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The publisher'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.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher'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 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.
Table of Contents
- Introduction
- 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 the Publisher
- 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
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Afimmune Biopharma Ltd
- Agios Pharmaceuticals Inc
- Alfasigma SpA
- Alkermes Plc
- Angiocrine Bioscience Inc
- Annexin Pharmaceuticals AB
- AntiRadical Therapeutics LLC
- Ascentage Pharma Group Inc
- Asklepion Pharmaceuticals LLC
- Aviceda Therapeutics Inc
- Axcella Health Inc
- Beam Therapeutics Inc
- Bellicum Pharmaceuticals Inc
- bluebird bio Inc
- Brooklyn ImmunoTherapeutics Inc
- C4X Discovery Holdings Plc
- Cannabis Science Inc
- Cell Source Inc
- Cellectis SA
- Cetya Therapeutics Inc
- Chugai Pharmaceutical Co Ltd
- CRISPR Therapeutics AG
- CSL Ltd
- Cyclerion Therapeutics Inc
- Daiichi Sankyo Co Ltd
- Editas Medicine Inc
- Emmaus Life Sciences Inc
- Epidestiny Inc
- Epizyme Inc
- Forma Therapeutics Inc
- Fulcrum Therapeutics Inc
- Functional Fluidics LLC
- Gamida Cell Ltd
- Garuda Therapeutics Inc
- Gilead Sciences Inc
- Global Blood Therapeutics Inc
- Glycan Therapeutics Inc
- GlycoMimetics Inc
- Graphite Bio Inc
- GreenLight Biosciences Holdings PBC
- Guangzhou Dazhou Biomedical Technology Co Ltd
- Hartis Pharma SA
- Hillhurst Biopharmaceuticals Inc
- Homology Medicines Inc
- IHP Therapeutics Inc
- IllExcor Therapeutics LLC
- Imago BioSciences 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
- Mind Biosciences LLC
- Mitobridge Inc
- Modus Therapeutics AB
- Molecules For Health Inc
- Monte Rosa Therapeutics Inc
- NicOx SA
- Novartis AG
- NuvOx Pharma LLC
- Oryzon Genomics SA
- Pfizer Inc
- PHD Biosciences
- Phoenicia Biosciences Inc
- Prolong Pharmaceuticals LLC
- Rare Partners Srl
- Regenacy Pharmaceuticals LLC
- ReNeuroGen LLC
- San Rocco Therapeutics LLC
- Sana Biotechnology Inc
- Sangamo Therapeutics Inc
- Secura Bio Inc
- Shanghai Bendao Gene Technology Co Ltd
- Syros Pharmaceuticals Inc
- Tarus Therapeutics Inc
- ThioLab LLC
- TransThera Sciences (Nanjing) Inc
- Vanguard Therapeutics Inc
- Vera Therapeutics Inc
- Vertex Pharmaceuticals Inc
- Vifor Pharma Ltd
