Hemophilia pipeline constitutes 75+ key companies continuously working towards developing 80+ Hemophilia treatment therapies, analyzes DelveInsight
LAS VEGAS, Oct. 19, 2022 /PRNewswire/ -- DelveInsight's 'Hemophilia Pipeline Insight – 2022' report provides comprehensive global coverage of available, marketed, and pipeline hemophilia therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the hemophilia pipeline domain.
Key Takeaways from the Hemophilia Pipeline Report
- DelveInsight's hemophilia pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for hemophilia treatment.
- Leading hemophilia companies such as 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 are evaluating novel hemophilia treatment drugs candidate to improve the treatment landscape.
- Key hemophilia pipeline therapies in various stages of development include Hemophilia B Research Project, Hemophilia A Research Project, AMA006, Hemophilia ET3 Research Project, Hemophilia ET8 Research Project, Hemophilia ET9 Research Project, MG1113A, Hemophilia EVADER Research Project, PF-06741086, Giroctocogene fitelparvovec, PF06838435, UB-854, GENV-HEM, GENV-001, TQG203, BAY1093884, ASC618, Marzeptacog Alfa, STSP-0601, SPK-8011, Fitusiran, Concizumab, SerpinPC, MOD-5014, FLT180a, SPK-8011, Mim 8, AskBio009, BBM-H901, PF 06838435, ASC618 and Etranacogene dezaparvovec, Efanesoctocog alfa, and others
- In September 2022, Pfizer and Sangamo Therapeutics announced that the Phase III AFFINE study evaluating giroctocogene fitelparvovec, investigational gene therapy for patients with moderately severe to severe hemophilia A, has re-opened recruitment. All trial sites are anticipated to be active by the end of 2022, and a pivotal readout is expected in the first half of 2024.
- In June 2022, Freeline Therapeutics Holdings announced that it had begun dosing the second cohort in its Phase I/II B-LIEVE dose confirmation trial of FLT180a in people with hemophilia B.
- In May 2022, uniQure announced that the FDA accepted for priority review the Biologics License Application (BLA) submitted for etranacogene dezaparvovec. Priority review of a BLA is reserved for medicines that, if approved, would be potentially significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
- In March 2022, The Food and Drug Administration (FDA) granted Fast Track designation to ASC618 for the treatment of hemophilia A.
- In January 2022, 2seventy bio announced that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A. This agreement builds upon a successful existing multi-year research collaboration between the two companies.
- In December 2021, Belief BioMed Group (BBM) announced that it had successfully dosed the first subject in the registrational gene therapy clinical trial by intravenous (IV) infusion of BBM-H901, an adeno-associated virus (AAV) vector expressing factor IX gene for treatment of adult male hemophilia B patients.
Request a sample and discover the recent advances in hemophilia treatment @Hemophilia Pipeline Report
The hemophilia pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage hemophilia products, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the hemophilia pipeline landscape.
Hemophilia Overview
Hemophilia is a genetic bleeding disorder in which the blood fails to clot properly. This can result in both spontaneous and post-injury or surgical bleeding. Blood contains numerous proteins known as clotting factors that can aid in the prevention of bleeding. Hemophilia patients have low levels of either factor VIII (8) or factor IX (9). The amount of factors in a person's blood determines the severity of their hemophilia. The less of the factor there is, the more likely bleeding will occur, which can lead to serious health problems. The hemophilia causes are mainly genetic.
The hemophilia symptoms vary depending on the clotting factor levels. If the clotting factor level is slightly low, you may only bleed after surgery or trauma. A person may bleed for no apparent reason if the deficiency is severe. Clotting factor tests are required for hemophilia diagnosis.
Find out more about hemophilia treatment @Hemophilia Gene Therapy
A snapshot of the Hemophilia Pipeline Drugs mentioned in the report:
Drugs |
Company |
Phase |
MoA |
RoA |
Etranacogene dezaparvovec |
uniQure |
Preregistration |
Gene transference |
Intravenous |
Fitusiran |
Alnylam Pharmaceuticals |
Phase III |
Antithrombin III expression inhibitors; Haemostasis stimulants; RNA interference |
Subcutaneous |
SPK-8011 |
Spark Therapeutics |
Phase III |
Factor VIII replacements; Gene transference |
Intravenous |
Mim8 |
Novo Nordisk |
Phase III |
Blood coagulation factor stimulant |
Subcutaneous |
SerpinPC |
Centessa Pharmaceuticals |
Phase II |
Activated protein C receptor modulators; Serine protease inhibitors |
Intravenous |
FLT-180a |
Freeline Therapeutics |
Phase I/II |
Factor IX replacements; Gene transference |
Intravenous |
ASC 618 |
ASC Therapeutics |
Phase I/II |
Factor VIII replacements; Gene transference |
Parenteral |
STSP 0601 |
Staidson Beijing BioPharmaceuticals |
Phase I |
Factor X stimulants |
Parenteral |
GENV-001 |
GeneVentiv |
Preclinical |
Gene transference |
Unspecified |
Learn more about the emerging hemophilia pipeline therapies @Hemophilia Clinical Trials
Hemophilia Therapeutics Assessment
The hemophilia pipeline report proffers an integral view of hemophilia emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.
Scope of the Hemophilia Pipeline Report
- Coverage: Global
- Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
- Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal
- Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule, Gene Therapy
- Therapeutics Assessment By Mechanism of Action: Activated protein C receptor modulators, Serine protease inhibitors, Antithrombin III expression inhibitors, Haemostasis stimulants, RNA interference, Factor VIII replacements, Gene transference, Factor IX replacements, Factor X stimulants
- Key Hemophilia Companies: Intellia tx, Amarna therapeutics, Expressi Ontherapeutics, 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
- Key Hemophilia Pipeline Therapies: Hemophilia B Research Project, Hemophilia A Research Project, AMA006, Hemophilia ET3 Research Project, Hemophilia ET8 Research Project, Hemophilia ET9 Research Project, MG1113A, Hemophilia EVADER Research Project, PF-06741086, PF07055480, PF06838435, UB-854, GENV-HEM, GENV-001, TQG203, BAY1093884, ASC618, Marzeptacog Alfa, STSP-0601, SPK-8011, Fitusiran, Concizumab, SerpinPC, MOD-5014, FLT180a, SPK-8011, Mim 8, AskBio009, BBM-H901, PF 06838435, ASC618 and Etranacogene dezaparvovec, Efanesoctocog alfa, and others
Dive deep into rich insights for drugs for hemophilia, visit @Hemophilia Medications
Table of Contents
1. |
Hemophilia Pipeline Report Introduction |
2. |
Hemophilia Pipeline Report Executive Summary |
3. |
Hemophilia Pipeline: Overview |
4. |
Analytical Perspective In-depth Commercial Assessment |
5. |
Hemophilia Pipeline Therapeutics |
6. |
Hemophilia Pipeline: Late Stage Products (Pre-registration) |
6.1 |
Etranacogene dezaparvovec: uniQure |
7. |
Hemophilia Pipeline: Late Stage Products (Phase III) |
7.1 |
Mim8: Novo Nordisk |
8. |
Hemophilia Pipeline: Mid Stage Products (Phase II) |
8.1 |
SerpinPC: Centessa Pharmaceuticals |
9. |
Hemophilia Pipeline: Early Stage Products (Phase I) |
9.1 |
STSP 0601: Staidson Beijing BioPharmaceuticals |
10. |
Hemophilia Pipeline Therapeutic Assessment |
11. |
Inactive Products in the Hemophilia Pipeline |
12. |
Company-University Collaborations (Licensing/Partnering) Analysis |
13. |
Key Companies |
14. |
Key Products in the Hemophilia Pipeline |
15. |
Unmet Needs |
16. |
Market Drivers and Barriers |
17. |
Future Perspectives and Conclusion |
18. |
Analyst Views |
19. |
Appendix |
For further information on the hemophilia pipeline therapeutics, reach out @Hemophilia Drugs
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