Las Vegas, USA, March 29, 2022 (GLOBE NEWSWIRE) -- Global Hemophilia A Pipeline Insights | Clinical Trials Evaluation Research Report 2022 by DelveInsight
Hemophilia A Pipeline constitutes 40+ key companies continuously working towards developing 45+ Hemophilia A treatment therapies, Analyzes DelveInsight
DelveInsight’s Hemophilia A Pipeline Insight 2022 report offers exhaustive global coverage of available, marketed, and pipeline therapies in different phases of clinical development, major pharmaceutical companies working to advance the pipeline space, and future growth potential of the Hemophilia A Pipeline domain.
Some of the essential takeaways from the Hemophilia A Pipeline report:
- DelveInsight’s Hemophilia A Pipeline analysis depicts a robust space with 40+ active players proactively working to develop 45+ pipeline treatment therapies.
- Some of the key Hemophilia A companies working to develop potential drug candidates to improve the Hemophilia A treatment scenario include Spark Therapeutics, Sigilon Therapeutics, ASC Therapeutics, Pfizer, Sanofi Genzyme, Novo Nordisk, Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd, Bayer, Ultragenix pharmaceutical, ApcinteX Ltd, Chia Tai Tianqing Pharmaceutical Group Co., Ltd, Expression Therapeutics, LLC, CSL Behring, ASC Therapeutics, Poseida Therapeutics, Staidson Beijing BioPharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, GeneVentiv Therapeutics, Jiangsu Gensciences, 2seventy bio, Generation Bio, Apitope Technology and many others.
- Essential Hemophilia A Pipeline therapies such as SPK-8011, SPK-8016, BIVV001, AGN-193408, Valoctocogene roxaparvovec, P-FVIII-101, STSP 0601, TQG203, SIG-001, Mim8, PF-07055480, Efanesoctocog alfa, WP 1301, Research program: gene therapies, GENV-HEM, Research program: gene editing therapeutics, NX-01, and others are under development in different phases of clinical studies.
- The U.S. FDA has granted Fast Track Designation (FTD) for Efanesoctocog alfa, developed by Sanofi for Hemophilia A treatment.
- In October 2021, Poseida Therapeutics and Takeda started working together to develop non-viral gene therapies for Hemophilia and other genetic diseases. Through a collaboration, Takeda Pharmaceutical will apply Poseida Therapeutics’ technologies to develop up to eight gene therapies including a Hemophilia A candidate under development by Poseida.
- In January 2022, 2seventy bio 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. Under the terms of this agreement, Novo Nordisk will have the option to license 2seventy bio’s proprietary mRNA-based megaTAL™ technology for the development of a new treatment approach for Hemophilia A patients with the goal of offering a sustained therapeutic effect.
- In March 2021, the US FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Valoctocogene roxaparvovec being developed by BioMarin. The company is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.
- In November 2021, US FDA granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for Hemophilia A and B patients, with or without clotting factor inhibitors.
- Giroctocogene fitelparvovec is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for Hemophilia A between Sangamo and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug (IND) application to Pfizer. The US FDA granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product Designation from the European Medicines Agency.
Request a sample and discover more about the scope of the report offerings @ Hemophilia A Emerging Therapies
The Hemophilia A Pipeline report lays down detailed profiles of the pipeline assets, a comparative analysis of clinical and non-clinical stage Hemophilia A products, inactive and dormant assets, comprehensive assessment of driving and restraining factors, as well as the opportunities and risks in the Hemophilia A Pipeline landscape.
Hemophilia A Overview
Hemophilia is an inherited rare bleeding disorder where blood doesn’t clot in a regular way because the person affected doesn’t make enough blood-clotting proteins (clotting factors). Without these factors, patients cannot stop bleeding when they are injured. Hemophilia symptoms depend on the level of clotting factors. If the clotting-factor level is mildly reduced, it might bleed only after surgery or trauma. If the deficiency is severe, one can bleed easily for seemingly no reason. Small cuts are not much dangerous but internal bleeding is very harmful to Hemophiliac patients. The main concern of this genetic disorder is internal bleeding in elbows, knees, ankles, and other joints. Internal bleeding can damage the organs and tissues and can also be potentially life-threatening.
There are several types of Hemophilia such as Hemophilia A, Hemophilia B, Hemophilia C, and Von Willebrand disease Hemophilia classification is done on the basis of clotting factors like Hemophilia A is caused by factor VIII, Hemophilia B is caused by factor IX. Hemophilia diagnosis includes screening tests such as Complete Blood Count (CBC), Activated Partial Thromboplastin Time (APTT) Test, Prothrombin Time (PT) Test, Fibrinogen Test, and clotting factor tests.
Find out more about the disease and recently developing strategies @ Hemophilia A Pipeline Assessment
| Drug | Company | Phase | MoA | RoA |
| P-FVIII-101 | Poseida Therapeutics | Preclinical | Factor VIII replacements; Gene transference | NA |
| Research program: gene therapies | Generation Bio | Preclinical | Gene transference | Parenteral |
| STSP 0601 | Staidson Beijing BioPharmaceuticals | Phase I/II | Factor X stimulants | Parenteral |
| TQG203 | Chia Tai Tianqing Pharmaceutical Group | Phase I | Factor VIIa stimulants | Intravenous |
| FRSW 117 | Jiangsu Gensciences | Phase II | Factor VIII replacements | Parenteral |
| OCTA101 | Octapharma | Phase I/II | Blood coagulation factor replacements; Factor X stimulants | Subcutaneous |
| SPK-8016 | Spark Therapeutics | Phase I/II | Factor VIII replacements; Gene transference | Intravenous |
| Mim8 | Novo Nordisk | Phase III | Blood coagulation factor stimulants | Subcutaneous |
| FRSW 107 | Jiangsu Gensciences | Phase III | Factor VIII replacements | Parenteral |
| Efanesoctocog alfa | Sanofi | Phase III | Blood coagulation factor replacements | Intravenous |
Learn more about the novel and emerging Hemophilia A Pipeline therapies @ Hemophilia A Pipeline Analysis
Hemophilia A Therapeutics Assessment
The Hemophilia A Pipeline report proffers an integral view of the Hemophilia A emerging novel therapies segmented by Stage, Product Type, Mechanism of Action, Route of Administration, and Molecule Type.
Scope of the Hemophilia A Pipeline Report
- Coverage: Global
- Therapeutic Assessment By Hemophilia A Product Type: Mono, Combination, Mono/Combination
- Therapeutic Assessment By Hemophilia A Clinical Trial Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III, Pre-registration, Inactive candidates
- Therapeutics Assessment By Hemophilia A Therapeutics Route of Administration: Oral, Intravenous, Inhalation, Subcutaneous
- Therapeutics Assessment By Hemophilia A Therapies Molecule Type: Gene therapy, Stem cell therapy, Small molecules
- Therapeutics Assessment By Hemophilia A Therapies Mechanism of Action: Blood coagulation factor replacements, Blood coagulation factor stimulants, Blood coagulation factor replacements, Factor X stimulants, Factor VIII replacements, Gene transference, Cell replacements, Factor X stimulants, Factor VIIa stimulants
- Key Hemophilia A Companies: Spark Therapeutics, Sigilon Therapeutics, ASC Therapeutics, Pfizer, Sanofi Genzyme, Novo Nordisk, Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd, Bayer, Ultragenix pharmaceutical, ApcinteX Ltd, Chia Tai Tianqing Pharmaceutical Group Co., Ltd, Expression Therapeutics, LLC, CSL Behring, Sigilon Therapeutics, ASC Therapeutics, Poseida Therapeutics, Staidson Beijing BioPharmaceuticals, Chia Tai Tianqing Pharmaceutical Group, GeneVentiv Therapeutics, Jiangsu Gensciences, 2seventy bio, Generation Bio, Apitope Technology and many others.
- Key Hemophilia A Pipeline Therapies: SPK-8011, SPK-8016, BIVV001, AGN-193408, OCTA101, Valoctocogene roxaparvovec, P-FVIII-101, STSP 0601, TQG203, SIG-001, Mim8, PF-07055480, Efanesoctocog alfa, WP 1301, Research program: gene therapies, GENV-HEM, Research program: gene editing therapeutics, NX-01, and others
Dive deep into rich insights for emerging drugs and Hemophilia pipeline assessment, visit @ Hemophilia A Emerging Therapies
Table of Contents
| 1 | Introduction |
| 2 | Executive Summary |
| 3 | Hemophilia A: Overview |
| 4 | Pipeline Therapeutics |
| 5 | Late Stage Products (Preregistration) |
| 5.1 | Valoctocogene roxaparvovec: BioMarin Pharmaceutical |
| 6 | Late Stage Products (Phase III) |
| 6.1 | Efanesoctocog alfa: Sanofi |
| 7 | Early Stage Products (Phase I/II) |
| 7.1 | SPK-8016: Spark Therapeutics |
| 8 | Early Stage Products (Phase I/II) |
| 8.1 | STSP 0601: Staidson Beijing BioPharmaceuticals |
| 9 | Early Stage Products (Phase I) |
| 9.1 | TQG203: Chia Tai Tianqing Pharmaceutical Group |
| 10 | Pre-clinical and Discovery Stage Products |
| 10.1 | P-FVIII-101: Poseida Therapeutics |
| 11 | Therapeutic Assessment |
| 11.1 | Inactive Products |
| 12 | Collaborations Licensing / Partnering / Funding |
| 13 | Hemophilia A Key Companies |
| 14 | Hemophilia A Key Products |
| 15 | Hemophilia A - Unmet Needs |
| 16 | Hemophilia A - Market Drivers and Barriers |
| 17 | Hemophilia A- Future Perspectives and Conclusion |
| 18 | Appendix |
| 19 | About DelveInsight |
For further information on the Hemophilia A current pipeline therapeutics, reach out @ Hemophilia A Ongoing Clinical Trials
Related Reports
DelveInsight's "Hemophilia A Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of Hemophilia A, historical and forecasted epidemiology as well as the Hemophilia A market trends, market drivers, market barriers and key companies involved such as Novo Nordisk, Shire, Sanofi, Bayer, Roche, Pfizer, Biomarin Pharmaceutical, Alnylam Pharmaceuticals, and many others.
DelveInsight's "Hemophilia B Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of Hemophilia B, historical and forecasted epidemiology, as well as the Hemophilia B market trends, market drivers, market barriers, and key companies, involved such as Novo Nordisk, Pfizer, UniQure Biopharma B.V., ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, and several others.
DelveInsight’s, “Hemophilia B Pipeline Insight, 2022,” report provides comprehensive insights about companies and pipeline drugs in the Hemophilia B pipeline landscape. It covers the pipeline drug profiles, including clinical and non-clinical stage products, and key companies involved such as Sigilon Therapeutics, Catalyst Biosciences, Centessa Pharmaceuticals, UniQure Biopharma, Sanofi Genzyme, and many more.
Acquired Hemophilia A Pipeline
DelveInsight’s, “Acquired Hemophilia A Pipeline Insight, 2022,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Acquired Hemophilia A pipeline landscape. It covers the pipeline drug profiles, including clinical, non-clinical stage products and key companies involved such as Pfizer, Novo Nordisk, Hema Biologics, Hoffman-La-Roche, Takeda, and others.
DelveInsight's "Advanced Liver Cancer Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of Advanced Liver Cancer, historical and forecasted epidemiology as well as the Advanced Liver Cancer market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
Familial Lipoprotein Lipase Deficiency Pipeline
DelveInsight’s, “Familial Lipoprotein Lipase Deficiency Pipeline Insight, 2022,” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in the Familial Lipoprotein Lipase Deficiency pipeline landscape. It covers the pipeline drug profiles, including clinical, non-clinical stage products, and key Familial Lipoprotein Lipase Deficiency companies such as Amryt Pharma, Arrowhead Pharmaceuticals, Ionis Pharmaceuticals, iMetabolic Biopharma, Precision Biosciences, Pfizer, Lipigon Pharmaceuticals, and many others.
DelveInsight's "Natural Killer Cell Therapy Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the NK Cell Therapy, historical and forecasted epidemiology as well as the NK Cell Therapy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
Vascular Graft Devices Market report delivers an in-depth understanding of the Vascular Graft Devices, historical and forecasted epidemiology as well as the Vascular Graft Devices market trends, drivers and barriers, and key Vascular Graft Devices companies such as BD, Getinge AB, Medtronic, B. Braun Melsungen AG, Cardinal Health, Terumo Corporation, W. L. Gore & Associates, Inc., JOTEC GmbH, LeMaitre Vascular, Perouse Medical, CryoLife, Inc, and several others.
DelveInsight's "Spinocerebellar Ataxias Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Spinocerebellar Ataxias, historical and forecasted epidemiology as well as the Spinocerebellar Ataxias market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
Interested to know more about the breakthrough happenings? Take a look at the posts below
- The Evolved Gene Therapy for Hemophilia
- A Royal Disease: Hemophilia
- Hemophilia B Market: How Pipeline Therapies are Transforming the Treatment Hemisphere?
Other Trending Reports
- Tay-Sachs Market
- Reactive Airway Disease Market
- Systemic Sclerosis-associated Interstitial Lung Disease Market
- Epidemic Parotitis Market
- Chronic Myelocytic Leukemia Market
- Congenital Hyperinsulinism Market
- Diabetes Insipidus Market
- Limbal Stem Cells Deficiency Market
- Syncope Market
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.
For more insights, visit Pharma, Healthcare, and Biotech News
