The global CGT market is expected to reach sales of $76.03 billion by 2030, at a CAGR of 44%, driven by gene-modified cell therapies and gene therapies.
The development of CGTs in oncology is still focused on blood cancers, but there are many solid tumor basket trials in Phase I and II. In hematology, four indications are the main focus for R&D: ß-thalassemia, SCD, hemophilia A, and hemophilia and B.
In the CNS disorders CGT space, the current focus is on rare diseases. However, in the pipeline, neurodegenerative diseases such as AD, PD and MS, are the primary industry focus. A similar trend in was noted in ophthalmology, specifically focused on rare diseases. However, larger indications such as AMD and glaucoma account for a vast number of pipeline therapies.
There will continue to be a need for greater flexibility and a more nuanced approach in cost-effectiveness assessments for CGTs.
Partnership deal-making between large pharma and biotechs has been the favored deal type as a shift in alternative sources to secure capital for CGT drug development. Oncology, CNS, and ophthalmology have had the biggest increases in venture funding over the past few years.
The development of CGTs in oncology is still focused on blood cancers, but there are many solid tumor basket trials in Phase I and II. In hematology, four indications are the main focus for R&D: ß-thalassemia, SCD, hemophilia A, and hemophilia and B.
In the CNS disorders CGT space, the current focus is on rare diseases. However, in the pipeline, neurodegenerative diseases such as AD, PD and MS, are the primary industry focus. A similar trend in was noted in ophthalmology, specifically focused on rare diseases. However, larger indications such as AMD and glaucoma account for a vast number of pipeline therapies.
There will continue to be a need for greater flexibility and a more nuanced approach in cost-effectiveness assessments for CGTs.
Partnership deal-making between large pharma and biotechs has been the favored deal type as a shift in alternative sources to secure capital for CGT drug development. Oncology, CNS, and ophthalmology have had the biggest increases in venture funding over the past few years.
Scope
- Overview of the current and future landscape of cell and gene therapies (CGT). Clients can explore the therapy areas and indications set to drive rapid growth in the CGT market over the near- and long-term. As well as gain an understanding of the CGT marketed and pipeline landscape, with launch date projections, market size, likelihood of approval analyses, and a case study that explores the challenging pricing and reimbursement (P&R) landscape for these therapies. In addition to commentary on deals, financials and valuations set to shape the CGT space over the coming years.
Reasons to Buy
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the CGT in the Oncology, Hemophilia A & B, CNS, and ophthalmology markets.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Oncology, Hemophilia A & B, CNS, and ophthalmology-targeting CGT therapeutics market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.
Table of Contents
(PowerPoint Deck)- Preface
- Contents
- Abbreviations
- 1. Overview - Cell and Gene Therapy Landscape
1.2. What is Cell and Gene Therapy?
1.3. CGT Landscape - Market Size by Molecule Type
1.4. CGT Landscape - Market Size by Therapy Area
1.5. CGT Landscape - Key Players (2023 Versus 2030)
1.6. CGT Landscape - Key Therapies of Top 10 Players (Prescription Sales)
- 2. Deep-Dive into the CGT Landscape - Oncology and Hematology
2.2. CGT in Oncology Overview - Allogeneic Versus Autologous Cell Therapies
2.3. CGT in Hematology Overview
2.4. CGT in Hematology Overview - Marketed Profiles
2.5. Hemophilia Gene Therapies Provide Unmatched Long-Term Bleed Protection Despite More Stringent Eligibility
2.6. Leading Players Have Adopted Similar Messaging and Marketing Strategies
2.7. Medical Needs in Hemophilia A and B Are Not Adequately Addressed
2.8. Major Players Intensify Hemophilia Competition Within One Year’s Time
2.9. Hemophilia CGT Segment Is Expected to Pass $2.14 Billion by 2029
2.10. Hemophilia CGT Landscape Is Mixed With Developers of Different Sizes
2.11. Key Takeaways
- 3. Deep-Dive into the CGT Landscape - Central Nervous System Disorders
3.2. History of the Development of CGT in Central Nervous System Disorders
3.3. Marketed Product Profiles
3.4. Commercial Activities of Marketed CGTs in Central Nervous System Disorders
3.5. Challenges and Opportunities in CGT in Central Nervous System Disorders
3.6. Top 20 Central Nervous System Disorders with CGT Development
3.7. Top Five Central Nervous System Disorders with the Most CGT Pipeline Assets
3.8. Additional Players Expected to Join the Competition Within Five Years
3.9. CGT Candidates Have Lower Likelihood of Approval and Phase Transition Success Rate Versus Indication Benchmarks
3.10. Key Takeaways
- 4. Deep-Dive into the CGT Landscape - Ophthalmology
4.2. History of the Development of CGT Therapies in Ophthalmology
4.3. Marketed Product Profiles
4.4. Commercial Activities of Marketed CGTs in Ophthalmology
4.5. Challenges and Opportunities in CGT in Ophthalmology
4.6. Top 20 Ophthalmology Indications with CGT Development
4.7. Top Five Ophthalmology Indications with the Most CGT Pipeline Assets
4.8. Additional Players Expected to Join the Competition Within Five Years
4.9. CGT Candidates Typically Have Lower LoA and PTSR Versus Indication Benchmarks
4.10. Key Takeaways
- 5. CGT Landscape -Pricing and Reimbursement Challenges and Access
5.2. Casgevy’s Route to Patient Access - Challenges and Opportunities
5.3. France - First to Grant (Early) Patient Access to Casgevy via the Autorisation d'Accès Précoce System
5.4. UK - Interim NHS Managed Access to Casgevy via the Innovative Medicines Fund
5.5. Current Challenges in Conducting Health Technology Assessments for CGTs
5.6. Future Challenges in Conducting Health Technology Assessments for CGTs
5.7. Innovative Payment Model Types with Potential for CGTs in the Future
5.8. Key Takeaways
- 6. CGT Landscape - Deals, Financials, and Valuations
6.2. Deals Landscape - Recent Mergers, Acquisitions, and Strategic Alliances in CGT for 2024
6.3. Deals Landscape - Top Mergers, Acquisitions, and Strategic Alliances in CGT for 2024
6.4. Private Companies Show Signs of Venture Financing Recovery
6.5. Downturn in CGT Investment Continues in 2024
6.6. US NIH SBIR/STTR Grant Funding Increases for CGT
6.7. Less Acquisition Spend as More Private Companies Go Public
6.8. CGT IPOs Completed in 2024, but Number Remains Low
6.9. Higher Trial Cost Estimates Remain for Gene Therapies
6.10. Average Venture Capital Deal Amount by Lead Asset's Molecule Type ($ Million)
6.11. Oncology Remains the Top Therapy Area for Venture Financing
6.12. Key Takeaways
- 7. CGT Landscape - Key Takeaways
- 8. Appendix
8.2. Methodology - Phase Transition Success Rate and Likelihood of Approval Analysis
8.3. GlobalData’s Team - About the Authors
- 9. Contact the Publisher
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Johnson & Johnson
- Legend Biotech Corp
- Gilead Sciences Inc
- Bristol-Myers Squibb Co
- Sarepta Therapeutics Inc
- Novartis AG
- Tenaya Therapeutics Inc
- CRISPR Therapeutics AG
- Intellia Therapeutics Inc
- Sana Biotechnology Inc
- Iovance Biotherapeutics Inc
- Atara Biotherapeutics Inc
- Rocket Pharmaceuticals Inc
- Beam Therapeutics Inc
- MeiraGTx Holdings Plc
- Ultragenyx Pharmaceutical Inc
- Vertex Pharmaceuticals Inc
- RegenxBio Inc
- Allogene Therapeutics Inc
- Krystal Biotech Inc
- bluebird bio Inc
- Pfizer Inc
- CSL Behring
- BioMarin Pharmaceutical Inc
- Roche Holding AG
- Spark Therapeutics Inc
- ASC Therapeutics Inc
- Expression Therapeutics Inc
- BIOCAD
- Vitalgen
- Gene Vector Barcelona
- Sichuan Real & Best Biotech Co., Ltd.
- Kyowa Kirin Co Ltd
- SanBio Co Ltd
- CorestemChemon Inc
- NIPRO CORPORATION
- Holostem Srl
- Aurion Biotech
- FujiFilm
- Japan Tissue Engineering Co., Ltd. (J-TEC)
- Hirosaki Lifescience Innovation, Inc.
- Reliance Life Sciences Inc
- MeiraGTx
- Sumitomo Pharma Co., Ltd
- Kala Bio
- 4D Molecular Therapeutics Inc
- Adverum Biotechnologies
- Astellas Pharma
- Lumos Pharma
- Lyell Immunopharma
- ImmPACT Bio
- Ocuphire Pharma
- Kiji Therapeutics
- REGiMMUNE
- Matica Bio
- Nona Biosciences
- OverT Bio
- MiNK Therapeutics
- Autonomous Therapeutics
- Alpine Immune Sciences
- AviadoBio
- Sangamo Therapeutics
- Umoja Biopharma
- Ascidian Therapeutics
- Voyager Therapeutics
- Xyphos Biosciences
- Kelonia Therapeutics
- China Resources Sanjiu Medical and Pharma (CR Sanjiu)
- Tasly Pharma Group
- Capricor Therapeutics
- Nippon Shinyaku
- Kyverna Therapeutics Inc
