(Nevada, Las Vegas) United States, DelveInsight’s, “Hemophilia- Pipeline Insight, 2023,” report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in the Hemophilia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Key Takeaways from the Hemophillia Pipeline Report
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Over 80+ companies and 80+ pipeline drugs in the Alpha1-Antitrypsin Deficiency (AATD) pipeline landscape and their anticipated acceptance in the Hemophilia market would significantly increase market revenue.
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Leading Hemophilia companies developing novel drug candidates to improve the Hemophilia treatment landscape include Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics
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Promising Hemophilia pipeline therapies in various stages of development include SerpinPC, Fitusiran, ASC 618, and many others
Hemophillia Overview
Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding and bleeding following injuries or surgery. Blood contains many proteins called clotting factors that can help to stop bleeding. People with hemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of hemophilia that a person has is determined by the amount of factors in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems. In rare cases, a person can develop hemophilia later in life. The majority of cases involve middle-aged or elderly people, or young women who have recently given birth or are in the later stages of pregnancy. This condition often resolves with appropriate treatment. Hemophilia is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot. This change or mutation can prevent the clotting protein from working properly or to be missing altogether. These genes are located on the X chromosome. Males have one X and one Y chromosome (XY) and females have two X chromosomes (XX). Males inherit the X chromosome from their mothers and the Y chromosome from their fathers. Females inherit one X chromosome from each parent. The X chromosome contains many genes that are not present on the Y chromosome. This means that males only have one copy of most of the genes on the X chromosome, whereas females have 2 copies. Thus, males can have a disease like hemophilia if they inherit an affected X chromosome that has a mutation in either the factor VIII or factor IX gene. Females can also have hemophilia, but this is much rarer. In such cases, both X chromosomes are affected or one is affected and the other is missing or inactive. In these females, bleeding symptoms may be similar to males with hemophilia.
Hemophillia Pipeline Analysis: Drug Profile
SerpinPC: Centessa Pharmaceuticals
SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.
Fitusiran: Alnylam Pharmaceuticals
Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by their collaborators at Sanofi Genzyme. Currently, the drug is in the Phase III stage of Clinical trial evaluation for the treatment of Hemophilia.
Discover more about the emerging Hemophillia drugs @ Hemophillia Treatment Drugs
Hemophillia Pipeline Therapies and Key Companies
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Mitapivat: Agios
And others
Hemophillia Pipeline Therapeutics Assessment
DelveInsight’s Report covers around 4+ products under different phases of clinical development like
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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Route of Administration
Alpha1-Antitrypsin Deficiency (AATD) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Oral
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Parenteral
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Subcutaneous
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Intramuscular
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Molecule Type
Products have been categorized under various Molecule types such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
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Product Type
Drugs have been categorized under various product types like mono, Combination, and Mono/Combination.
Scope of the Hemophillia Pipeline Report
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Coverage: Global
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Key Alpha1 Antitrypsin Deficiency Companies: Agios, Inc and many others.
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Key Hemophillia Pipeline Therapies: Mitapivat and many others
Find out more about the Hemophillia treatment options in development @ Hemophillia Clinical Trials
Table of Contents
1. Hemophillia Introduction
2. Hemophillia Executive Summary
3. Hemophillia Overview
4. Hemophillia Pipeline Therapeutics
5. Hemophilia Late-Stage Products (Phase III)
6. Hemophilia Mid-Stage Products (Phase II)
7. Hemophilia Early Stage Products (Phase I/II)
8. Hemophillia Preclinical Stage Products
9. Hemophillia Discovery Stage Products
10. Hemophillia Therapeutic Assessment
11. Hemophillia Inactive Products
12. Hemophillia Collaborations Assessment- Licensing / Partnering / Funding
13. Hemophillia Unmet Needs
14. Hemophillia Market Drivers and Barriers
15. Appendix
16. About DelveInsight
Media Contact
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