Hemophilia A - Pipeline Insight, 2021

DelveInsight’s, “Hemophilia A - Pipeline Insight, 2021,” report provides comprehensive insights about 30+ companies and 40+ pipeline drugs in Hemophilia A 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.
Geography Covered
• Global coverage
Hemophilia A Understanding
Hemophilia A: Overview
Hemophilia A, also known as classical hemophilia, is a genetic bleeding disorder caused by insufficient levels of a blood protein called factor VIII. Factor VIII is a clotting factor. Hemophilia A is caused by disruptions or changes (mutations) to the F8 gene located on the X chromosome. This mutation may be inherited or occur randomly with no previous family history of the disorder (spontaneously). Hemophilia A is mostly expressed in males but some females who carry the gene may have mild or, rarely, severe symptoms of bleeding.
A diagnosis of hemophilia A is based upon identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized laboratory tests. The main medication to treat hemophilia A is concentrated FVIII product, called clotting factor or simply factor. There are two types of clotting factor: plasma-derived and recombinant. The U.S. Food and Drug Administration (FDA) has approved NovoSeven RT, a genetically engineered (recombinant) version of activated coagulation factor VII (factor VIIa), for the treatment of inhibitors in hemophilia A. In 2020, FDA approved Sevenfact (recombinant human coagulation factor VIIa expressed in the mammary gland of genetically engineered rabbits and secreted into the rabbits’ milk) for treatment and control of bleeding in adults and adolescents age 12 and older with hemophilia A or B with inhibitors (neutralizing antibodies).
Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia A R&D. The therapies under development are focused on novel approaches for Hemophilia A.
Hemophilia A Emerging Drugs Chapters
This segment of the Hemophilia A report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hemophilia A Emerging Drugs
• SPK-8011: Spark Therapeutics
Spark Therapeutics is developing SPK-8011, an investigational gene therapy for hemophilia A, or factor VIII deficiency. Spark retains global commercialization rights to its SPK-FVIII program includes both SPK-8011 and SPK-8016 for hemophilia A. It is currently in Phase III stage of development and is being developed by Spark Therapeutics.
• BIVV001: Sanofi Genzyme
BIVV001 (efanesoctocog alfa) is a recombinant coagulation factor VIII Fc – von Willebrand Factor – XTEN Fusion protein which is being developed by Sanofi Genzyme for Haemophilia A. It is currently in Phase III stage of dvelopemnt.
Further product details are provided in the report……..
Hemophilia A: Therapeutic Assessment
This segment of the report provides insights about the different Hemophilia A drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players working on Hemophilia A
There are approx. 30+ key companies which are developing the Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. Phase III include, Spark Therapeutics.
• Phases
DelveInsight’s report covers around 40+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

• Route of Administration
Hemophilia A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical

• Molecule Type
Products have been categorized under various Molecule types such as
• Vaccines
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule

• Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hemophilia A: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Hemophilia A therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hemophilia A drugs.

Hemophilia A Report Insights
• Hemophilia A Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
Hemophilia A Report Assessment
• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions
Current Scenario and Emerging Therapies:
• How many companies are developing Hemophilia A drugs?
• How many Hemophilia A drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for Hemophilia A?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hemophilia A and their status?
• What are the key designations that have been granted to the emerging drugs?
Key Players
• Spark Therapeutics
• Sigilon Therapeutics
• ASC Therapeutics
• Pfizer
• Sanofi Genzyme
• Novo Nordisk

Key Products
• SPK-8011
• SPK-8016
• SIG-001
• ASC618
• ASC518
• PF-07055480
• Fitusiran
• BIVV001
• Mim 8


Introduction
Executive Summary
Hemophilia A: Overview
• Structure
• Mechanism of Action
Pipeline Therapeutics
• Comparative Analysis
Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
Late Stage Products (Phase III)
• Comparative Analysis
SPK-8011: Spark Therapeutics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report
Mid Stage Products (Phase II)
• Comparative Analysis
BT200: Band Therapeutics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report
Early Stage Products (Phase I/II)
• Comparative Analysis
ASC618: ASC Therapeutics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report
Preclinical Stage Products
• Comparative Analysis
ASC518: ASC Therapeutics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report
Inactive Products
• Comparative Analysis
Hemophilia A Key Companies
Hemophilia A Key Products
Hemophilia A- Unmet Needs
Hemophilia A- Market Drivers and Barriers
Hemophilia A- Future Perspectives and Conclusion
Hemophilia A Analyst Views
Hemophilia A Key Companies
Appendix
Table 1 Total Products for Hemophilia A
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Figure 1 Total Products for Hemophilia A
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

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