Hemophilia Pipeline Report, 2023 Updates: In-depth Analysis into the Clinical Trials, Latest FDA, EMA, and PMDA Approvals, Emerging Drugs, and Growth Prospects | Poseida Therapeutics, Biopharma Crp.

The Hemophilia Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

“Hemophilia Pipeline Insight, 2023“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Hemophilia Market.

Some of the key takeaways from the Hemophilia Pipeline Report:

• Companies across the globe are diligently working toward developing novel Hemophilia treatment therapies with a considerable amount of success over the years. 
• Hemophilia companies working in the treatment market are Poseida Therapeutics, Biopharma Corp, ASC Therapeutics, Bayer, Centessa Pharmaceuticals plc, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, Pfizer, Nordisk A/S, uniQure, Alnylam Pharmaceuticals, Spark Therapeutics, Freeline Therapeutics, GeneVentiv, and others, are developing therapies for the Hemophilia treatment 

• Emerging Hemophilia therapies in the different phases of clinical trials are- P-FVIII-101, MG1113 GC, ASC618, BAY2599023, SerpinPC, STSP-0601, SPK-8011, PF-07055480, Mim8Novo, Etranacogene dezaparvovec, Fitusiran, SPK-8011, FLT-180a, GENV-001, and others are expected to have a significant impact on the Hemophilia market in the coming years.   
• In September 2022, Giroctocogene fitelparvovec is an investigational gene therapy for people with moderately to severely severe haemophilia A, according to a recent announcement from Pfizer and Sangamo Therapeutics. A crucial readout is predicted for the first half of 2024, and all trial sites are scheduled to be operational by the end of 2022.
• In June 2022, The second cohort of its Phase I/II B-LIEVE dose confirmation trial of FLT180a in patients with haemophilia B has started receiving doses, according to a release from Freeline Therapeutics Holdings.
• In May 2022, The FDA has approved the biologics licence application (BLA) filed for etranacogene dezaparvovec for priority review, according to a statement from uniQure. For drugs that, if approved, could significantly improve the safety or efficacy of the treatment, diagnosis, or prevention of serious illnesses when compared to standard applications, a BLA is given priority evaluation.
• In March 2022, ASC618 was given Fast Track designation by the Food and Drug Administration (FDA) for the treatment of haemophilia A.
• In January 2022, In order to collaborate on the research and development of an in vivo gene editing therapy for haemophilia A, 2seventy Bio has entered into an option and licence agreement with Novo Nordisk. The successful multi-year research partnership between the two companies is built upon by this arrangement.
• In December 2021, In order to assess the effectiveness and safety of NNC0365-3769 (Mim8) in Adults and Adolescents with Haemophilia A with or without Inhibitors, Novo Nordisk started a Phase III trial.

Hemophilia Overview

A uncommon bleeding illness called haemophilia causes the blood to clot improperly. This may result in issues with excessive bleeding following an operation or accident. Haemophilia can also cause unexpected internal bleeding, such as in the joints, muscles, and organs.

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Emerging Hemophilia Drugs Under Different Phases of Clinical Development Include:

• P-FVIII-101: Poseida Therapeutics
• MG1113 GC: Biopharma Corp
• ASC618: ASC Therapeutics
• BAY2599023: Bayer
• SerpinPC: Centessa Pharmaceuticals plc
• STSP-0601: Staidson Beijing BioPharmaceuticals
• SPK-8011: Spark Therapeutics
• PF-07055480: Pfizer
• Mim8Novo: Nordisk A/S
• Etranacogene dezaparvovec: uniQure
• Fitusiran: Alnylam Pharmaceuticals
• SPK-8011: Spark Therapeutics
• FLT-180a: Freeline Therapeutics
• GENV-001: GeneVentiv

Hemophilia Route of Administration

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 Molecule Type

Hemophilia Products have been categorized under various Molecule types, such as

• Oligonucleotide
• Peptide
• Small molecule

Hemophilia Pipeline Therapeutics Assessment

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

DelveInsight’s Hemophilia 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)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Further Hemophilia product details are provided in the report. Download the Hemophilia pipeline report to learn more about the emerging Hemophilia therapies

Some of the key companies in the Hemophilia Therapeutics Market include:

Key companies developing therapies for Hemophilia are – Intellia tx, Amarna therapeutics, Expression Therapeutics, GC Pharma, Chameleon Biosciences, Pfizer, UBI Pharma, GeneVentiv, Chia Tai Tianqing Pharmaceutical Group, Bayer, ASC Therapeutics, Catalyst Biosciences, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, CSL Behring,  Sanofi, Novo Nordisk, Centessa Pharmaceuticals, OPKO Health, Freeline Therapeutics, Spark Therapeutics, Novo Nordisk, Asklepios BioPharmaceutical, Belief Biomed, ASC Therapeutics, uniQure, Sanofi, Bioverativ, and others.

Hemophilia Pipeline Analysis:

The Hemophilia pipeline report provides insights into 

• The report provides detailed insights about companies that are developing therapies for the treatment of Hemophilia with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hemophilia Treatment.
• Hemophilia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Hemophilia 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.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

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Hemophilia Pipeline Market Drivers

• Growing burden on regulatory bodies towards Hemophilia treatment is resulted to increase R&D efforts, Advancements in rare disease gene therapy market coupled with increasing research and development activities will favor the growth of hemophilia treatment market are some of the important factors that are fueling the Hemophilia Market.

Hemophilia Pipeline Market Barriers 

• However, Possible market access issues for gene therapies due to their high upfront costs, lack of long-term data, and robust evidence of efficacy and safety, high cost of the treatment and other factors are creating obstacles in the Hemophilia Market growth.

Scope of Hemophilia Pipeline Drug Insight    

• Coverage: Global
• Key Hemophilia Companies: Poseida Therapeutics, Biopharma Corp, ASC Therapeutics, Bayer, Centessa Pharmaceuticals plc, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, Pfizer, Nordisk A/S, uniQure, Alnylam Pharmaceuticals, Spark Therapeutics, Freeline Therapeutics, GeneVentiv, and others
• Key Hemophilia Therapies: P-FVIII-101, MG1113 GC, ASC618, BAY2599023, SerpinPC, STSP-0601, SPK-8011, PF-07055480, Mim8Novo, Etranacogene dezaparvovec, Fitusiran, SPK-8011, FLT-180a, GENV-001, and others
• Hemophilia Therapeutic Assessment: Hemophilia current marketed and Hemophilia emerging therapies
• Hemophilia Market Dynamics: Hemophilia market drivers and Hemophilia market barriers 

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Table of Contents 

1. Hemophilia Report Introduction

2. Hemophilia Executive Summary

3. Hemophilia Overview

4. Hemophilia- Analytical Perspective In-depth Commercial Assessment

5. Hemophilia Pipeline Therapeutics

6. Hemophilia Late Stage Products (Phase II/III)

7. Hemophilia Mid Stage Products (Phase II)

8. Hemophilia Early Stage Products (Phase I)

9. Hemophilia Preclinical Stage Products

10. Hemophilia Therapeutics Assessment

11. Hemophilia Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Hemophilia Key Companies

14. Hemophilia Key Products

15. Hemophilia Unmet Needs

16 . Hemophilia Market Drivers and Barriers

17. Hemophilia Future Perspectives and Conclusion

18. Hemophilia Analyst Views

19. Appendix

20. About DelveInsight

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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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