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

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?

Biomarin
Pfizer
ASC Therapeutics
Bayer
Roche
Biocad
Expression Therapeutics
Belief Biomed
Sichuan Zhishan Weixin Biotechnology
CSL Behring
Vitalgen

Table of Contents

1. Preface

1.1. Contents

1.2. Abbreviations

1.3. Related Reports

2. Executive Summary

3. Disease Overview

3.1. Overview of Hemophilia A & B

4. Current Treatment Options

4.1. Hemophilia GTs Provide Unmatched Long-Term Bleed Protection Despite a More Stringent Patient Requirement

4.2. KOLs Highlighted Vastly Different Priorities From Physician and Patient Perspectives on GTs

4.3. BioMarin and CSL Behring Have Adopted Similar Messaging and Marketing Strategies

4.4. Medical Needs in Hemophilia A & B Are Not Adequately Addressed in the Near Future

4.5. KOLs Shared their Concerns on the Limitations of Current Hemophilia Gene Therapies

5. Future Market Assessment

5.1. Major Players Are Expected To Intensify the Competition Within a Year’s Time

5.2. Reimbursement Uncertainties Exist for Hemophilia Gene Therapies

5.3. The FDA Provides Industry Guidance on Developing Hemophilia Gene Therapies

5.4. KOLs Highlighted the Difficulties With Reimbursement for GTs While PROs May Add Support

5.5. Hemophilia A & B Pipeline Is Concentrated in Phase I/II Trials

5.6. Hemophilia A CGT Landscape Is Becoming More Active With Diversified Players

5.7. Early Hemophilia B GT Landscape Is Concentrated With Developers From Asia

5.8. KOLs Compares the Narrower Patient Eligibility of Pfizer’s Beqvez With CSL’s Hemgenix

5.9. KOLs Have Reservations on SPK-8011 and the ET-3 Payload-Carrying GTs

5.10. Hemophilia CGT Segment Are Expected to Pass $2.14 Billion by 2029

5.11. Hemophilia CGT Landscape Is Mixed With a Wide Variety of Developers

6. Likelihood of Approval and Phase-Transition Success Rate Analysis

6.1. Hemophilia CGT Candidates Have Varying LOAs and PTSRs

7. Appendix

7.1. Primary Research: Key Opinion Leader Information

7.2. Bibliography

7.3. About the Authors

8. Contact Us

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