Guest Column | January 28, 2025

Catalysts of Change: How the Cell and Gene Therapy Market Has Evolved

By Joseph Pategou

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In 2021, I conducted an in-depth analysis of the cell and gene therapy landscape in the article “Cell & Gene Therapy Financing: What Is The Role for Private Equity and SPACs?” A few key takeaways stood out:

  • The global cell and gene therapy market was projected to reach €1.8 billion in 2021, with North America as the dominant region, housing over 400 companies and controlling 60% of the market share in 2018.
  • Europe and the Asia-Pacific (APAC) regions followed as significant players in the market.
  • Between 2018 and 2019, public and private institutions in the U.S. and Europe invested more than $15 billion in cell and gene therapies, bolstering product development pipelines.
  • Deal activity surged in 2020, with 141 deals (up from 83 in 2019), including 86 licensing agreements, 25 strategic R&D collaborations, 23 corporate acquisitions, 4 product acquisitions, 2 joint ventures, and 1 other transaction.
  • Stock performance for cell and gene therapy companies was remarkable, with gene therapy firms seeing a 70% increase and regenerative medicine firms achieving a 44% increase.

Evolution of CGT: Key Milestones and Industry Growth

The past four years have brought transformative progress to the cell and gene therapy (CGT) market, with remarkable advancements, new therapeutic frontiers, and significant investments shaping the industry. 

The journey of cell and gene therapy dates to 1972 when Theodore Friedmann first conceptualized gene therapy. Fast forward to 2017, the FDA approved LUXTURNA, KYMRIAH, and YESCARTA, signaling a pivotal moment for the field. A year later, ZOLGENSMA was approved, setting new standards for treating genetic diseases. In 2019, FDA Commissioner Scott Gottlieb projected that by 2025, the agency would approve 10 to 20 CGTs per year. By 2021, ZOLGENSMA had achieved blockbuster status, with annual revenues exceeding $1 billion. In 2022, YESCARTA joined the ranks of blockbuster products. The year 2023 saw the FDA approve five new gene therapies targeting rare genetic diseases, while 2024 marked a record with nine CGTs gaining approval, including the first two adoptive cell therapies for solid tumors.

Recent breakthroughs in oncology have advanced adoptive cell therapy for treating solid tumors, an area that has historically been challenging for cell therapies. Iovance Biotherapeutics achieved the first-ever FDA approval for tumor-infiltrating lymphocyte (TIL) therapy, a treatment for metastatic melanoma. Adaptimmune Therapeutics also received FDA approval for its T-cell receptor (TCR) therapy, which targets synovial sarcoma. These approvals underscore the significant progress being made in the treatment of solid tumors.

In addition to advancements in oncology, in-vivo cell and gene therapies have opened new doors for disease treatment. In-vivo approaches, which involve delivering genetic modifications directly into the patient’s body, have improved both treatment efficacy and accessibility. Interius BioTherapeutics recently initiated the first-ever in-human clinical trial of in-vivo CAR gene therapy, marking a major milestone in precision medicine. Similarly, Intellia Therapeutics entered Phase 3 trials for an in-vivo CRISPR gene-editing therapy aimed at treating hereditary angioedema, becoming only the second in-vivo CRISPR treatment to reach this advanced stage. Myeloid Therapeutics has also administered its first in-vivo mRNA CAR therapy to treat advanced hepatocellular carcinoma, further advancing mRNA technology in oncology. Finally, Umoja Biopharma has advanced clinical trials for a gene therapy that generates CD19 CAR-T cells in situ, targeting relapsed/refractory large B-cell lymphoma and chronic lymphocytic leukemia.

CGT Market Growth and Investment Surge: A Path to Blockbuster Therapies

Today, five CGTs generate over $500 million annually, a figure expected to grow to more than 30 therapies by 2030, fueled by both existing products and late-stage pipeline therapies. Several emerging treatments are poised to achieve blockbuster status, including therapies for Danon disease, which affects between 15,000 and 30,000 patients in the U.S. and EU, dystrophic epidermolysis bullosa, with 9,000 global cases, Duchenne muscular dystrophy, impacting 39,000 patients in the U.S. and EU, and severe sickle cell disease, which affects 25,000 individuals across these regions. Notably, 13 of the 15 largest biopharma companies by market capitalization are actively investing in the development and commercialization of CGTs. By the end of 2024, the number of CGT developers reach 2,936 and 1,975 clinical trials underway, representing annual growth rates of 6% and 3%, respectively. The U.S. continues to lead the field across all these dimensions.

Investment in cell and gene therapy has surged to $15.2 billion in 2024, reflecting a 30% year-over-year increase. Public financings have seen substantial growth, with Insmed raising $650 million in May 2024 and Kyverna’s IPO generating $319 million to support its autoimmune cell therapy mission. Venture capital investment has also been robust, with ArsenalBio securing $325 million in a Series C funding round, Capstan Therapeutics raising $175 million through an oversubscribed Series B, and Obsidian Therapeutics closing a $160.5 million Series C round to advance OBX-115 clinical development.

Strategic Mergers and Acquisitions: Shaping the Future of CGT

Mergers and acquisitions have further bolstered the industry. Roche acquired Poseida Therapeutics for approximately $1.5 billion, strengthening its CAR-T therapy portfolio. Novartis signed a $1.1 billion deal to acquire Kate, a gene therapy biotech company, while MilliporeSigma expanded its manufacturing capabilities through the $600 million acquisition of Mirus Bio.

Nova One Advisor reports that the global cell and gene therapy market was valued at USD 18.13 billion in 2023 and is forecasted to grow at a CAGR of 18.3% from 2024 to 2033, reaching an estimated USD 97.33 billion by 2033. Notably, cell therapy dominated the market in 2023 with a valuation of USD 15.6 billion and is expected to maintain its leading position in the coming years

Conclusion

Since 2021, the CGT market has continued to grow, with new drug approvals, innovative treatments aimed at improving patients’ lives and increasing investments in the sector. CGTs are expanding into new disease areas and employing more advanced therapeutic approaches. However, challenges remain, particularly in the regulatory landscape, which needs to evolve to support not only mergers and acquisitions but also the approval process for these groundbreaking therapies.

Acknowledgment

I want to express my gratitude to the Alliance for Regenerative Medicine for giving me access to data link to Cell and Gene Therapy landscape. The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies.

About the Author

Joseph Pategou has authored over 30 articles in esteemed journals such as Cell and Gene Therapy and Drug Discovery & Development. He is a former consultant at Boston Consulting Group in New York. He continues to hold operational roles in biopharma companies. The views expressed are his own. He holds an MBA from New York University and a master’s degree from HEC Paris. 

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