Vienna, Austria
May 12, 2022: Agios to present clinical and translational data at the European Hematology Association Annual Congress
Apr 11, 2022: Emmaus Life Sciences announces launch of full-service telehealth solution
Apr 07, 2022: Emmaus Life Sciences presented positive real-world data on the efficacy of Endari in preventing acute complications from sickle cell disease at the 62nd Annual Scientific Meeting of the British Society for Haematology
Mar 29, 2022: Emmaus Life Sciences announces Endari to be added to the Florida Medicaid Preferred Drug List
Mar 23, 2022: Emmaus Life Sciences receives U.A.E. Marketing Authorization for Endari
Mar 04, 2022: Emmaus Life Sciences' Real World Data on Endari accepted for E-Poster at the 62nd Annual Scientific Meeting of the British Society for Haematology
Mar 02, 2022: Nicox’s Partner Fera Pharmaceuticals obtains Orphan Drug Designation from the U.S. FDA for Naproxcinod for the treatment of sickle cell disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

LIST OF TABLES

Number of Products under Development for Sickle Cell Disease, 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 Companies, 2022 (Contd..3)
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 Companies, 2022 (Contd..4)
Products under Development by Companies, 2022 (Contd..5)
Products under Development by Companies, 2022 (Contd..6)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Target, 2022 (Contd..1)
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Mechanism of Action, 2022 (Contd..1)
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Sickle Cell Disease - Pipeline by Afimmune Biopharma Ltd, 2022
Sickle Cell Disease - Pipeline by Agios Pharmaceuticals Inc, 2022
Sickle Cell Disease - Pipeline by Alfasigma SpA, 2022
Sickle Cell Disease - Pipeline by Alkermes Plc, 2022
Sickle Cell Disease - Pipeline by Angiocrine Bioscience Inc, 2022
Sickle Cell Disease - Pipeline by Annexin Pharmaceuticals AB, 2022
Sickle Cell Disease - Pipeline by AntiRadical Therapeutics LLC, 2022
Sickle Cell Disease - Pipeline by Ascentage Pharma Group Inc, 2022
Sickle Cell Disease - Pipeline by Asklepion Pharmaceuticals LLC, 2022
Sickle Cell Disease - Pipeline by Aviceda Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Axcella Health Inc, 2022
Sickle Cell Disease - Pipeline by Beam Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Bellicum Pharmaceuticals Inc, 2022
Sickle Cell Disease - Pipeline by bluebird bio Inc, 2022
Sickle Cell Disease - Pipeline by Brooklyn ImmunoTherapeutics Inc, 2022
Sickle Cell Disease - Pipeline by C4X Discovery Holdings Plc, 2022
Sickle Cell Disease - Pipeline by Cannabis Science Inc, 2022
Sickle Cell Disease - Pipeline by Cell Source Inc, 2022
Sickle Cell Disease - Pipeline by Cellectis SA, 2022
Sickle Cell Disease - Pipeline by Cetya Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Chugai Pharmaceutical Co Ltd, 2022
Sickle Cell Disease - Pipeline by CRISPR Therapeutics AG, 2022
Sickle Cell Disease - Pipeline by CSL Ltd, 2022
Sickle Cell Disease - Pipeline by Cyclerion Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Daiichi Sankyo Co Ltd, 2022
Sickle Cell Disease - Pipeline by Editas Medicine Inc, 2022
Sickle Cell Disease - Pipeline by Emmaus Life Sciences Inc, 2022
Sickle Cell Disease - Pipeline by Epidestiny Inc, 2022
Sickle Cell Disease - Pipeline by Epizyme Inc, 2022
Sickle Cell Disease - Pipeline by Forma Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Fulcrum Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Functional Fluidics LLC, 2022
Sickle Cell Disease - Pipeline by Gamida Cell Ltd, 2022
Sickle Cell Disease - Pipeline by Garuda Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Gilead Sciences Inc, 2022
Sickle Cell Disease - Pipeline by Global Blood Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Glycan Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by GlycoMimetics Inc, 2022
Sickle Cell Disease - Pipeline by Graphite Bio Inc, 2022
Sickle Cell Disease - Pipeline by GreenLight Biosciences Holdings PBC, 2022
Sickle Cell Disease - Pipeline by Guangzhou Dazhou Biomedical Technology Co Ltd, 2022
Sickle Cell Disease - Pipeline by Hartis Pharma SA, 2022
Sickle Cell Disease - Pipeline by Hillhurst Biopharmaceuticals Inc, 2022
Sickle Cell Disease - Pipeline by Homology Medicines Inc, 2022
Sickle Cell Disease - Pipeline by IHP Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by IllExcor Therapeutics LLC, 2022
Sickle Cell Disease - Pipeline by Imago BioSciences Inc, 2022
Sickle Cell Disease - Pipeline by Intellia Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Invenux LLC, 2022
Sickle Cell Disease - Pipeline by Jasper Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Jazz Pharmaceuticals Plc, 2022
Sickle Cell Disease - Pipeline by KM Biologics Co Ltd, 2022
Sickle Cell Disease - Pipeline by Medeor Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Merck & Co Inc, 2022
Sickle Cell Disease - Pipeline by Micelle BioPharma Inc, 2022
Sickle Cell Disease - Pipeline by MimeTech Srl, 2022
Sickle Cell Disease - Pipeline by Mind Biosciences LLC, 2022
Sickle Cell Disease - Pipeline by Mitobridge Inc, 2022
Sickle Cell Disease - Pipeline by Modus Therapeutics AB, 2022
Sickle Cell Disease - Pipeline by Molecules For Health Inc, 2022
Sickle Cell Disease - Pipeline by Monte Rosa Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by NicOx SA, 2022
Sickle Cell Disease - Pipeline by Novartis AG, 2022
Sickle Cell Disease - Pipeline by NuvOx Pharma LLC, 2022
Sickle Cell Disease - Pipeline by Oryzon Genomics SA, 2022
Sickle Cell Disease - Pipeline by Pfizer Inc, 2022
Sickle Cell Disease - Pipeline by PHD Biosciences, 2022
Sickle Cell Disease - Pipeline by Phoenicia Biosciences Inc, 2022
Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals LLC, 2022
Sickle Cell Disease - Pipeline by Rare Partners Srl, 2022
Sickle Cell Disease - Pipeline by Regenacy Pharmaceuticals LLC, 2022
Sickle Cell Disease - Pipeline by ReNeuroGen LLC, 2022
Sickle Cell Disease - Pipeline by San Rocco Therapeutics LLC, 2022
Sickle Cell Disease - Pipeline by Sana Biotechnology Inc, 2022
Sickle Cell Disease - Pipeline by Sangamo Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Secura Bio Inc, 2022
Sickle Cell Disease - Pipeline by Shanghai Bendao Gene Technology Co Ltd, 2022
Sickle Cell Disease - Pipeline by Syros Pharmaceuticals Inc, 2022
Sickle Cell Disease - Pipeline by Tarus Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by ThioLab LLC, 2022
Sickle Cell Disease - Pipeline by TransThera Sciences (Nanjing) Inc, 2022
Sickle Cell Disease - Pipeline by Vanguard Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Vera Therapeutics Inc, 2022
Sickle Cell Disease - Pipeline by Vertex Pharmaceuticals Inc, 2022
Sickle Cell Disease - Pipeline by Vifor Pharma Ltd, 2022
Sickle Cell Disease - Dormant Projects, 2022
Sickle Cell Disease - Dormant Projects, 2022 (Contd..1)
Sickle Cell Disease - Dormant Projects, 2022 (Contd..2)
Sickle Cell Disease - Dormant Projects, 2022 (Contd..3)
Sickle Cell Disease - Discontinued Products, 2022

LIST OF FIGURES

Number of Products under Development for Sickle Cell Disease, 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


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