Hemophilia Pipeline, FDA Approvals, Clinical Trials Developments and Companies 2023 | Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics

December 07 18:41 2023
Hemophilia Pipeline, FDA Approvals, Clinical Trials Developments and Companies 2023 | Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics

DelveInsight’s, “Hemophilia Pipeline Insight 2023” report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in Hemophilia pipeline landscape. It covers the Hemophilia pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hemophilia pipeline 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 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.
  • The leading companies working in the Hemophilia Market include Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, and others.
  • Promising Hemophilia Pipeline Therapies in the various stages of development include MG1113, SerpinPC, Emicizumab, Biostate, rFVIIIFc, BeneFIX, ADYNOVI, rFIXFc, Emicizumab, and others.
  • December 2023: Suzhou Alphamab Co. Ltd. announced a study of Phase 2 clinical trials for KN057 Dose I. This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous doses of KN057 in subjects with hemophilia A or B, with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). 24 adult participants 18 to 70 years of age with moderately severe to severe hemophilia A or hemophilia B (defined as FVIII or FIX activity ≤2%, respectively) with or without inhibitors (including 18 HA/HB patients without inhibitors and 6 HA/HB patients with inhibitors) are expected to be enrolled in this study during which they will receive prophylaxis treatment (defined as treatment by SC injection once weekly of KN057).
  • December 2023: Pfizer announced a study of Phase 3 clinical trials for Marstacimab. The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients. This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.

 

Request a sample and discover the recent advances in Hemophilia Treatment Drugs @ Hemophilia Pipeline Outlook Report

 

In the Hemophilia pipeline report, a detailed description of the drug is given which includes the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hemophilia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Hemophilia Overview

Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as 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 factor in the blood.

 

Find out more about Hemophilia Treatment Landscape @ Drugs for Hemophilia Treatment

 

Hemophilia Emerging Drugs Profile

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

 

Hemophilia Pipeline Therapeutics Assessment

There are approx. 80+ key companies which are developing the therapies for Hemophilia. The Hemophilia companies which have their Hemophilia drug candidates in the most advanced stage, i.e. Phase III include, Alnylam Pharmaceuticals.

 

DelveInsight’s Hemophilia Pipeline 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

  • Intra-articular
  • Intraocular
  • Intrathecal
  • Intravenous
  • Ophthalmic
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical
  • Transdermal

 

Hemophilia Products have been categorized under various Molecule types such as

  • Oligonucleotide
  • Peptide
  • Small molecule

 

Learn more about the emerging Hemophilia Pipeline Therapies @ Hemophilia Clinical Trials Assessment

 

Scope of the Hemophilia Pipeline Report

  • Coverage- Global
  • Hemophilia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
  • Hemophilia Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
  • Hemophilia Companies- Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, and others.
  • Hemophilia Pipeline Therapies- MG1113, SerpinPC, Emicizumab, Biostate, rFVIIIFc, BeneFIX, ADYNOVI, rFIXFc, Emicizumab, and others.

 

Dive deep into rich insights for new drugs for Hemophilia Treatment, Visit @ Hemophilia Market Drivers and Barriers, and Future Perspective

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Hemophilia: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Hemophilia– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. Fitusiran: Alnylam Pharmaceuticals
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. SerpinPC: Centessa Pharmaceuticals
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I)
  14. Drug name: Company Name
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. Drug name: Company Name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Hemophilia Key Companies
  21. Hemophilia Key Products
  22. Hemophilia- Unmet Needs
  23. Hemophilia- Market Drivers and Barriers
  24. Hemophilia- Future Perspectives and Conclusion
  25. Hemophilia Analyst Views
  26. Hemophilia Key Companies
  27. Appendix

 

For further information on the Hemophilia Pipeline Therapeutics, reach out @ Hemophilia Unmet Needs and Analyst Views

 

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