Sickle Cell Disease – Drugs In Development, 2024

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Published: June 05, 2024 Report Code: GDGMDHC23061IDB-L5

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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.

The Sickle Cell Disease drugs in development market research report provide comprehensive information on the therapeutics under development for Sickle Cell Disease, 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 and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Sickle Cell Disease and features dormant and discontinued products.

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* This is an “on-demand” report and will be delivered within 2 business days (excluding weekends and holidays) of the purchase.

* Certain sections in the report may be removed or altered based on the availability and relevance of data.

Quick View – Sickle Cell Disease
Key Targets
· Hemoglobin Subunit Beta; B Cell Lymphoma/Leukemia 11A; Hemoglobin Subunit Gamma 1; Histone Deacetylase; Hemoglobin; DNA (Cytosine 5) Methyltransferase 1; DNA Directed RNA Polymerase; P Selectin; Lysine Specific Histone Demethylase 1A; and Pyruvate Kinase PKLR
Key Mechanisms of Action
· Hemoglobin Subunit Beta Activator; B Cell Lymphoma/Leukemia 11A Inhibitor; Histone Deacetylase Inhibitor; Hemoglobin Subunit Gamma 1 Activator; Lysine Specific Histone Demethylase 1A Inhibitor; DNA (Cytosine 5) Methyltransferase 1 Inhibitor; DNA Directed RNA Polymerase Inhibitor; P Selectin Inhibitor; Pyruvate Kinase PKLR Activator; and Ribonucleoside Diphosphate Reductase Inhibitor
Key Routes of Administration
· Oral; Intravenous; Subcutaneous; Parenteral; Topical; Vaginal; Ophthalmic; Cutaneous; and Intraarticular
Key Molecule Types
· Small Molecule; Gene-Modified Cell Therapy; Gene Therapy; Monoclonal Antibody; Cell Therapy; Oligonucleotide; Protein; Polysaccharide; Recombinant Protein; and Blood Derivative
Major Companies
· Pfizer Inc; Novartis AG; Centre for Stem Cell Research; IllExcor Therapeutics LLC; Hartis Pharma SA; Hillhurst Biopharmaceuticals Inc; IMMvention Therapeutix Inc; Jasper Therapeutics Inc; Novo Nordisk AS; and NuvOx Pharma LLC

Scope

  • Therapeutics in Development: Covering 135 molecules, with 120 developed by companies and the rest by universities/institutes.

  • Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.

  • Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.

  • Development History: Access the complete research and development history.

  • Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.

Reasons to Buy

  • Holistic insights: Understand the broad spectrum of Sickle Cell Disease therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.

  • Pipeline Exploration: Explore the detailed Sickle Cell Disease pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.

  • Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Sickle Cell Disease treatments for adaptable decision-making.

  • Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.

Key Players

AB Science SA
Addmedica SAS
Afimmune Biopharma Ltd
Agios Pharmaceuticals Inc
AkiraBio Inc
Alexion Pharmaceuticals Inc
Alfasigma SpA
Alkermes Plc
Angiocrine Bioscience Inc
Annexin Pharmaceuticals AB
AntiRadical Therapeutics LLC
Ariya Bio
Ascentage Pharma Group Inc
Asklepion Pharmaceuticals LLC
Assistance Publique - Hopitaux de Paris
AstraZeneca Plc
Aviceda Therapeutics Inc
Bausch Health Companies Inc
Beam Therapeutics Inc
Bellicum Pharmaceuticals Inc
BioLineRx Ltd
bluebird bio Inc
Bristol-Myers Squibb Co
BRL Medicine Inc
Cell Source Inc
Cellectis SA
Centre for Stem Cell Research
Cetya Therapeutics Inc
CHA Biotech Co Ltd
Children's Hospital of Philadelphia
Chugai Pharmaceutical Co Ltd
CRISPR Therapeutics AG
CSL Ltd
Daiichi Sankyo Co Ltd
Duke University
Editas Medicine Inc
Emmaus Life Sciences Inc
Eterna Therapeutics Inc
F. Hoffmann-La Roche Ltd
Fera Pharmaceuticals LLC
Fulcrum Therapeutics Inc
Function Therapeutics Inc
Functional Fluidics LLC
Gamida Cell Ltd
Garuda Therapeutics Inc
Genethon SA
GluBio Therapeutics Inc
GlycoMimetics Inc
GreenLight Biosciences Holdings PBC
GSK plc
Guangzhou Dazhou Biomedical Technology Co Ltd
Hartis Pharma SA
Hillhurst Biopharmaceuticals Inc
Homology Medicines Inc
IHP Therapeutics Inc
IllExcor Therapeutics LLC
Imago BioSciences Inc
Intas Pharmaceuticals Ltd
Invenux LLC
Jasper Therapeutics Inc
Jazz Pharmaceuticals Plc
KARE Biosciences Inc
MabTics
Medeor Therapeutics Inc
Merck & Co Inc
Micelle BioPharma Inc
MimeTech Srl
Mina Therapeutics Ltd
Mind Biosciences LLC
Molecules For Health Inc
Monte Rosa Therapeutics Inc
Novartis AG
Novo Nordisk AS
NuvOx Pharma LLC
Oryzon Genomics SA
Pfizer Inc
PHD Biosciences
Phoenicia Biosciences Inc
Portage Biotech Inc
Prolong Pharmaceuticals LLC
Rare Partners Srl
Regenacy Pharmaceuticals LLC
ReNeuroGen LLC
San Rocco Therapeutics LLC
Sana Biotechnology Inc
Sanofi
Shanghai BDgene Therapeutics Co Ltd
Syros Pharmaceuticals Inc
Takeda Pharmaceutical Co Ltd
TransThera Sciences (Nanjing) Inc
U.S. National Institutes of Health
University of California Los Angeles
University of California San Francisco
University of Illinois at Chicago
University of Tennessee Health Science Center
University of Texas at Dallas
Vanguard Therapeutics Inc
Vascular Vision Pharmaceuticals Co
Vertex Pharmaceuticals Inc
Vifor Pharma Management Ltd
Weill Cornell Medical College
Zr Pharma & Gmbh

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Overview

Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Drugs Under Development by Companies

Drugs under Development by Universities/Institutes

Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Companies Involved in Therapeutic Development

Drug Profiles

Dormant Projects

Discontinued Drugs

Product Development Milestones

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Drugs under Development by Companies, 2024

Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Stage and Target, 2024

Number of Drugs by Stage and Mechanism of Action, 2024

Number of Drugs by Stage and Route of Administration, 2024

Number of Drugs by Stage and Molecule Type, 2024

Dormant Projects, 2024

Discontinued Drugs, 2024

Figures

List of Figures

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Top 10 Targets, 2024

Number of Drugs by Stage and Top 10 Targets, 2024

Number of Drugs by Top 10 Mechanism of Actions, 2024

Number of Drugs by Stage and Top 10 Mechanism of Actions, 2024

Number of Drugs by Top 10 Routes of Administration, 2024

Number of Drugs by Stage and Top 10 Routes of Administration, 2024

Number of Drugs by Top 10 Molecule Types, 2024

Number of Drugs by Stage and Top 10 Molecule Types, 2024

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