Cell and Gene Therapies in Hemophilia A and B – Disease Overview, Treatment Options and Future Market Assessment
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Cell and Gene Therapies (CGT) in Hemophilia A and B Market Report Overview
The hemophilia A and B CGT market reached $12 million in 2023 with massive expansions across therapeutic areas.
The CGT in Hemophilia A and B market research report will aid clients in gaining insight into the competitive landscape of leading CGT agents in Hemophilia A & B including launch date projections, key clinical trial analysis, analyst consensus forecasts, likelihood of approval analysis, and commentary on current and future players. The report also includes an outlook from four key opinion leaders in the 5EU, US, and China and analyzes key unmet needs in the space. Additionally, the report includes commentary on the regulatory landscape of CGTs and the reimbursement environment.
Key Players | · BioMarin
· CSL Behring · Pfizer · Spark · Roche · ASC Therapeutics |
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Cell and Gene Therapies (CGT) in Hemophilia A and B – Key Drivers
- Increasing patient awareness and preference for more convenient therapies that do not require regular prophylaxis.
- Ongoing trials on patients with AAV antibodies and long-term follow-up data further instill physician confidence and expand CGTs’ applicability, allowing a more robust growth potential for hemophilia CGTs.
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Cell and Gene Therapies (CGT) in Hemophilia A and B – Current Treatment Options
The current treatment options including adeno-associated virus (AAV)-based GTs, Roctavian, and Hemgenix, aim to provide long-term bleed protection, minimizing patient burden. However, the current and pipeline hemophilia CGTs are not robust enough to address the concerns about the modality’s durability, safety, and patient access. The limited geographical reach of GTs from established pharmaceuticals provides a market opportunity for biotechs with niche modality focus and domestic expertise to emerge.
Cell and Gene Therapies (CGT) in Hemophilia A and B – Future Market Assessment
Within two years, the hemophilia space will see greater competition pressure due to the presence of the market-leading BioMarin and well-established pharmaceuticals with strong marketing capabilities. Asian players are seeking “blue ocean” opportunities beyond the Western market. New GT players must seek clinical differentiation in their products, in addition to more robust HCP engagement strategies, to break into the hemophilia market.
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Cell and Gene Therapies (CGT) in Hemophilia A and B – Competitive Landscape
The hemophilia CGT pipeline is dispersed over drastically different developers, ranging from cross-TA, international pharmaceuticals (Pfizer, Roche) with extensive commercialization experience, to pre-revenue biotechs with a niche focus on gene editing or delivery technologies. Despite Pfizer and CSL leading the race as well-established pharmaceuticals, the limited geographical reach of their GT clinical development provides a market opportunity for smaller, domestic biotechs to emerge in hemophilia. Smaller future players are equipped with novel payload designs, alternative delivery strategies, and robust manufacturing capabilities to approach the market with higher readiness.
Key CGT in Hemophilia A and B Players
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Scope
Contents include –
Executive Summary
Disease Overview
Current Treatment Options and Unmet Needs
Future Market Assessment
Likelihood of Approval and Phase Transition Success Rate Analysis
Appendix
Key Highlights
Report deliverables include a PowerPoint report
Clinical trial analysis includes all countries
4 KOLs interviewed
Reasons to Buy
OBTAIN A COMPETITIVE ASSESMENT FOR CELL & GENE THERAPIES IN HEMOPHILIA A & B ACROSS MULTIPLE REGIONS
Our modality-specific reports answer questions such as –
• What is the future global market value of CGT in Hemophilia A&B?
• Which patient groups are more likely to receive these therapies?
• What is the price of these agents and what is the reimbursement landscape?
• What is the key opinion leaders’ outlook for the products?
• What are key launch dates and other catalysts in the area?
Pfizer
ASC Therapeutics
Bayer
Roche
Biocad
Expression Therapeutics
Belief Biomed
Sichuan Zhishan Weixin Biotechnology
CSL Behring
Vitalgen
Table of Contents
Frequently asked questions
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How can patients with Hemophilia A and B be treated?
Patients can be treated in a prophylactic manner, whereby regular injections ensure proper maintenance of factor levels; or by episodic, on-demand treatment.
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What is driving the CGT in the Hemophilia A and B market?
Increasing patient awareness and preference for more convenient therapies that do not require regular prophylaxis is driving the CGT in the Hemophilia A and B market.
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Which are the current treatment options in the CGT in the Hemophilia A and B market?
Current AAV-based gene therapies are used for treating CGT in the Hemophilia A and B market.
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Which are the key players in the CGT in the Hemophilia A and B market?
BioMarin, CSL Behring, Pfizer, Spark, Roche, and ASC Therapeutics are a few of the key players in the CGT in the Hemophilia A and B market.
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