DelveInsight’s, “Hemophilia Pipeline Insight, 2022,” report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in Hemophilia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the 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 Hemophilia Pipeline Report
- DelveInsight’s Hemophilia Pipeline Report depicts a robust space with 80+ active players working to develop 80+ pipeline therapies for Hemophilia.
- 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.
- The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia.
- In the report, detailed description of the drug is given which includes 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.
Request a sample and discover the recent advances in hemophilia treatment @ Hemophilia Pipeline Report
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. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems. In rare cases, a person can develop hemophilia later in life. The majority of cases involve middle-aged or elderly people, or young women who have recently given birth or are in the later stages of pregnancy. This condition often resolves with appropriate treatment. Hemophilia is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot. This change or mutation can prevent the clotting protein from working properly or to be missing altogether. These genes are located on the X chromosome. Males have one X and one Y chromosome (XY) and females have two X chromosomes (XX). Males inherit the X chromosome from their mothers and the Y chromosome from their fathers.
Latest Breakthroughs of the Hemophilia Treatment Landscape
- 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.
Find out more about Hemophilia Gene Therapy @ Hemophilia Treatment
Hemophilia Emerging Drugs
- SerpinPC: Centessa Pharmaceuticals
- Fitusiran: Alnylam Pharmaceuticals
- ASC 618: ASC Therapeutics
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
Learn more about the emerging Hemophilia pipeline therapies @ Hemophilia Clinical Trials
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 Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal
- Hemophilia Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule, Gene Therapy
- Hemophilia 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 new drugs for Hemophilia treatment, visit @ Hemophilia Medications
Table of Content
- Introduction
- Executive Summary
- Hemophilia: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Hemophilia– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Fitusiran: Alnylam Pharmaceuticals
- Mid Stage Products (Phase II)
- SerpinPC: Centessa Pharmaceuticals
- Early Stage Products (Phase I)
- Drug name: Company Name
- Preclinical and Discovery Stage Products
- Inactive Products
- Hemophilia Key Companies
- Hemophilia Key Products
- Hemophilia- Unmet Needs
- Hemophilia- Market Drivers and Barriers
- Hemophilia- Future Perspectives and Conclusion
- Hemophilia Analyst Views
- Hemophilia Key Companies
- Appendix
For further information on the hemophilia pipeline therapeutics, reach out @ Hemophilia Companies
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