Strategic Intelligence: Cell and Gene Therapy Investment Trends
Description
Strategic Intelligence: Cell and Gene Therapy Investment Trends
Summary
Innovative cell and gene therapies (CGTs) have significantly disrupted the treatment landscape for several genetic diseases.
CGT development is still focused on oncology, hematological/blood disorders (such as sickle cell disease, hemophilia, and beta-thalassemia), and neurology. The majority of pipeline cell therapies are autologous therapies. Allogeneic ones are becoming are focused on hematological malignancies.
Investor attention in CGTs peaked in 2021. Since then, dealmaking and fundraising has reduced due to harsher macroeconomic factors and commercial and pricing pressures.
However, funds and strategic pharma investors are re-engaging and favoring companies with clear regulatory paths and differentiated technology (such as novel adeno-associated virus (AAV) capsids, allogeneic platforms), and structuring financing rounds around milestone-based contracts to manage risk.
More recently, investor sentiment toward CGTs has focused on platform scalability, manufacturing capabilities, and clinical validation.
High manufacturing costs, complex supply chains, and reimbursement models, along with renewed safety concerns for some CGTs, have affected the landscape and rendered the funding environment more stringent.
Partnerships, co-development, and licensing deals have emerged as a solution to the challenges of manufacturing complexity and valuation uncertainty. Manufacturing scalability, cost reduction, and automation are now central to deal-making and investors are looking to invest in technologies such as vector optimization, cell engineering platforms rather than only therapeutic developers.
Pharma partners increasingly seek in-house or shared GMP capabilities to mitigate supply chain risks and improve control over quality.
Investors have shifted their interest toward modalities with faster development timelines, broader populations, and clearer regulatory pathways.
Early CGT investments were concentrated in rare and ultra-rare disorders; however, investors are now backing platforms capable of addressing more prevalent diseases such as central nervous system (CNS) disorders, cardiovascular disorders, and metabolic disorders.
Oncology remains an anchor especially for chimeric antigen receptor (CAR) T-cell therapies but there is also a clear push toward non-oncology diseases.
The market size of CGT products is expected to continue to rise by 2031 and reach $63 billion.
CGTs have yielded commercial gains for several pharma companies. For example, some of the top-selling CGT products include Gilead Sciences’ Yescarta (axicabtagene ciloleucel) and Novartis’s Zolgensma (onasemnogene abeparvovec) which reported sales of $1.5 billion and $1.2 billion in 2024, respectively. Additionally, Johnson and Johnson’s Carvykti earned $963 million while Sarepta’s Elevidys earned $820 million in 2024.
Scope
This report provides -
Summary
Innovative cell and gene therapies (CGTs) have significantly disrupted the treatment landscape for several genetic diseases.
CGT development is still focused on oncology, hematological/blood disorders (such as sickle cell disease, hemophilia, and beta-thalassemia), and neurology. The majority of pipeline cell therapies are autologous therapies. Allogeneic ones are becoming are focused on hematological malignancies.
Investor attention in CGTs peaked in 2021. Since then, dealmaking and fundraising has reduced due to harsher macroeconomic factors and commercial and pricing pressures.
However, funds and strategic pharma investors are re-engaging and favoring companies with clear regulatory paths and differentiated technology (such as novel adeno-associated virus (AAV) capsids, allogeneic platforms), and structuring financing rounds around milestone-based contracts to manage risk.
More recently, investor sentiment toward CGTs has focused on platform scalability, manufacturing capabilities, and clinical validation.
High manufacturing costs, complex supply chains, and reimbursement models, along with renewed safety concerns for some CGTs, have affected the landscape and rendered the funding environment more stringent.
Partnerships, co-development, and licensing deals have emerged as a solution to the challenges of manufacturing complexity and valuation uncertainty. Manufacturing scalability, cost reduction, and automation are now central to deal-making and investors are looking to invest in technologies such as vector optimization, cell engineering platforms rather than only therapeutic developers.
Pharma partners increasingly seek in-house or shared GMP capabilities to mitigate supply chain risks and improve control over quality.
Investors have shifted their interest toward modalities with faster development timelines, broader populations, and clearer regulatory pathways.
Early CGT investments were concentrated in rare and ultra-rare disorders; however, investors are now backing platforms capable of addressing more prevalent diseases such as central nervous system (CNS) disorders, cardiovascular disorders, and metabolic disorders.
Oncology remains an anchor especially for chimeric antigen receptor (CAR) T-cell therapies but there is also a clear push toward non-oncology diseases.
The market size of CGT products is expected to continue to rise by 2031 and reach $63 billion.
CGTs have yielded commercial gains for several pharma companies. For example, some of the top-selling CGT products include Gilead Sciences’ Yescarta (axicabtagene ciloleucel) and Novartis’s Zolgensma (onasemnogene abeparvovec) which reported sales of $1.5 billion and $1.2 billion in 2024, respectively. Additionally, Johnson and Johnson’s Carvykti earned $963 million while Sarepta’s Elevidys earned $820 million in 2024.
Scope
- This report examines the evolving funding landscape of cell and gene therapies (CGT) across venture capital (VC) investments, licensing deals, and mergers and acquisitions.
- It identifies key investment trends, analyzes which therapeutic areas and technologies/modalities attract the most capital, and explores the strategic priorities driving big pharma’s involvement in CGTs.
This report provides -
- An in-depth view of the shifting cell and gene therapy (CGT) funding environment covering major venture capital (VC) investments, licensing deals, and mergers and acquisitions.
- Insight into the therapeutic areas, development stage and modalities receiving the most investment.
- Data on the which major VC firms are funding specialized CGTs
- Insight into biopharma’s strategy for acquiring assets and platforms
- Analysis of big pharma’s modality-driven growth strategies and long-term CGT positioning
- Evaluation of the challenges posed by regulatory and commercialization hurdles
- An understanding of the strategic opportunities for both emerging CGT biotechs and established pharmaceutical companies as the field matures
Table of Contents
36 Pages
- Executive Summary
- Introduction
- CGT Approvals and Reimbursement Landscape
- Venture Capital Financing for CGTs
- M&A and Licensing Deals for CGTs
- CGT Catalysts
- Key Takeaways
- Appendix -
- Abbreviations
- Related Reports
- Authors
- Contact Us
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