Hemophilia Clinical and Non-Clinical Studies, Key Companies, Therapeutic Assessment, Emerging Therapies, Treatment Algorithm, and Pipeline Analysis | Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therape

Hemophilia Pipeline constitutes 80+ key companies continuously working towards developing 80+ Hemophilia treatment therapies, analyzes DelveInsight.

Hemophilia Overview:

Hemophilia is a genetic bleeding disorder where the blood doesn't clot properly due to low levels of clotting factors, either factor VIII (Hemophilia A) or factor IX (Hemophilia B). This can result in spontaneous bleeding or excessive bleeding after injuries or surgery. The severity depends on how low the clotting factor is, with lower levels leading to more frequent bleeding episodes, which can cause serious health issues.

While hemophilia is usually inherited, in rare cases, it can develop later in life, particularly in middle-aged or elderly individuals, or in women during or after pregnancy. The condition occurs due to mutations in genes on the X chromosome, which are responsible for producing the clotting factors. Males are more commonly affected because they have only one X chromosome, while females would need both X chromosomes to be defective for the disease to manifest, making it rarer in women.

The two most common types of hemophilia are Hemophilia A (lack of factor VIII) and Hemophilia B (lack of factor IX). Symptoms include joint bleeding, bruising, bleeding gums, and difficulty stopping bleeding after minor injuries. Treatment involves replacing the missing clotting factor through infusions, which can be done at home to manage or prevent bleeding episodes.

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"Hemophilia Pipeline Insight 2024" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Hemophilia Therapeutics Market.

Key Takeaways from the Hemophilia Pipeline Report

DelveInsight's Hemophilia pipeline report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia treatment.
In December 2024, Be Biopharma launched its first-in-human Phase 1/2 clinical trial for BE-101, a B-cell treatment candidate for hemophilia B. The trial, called BeCoMe-9 (NCT06611436), is recruiting patients at two U.S. sites. BE-101 is a groundbreaking therapy aimed at addressing the unmet needs of hemophilia B patients, particularly those with frequent bleeding episodes. The two-part trial will assess the safety and clinical effectiveness of a single intravenous infusion of BE-101 in adults with moderately severe to severe hemophilia B.
In April 2024, Pfizer Inc. announced that the U.S. FDA approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for treating adults with moderate to severe hemophilia B. This approval is for patients on factor IX (FIX) prophylaxis therapy who have experienced life-threatening bleeding or recurrent serious spontaneous bleeding episodes and do not have neutralizing antibodies to AAVRh74var capsid. BEQVEZ is a one-time treatment that enables patients to produce FIX on their own, eliminating the need for regular intravenous FIX infusions.
In February 2024, Intellia Therapeutics and Regeneron announced plans to begin the first human trial of a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for hemophilia B by mid-2024. This comes after the FDA approved the investigational new drug (IND) application filed by the companies.
Key Hemophilia companies such as Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, and others are evaluating new drugs for Hemophilia to improve the treatment landscape.
Promising Hemophilia pipeline therapies in various stages of development include SerpinPC, Fitusiran, ASC 618, and others.

Hemophilia Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Hemophilia Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Hemophilia market.

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Hemophilia Emerging Drugs

SerpinPC: Centessa Pharmaceuticals
Fitusiran: Alnylam Pharmaceuticals
ASC 618: ASC Therapeutics

Hemophilia Companies

Over 80 major companies are working on developing therapies for Hemophilia. Among them, Alnylam Pharmaceuticals has drug candidates for Hemophilia in the most advanced stage, Phase III.

DelveInsight's report covers around 80+ products under different phases of clinical development like

Late stage products (Phase III)
Mid-stage products (Phase II)
Early-stage product (Phase I) along with the details of
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates

Hemophilia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

Intravenous
Subcutaneous
Oral
Intramuscular

Hemophilia Products have been categorized under various Molecule types such as

Monoclonal antibody
Small molecule
Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging Hemophilia Therapies and Key Companies: Hemophilia Clinical Trials and advancements @ https://www.delveinsight.com/report-store/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Hemophilia Pipeline Therapeutic Assessment
• Hemophilia Assessment by Product Type
• Hemophilia By Stage
• Hemophilia Assessment by Route of Administration
• Hemophilia Assessment by Molecule Type

Download Hemophilia Sample report to know in detail about the Hemophilia treatment market @ Hemophilia Therapeutic Assessment @ https://www.delveinsight.com/sample-request/hemophilia-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Content

1. Report Introduction
2. Executive Summary
3. Hemophilia Current Treatment Patterns
4. Hemophilia - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Hemophilia Late-Stage Products (Phase-III)
7. Hemophilia Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Hemophilia Discontinued Products
13. Hemophilia Product Profiles
14. Hemophilia Key Companies
15. Hemophilia Key Products
16. Dormant and Discontinued Products
17. Hemophilia Unmet Needs
18. Hemophilia Future Perspectives
19. Hemophilia Analyst Review
20. Appendix
21. Report Methodology

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Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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