Hereditary Angioedema - Drug Pipeline Landscape, 2023
Description
Hereditary Angioedema - Drug Pipeline Landscape, 2023
Hereditary Angioedema is an infrequent inherited disease that causes substantial swelling in various body tissues, such as the abdomen or face caused by a low level or improper function of a protein called the C1 inhibitor. There are three types of hereditary angioedema namely type I, II, and III.
Hereditary Angioedema is mainly caused by a low level or improper function of a protein called C1 inhibitor.
Symptoms are severe abdominal pain and cramping, dehydration, diarrhea and shock, hoarse voice, difficulty swallowing, and difficulty breathing.
Hereditary Angioedema is diagnosed by blood tests that detect decreased levels of complement proteins and erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) levels.
Hereditary Angioedema is treated by medications, such as corticosteroids, antihistamines and epinephrine.
Major pharmaceutical companies are involved in the development of potential drug candidates to improve the treatment of Hereditary Angioedema treatment such as CSL312, Sebetralstat, BMN 331, PHVS719, and others. Key players involved in the development of therapies to treat Hereditary Angioedema are CSL Behring, Kalvista Pharmaceuticals and others. Four drugs are under late-stage Phase III clinical trials and three drugs are in Phase II clinical trials and some other drugs are under Phase I, preclinical and discovery stages of development.
In Nov 2022, Ionis Pharmaceuticals Inc presented positive Phase II study of open label extension results of donidalosen. In Oct 2022, KalVista Pharmaceuticals announced positive Phase I data of sebetralstat. In August 2022, Global Biotechnology announed positive top-line Phase III results for garadacimab.
Report Highlights
Global Insight Service's, Hereditary Angioedema - Drug Pipeline Landscape, 2022 report provides an overview of the Hereditary Angioedema pipeline drugs. This report covers detailed insights on Hereditary Angioedema drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Hereditary Angioedema pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.
Methodology
The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.
Scope
The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.
Reasons to Buy
Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.
Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.
Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.
Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.
Please Note: This report will be delivered by publisher within 3-4 business days of order confirmation.
Hereditary Angioedema is an infrequent inherited disease that causes substantial swelling in various body tissues, such as the abdomen or face caused by a low level or improper function of a protein called the C1 inhibitor. There are three types of hereditary angioedema namely type I, II, and III.
Hereditary Angioedema is mainly caused by a low level or improper function of a protein called C1 inhibitor.
Symptoms are severe abdominal pain and cramping, dehydration, diarrhea and shock, hoarse voice, difficulty swallowing, and difficulty breathing.
Hereditary Angioedema is diagnosed by blood tests that detect decreased levels of complement proteins and erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) levels.
Hereditary Angioedema is treated by medications, such as corticosteroids, antihistamines and epinephrine.
Major pharmaceutical companies are involved in the development of potential drug candidates to improve the treatment of Hereditary Angioedema treatment such as CSL312, Sebetralstat, BMN 331, PHVS719, and others. Key players involved in the development of therapies to treat Hereditary Angioedema are CSL Behring, Kalvista Pharmaceuticals and others. Four drugs are under late-stage Phase III clinical trials and three drugs are in Phase II clinical trials and some other drugs are under Phase I, preclinical and discovery stages of development.
In Nov 2022, Ionis Pharmaceuticals Inc presented positive Phase II study of open label extension results of donidalosen. In Oct 2022, KalVista Pharmaceuticals announced positive Phase I data of sebetralstat. In August 2022, Global Biotechnology announed positive top-line Phase III results for garadacimab.
Report Highlights
Global Insight Service's, Hereditary Angioedema - Drug Pipeline Landscape, 2022 report provides an overview of the Hereditary Angioedema pipeline drugs. This report covers detailed insights on Hereditary Angioedema drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Hereditary Angioedema pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.
Methodology
The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.
Scope
The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clinical trials, and key upcoming milestones are included.
Reasons to Buy
Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.
Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.
Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.
Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.
Please Note: This report will be delivered by publisher within 3-4 business days of order confirmation.
Table of Contents
91 Pages
- 1. Introduction
- 1.1 Hereditary Angioedema - Pipeline Drugs, 2023-Coverage
- 2. Disease Overview - Hereditary Angioedema
- 2.1 Causes
- 2.2 Signs and Symptoms
- 2.3 Diagnosis
- 2.4 Disease Management
- 3. Hereditary Angioedema - Pipeline Drugs Development - Overview
- 3.1 Comparative Analysis by Stage of Development
- 3.2 Number of Products under Development by Companies, 2023
- 3.3 Products under Development by Companies, 2023
- 4. Assessment of Therapeutics
- 4.1 Assessment by Target
- 4.2 Assessment by Mechanism of Action
- 4.3 Assessment by Molecule Type
- 4.4 Assessment by Route of Administration
- 5. Drug Profiles
- 5.1 Clinical Stage Drugs - Phase III
- 5.1.1 CSL312
- 5.1.2 Donidalorsen
- 5.1.3 PHVS416
- 5.1.4 Sebetralstat
- 5.2 Clinical Stage Drugs - Phase II
- 5.2.1 BMN 331
- 5.2.2 NTLA-2002
- 5.2.3 OCTA-C1-INH
- 5.3 Clinical Stage Drugs - Phase I
- 5.3.1 ATN-249
- 5.3.2 CU105
- 5.3.3 PHVS719
- 5.3.4 STAR-0215
- 5.4 Clinical Stage Drugs - Preclinical
- 5.4.1 FACTOR XII
- 5.4.2 OTL-105
- 5.4.3 RNA Actuators
- 5.4.4 VE 4062
- 5.4.5 VE-4666
- 5.5 Clinical Stage Drugs - Discovery
- 5.5.1 ATN-XXX
- 5.5.2 Gene therapy
- 6. Key Regulatory Designations
- 7. Key Deals
- 8. Key Upcoming Milestones
- 9. Key Companies Involved
- 9.1 Astria Therapeutics Inc
- 9.2 Attune Pharmaceuticals
- 9.3 BioMarin Pharmaceutical
- 9.4 Bridge Medicines LLC
- 9.5 CAMP4 Therapeutics Corp
- 9.6 CSL Behring
- 9.7 Curacle Co Ltd
- 9.8 Intellia Therapeutics
- 9.9 Ionis Pharmaceuticals Inc
- 9.10 Kalvista Pharmaceuticals Ltd
- 9.11 Octapharma AG
- 9.12 Pharming Technologies B.V
- 9.13 Pharvaris Netherlands B.V
- 9.14 Spark Therapeutics Inc
- 9.15 Verseon Corp
- 10. Dormant Drugs
- 10.1 Inactive Drugs
- 10.2 Discontinued Drugs
- 11. Appendix
- List of Tables
- Table 1. 2 Number of Products under Development by Companies
- Table 1. 3 Products under Development by Companies
- Table 1. 4 Products by Targets
- Table 1. 5 Products by Mechanism of Action
- Table 1. 6 Products by Molecule Type
- Table 1. 7 Products by Route of Administration
- Table 2. 1 Clinical Trial Details - Sebetralstat/KalVista Pharmaceuticals Ltd
- Table 2. 2 Clinical Trial Details - ATN-249/Attune Pharmaceuticals
- Table 2. 3 Clinical Trial Details - NTLA-2002/Intellia Therapeutics
- Table 2. 4 Clinical Trial Details - Donidalorsen/Ionis Pharmaceuticals Inc
- Table 2. 5 Clinical Trial Details - CSL312/CSL Behring
- Table 2. 6 Clinical Trial Details - BMN 331/BioMarin Pharmaceutical
- Table 2. 7 Clinical Trial Details - PHVS416/Pharvaris Netherlands B.V
- Table 2. 8 Clinical Trial Details - STAR-0215/Astria Therapeutics Inc
- Table 2. 9 Clinical Trial Details - OCTA-C1-INH/Octapharma AG
- Table 3. 1 Regulatory Designations
- Table 4. 1 Inactive Drugs
- Table 4.2 Discontinued Drugs
- List of Figures
- Figure 1. 1 Number of Products under Development for Hereditary Angioedema, 2023
- Figure 1. 2 Products by Top 5 Targets and Stage of Development for Hereditary Angioedema, 2023
- Figure 1. 3 Products by Top 5 Mechanism of Action for Hereditary Angioedema, 2023
- Figure 1. 4 Products by Top 5 Molecule Type for Hereditary Angioedema, 2023
- Figure 1. 5 Products by Top 5 Route of Administration for Hereditary Angioedema, 2023
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