Cell and gene therapy sector to enter disciplined, sustainable growth phase: ARM

The Alliance for Regenerative Medicine (ARM) launched its annual Cell and Gene State of the Industry Briefing on Monday at the 2026 Biotech Showcase in San Francisco with a bullish message: The cell and gene therapy (CGT) sector is entering a disciplined and sustainable growth cycle, despite persistent headwinds.

ARM CEO Tim Hunt made his case as to why the sector is at an inflection point. Hunt pointed to an industry that he says better understands its challenges and is adapting accordingly, accelerating scientific and competitive forces that are modernizing the ecosystem, while expanding commercial opportunities.

Commercial momentum, capital discipline

Hunt emphasized that commercial and investor interest in CGTs remains strong. Twenty of the 30 largest biopharma companies by market capitalization are currently investing in the development or commercialization of cell and gene therapies, he said.

Four CGT products launched between 2021 and 2025 — Zolgensma, Yescarta, Carvykti, and Breyanzi — have already achieved blockbuster status, according to Hunt, and are fueled by massive year-over-year growth. Collectively, these therapies are poised to reach $2 billion in revenue between 2026 and 2031. Furthermore, ARM is forecasting up to 10 CGT blockbusters by 2031.

According to Hunt, investors and strategic biopharma are driving a disciplined growth cycle. Despite 2025, the CGT sector attracted $11.1 billion in capital across multiple funding sources, with equity offerings accounting for 49% of total funding and venture capital representing 28%. Strategic acquisitions also accelerated during the year, with major transactions involving AbbVie, Bristol Myers Squibb, and Eli Lilly underscoring long-term confidence in the space.

Maturing industry learns hard lessons

During his presentation, Hunt drew on the historical development of monoclonal antibodies as an analogy for the current state of the CGT sector. Like cell and gene therapies today, monoclonal antibodies experienced cycles of advancement and setbacks before ultimately becoming a $250 billion global market.

“That non-linear path is just what you get when you have hot emerging technology, and cell and gene therapy will follow a very similar trajectory, in our view,” Hunt says.

Learning from those earlier cycles, companies investing in CGTs are increasingly prioritizing best-in-class therapies aimed at larger patient populations with high unmet medical need. Hunt cited active development programs targeting Rett syndrome, Duchenne muscular dystrophy (DMD), Huntington’s disease, AATD, Parkinson’s disease, and Multiple Myeloma.

At the same time, developers are addressing long-standing barriers to access by emphasizing outpatient treatment models, advancing in vivo approaches, and refining development strategies to improve safety and durability.

Accelerating modernization

Global competition has emerged as a powerful catalyst in the CGT landscape. Hunt noted that in 2025, the Asia-Pacific region surpassed North America in the number of CGT clinical trials for the first time, driven by China’s efforts to attract development.

Other regions are becoming increasingly invested in this space as well. Countries in the Middle East are launching ambitious national strategies to attract biotech investment, while the European Union is advancing the EU Biotech Act, which was released in December 2025, and is designed to strengthen and streamline regulatory processes for clinical trials and advanced therapies, including CGTs.

That international momentum is reshaping expectations for how quickly and efficiently CGTs must be developed and evaluated worldwide.

US regulatory evolution

Amid intensifying global competition, Hunt described how ARM and its board engaged throughout 2025 with U.S. policymakers and regulators, including FDA Commissioner Marty Makary, CMS Administrator Mehmet Oz, and HHS Secretary Robert F. Kennedy Jr.

According to Hunt, those discussions reinforced alignment between ARM’s mission and the Make America Healthy Again (MAHA) initiative, particularly around shifting healthcare toward addressing root causes and away from chronic disease management.

That alignment was underscored by new FDA guidance issued on Jan. 11, which grants increased regulatory flexibility around chemistry, manufacturing, and controls (CMC) requirements for cell and gene therapies in an effort to expedite patient access.

The guidance is intended to better accommodate the characteristics of CGTs while maintaining rigorous quality standards through appropriate control measures.

“The agency’s more flexible approach has been, and is expected to continue to be, helpful in expediting product development and will help guide the FDA’s evaluation of development strategies in preparation for a Biologics License Application (BLA) submission,” the FDA said in a release.

According to Hunt, ARM hopes these regulatory changes will help unlock new development pathways and reduce costs for both ultra-rare diseases and broader patient populations, including through the FDA’s New Plausible-Mechanism Pathway and the use of umbrella trials for gene-editing therapies.

Patients are the industry’s north star

Hunt identified patient impact as a guiding light for the CGT sector. While not every therapy works for every patient, he argued that when cell and gene therapies succeed, their effects are life changing.

“The patient impact is irrefutable, I think most people would agree with that,” says Hunt. “Not that every product is perfect, not that every patient has a great response to every therapy, but when these therapies work, they’re transformative.”

Hunt highlighted two key patient stories in this space that have come to define the promise of CGTs. Among them was Baby KJ, whose life was saved through a bespoke CRISPR-based treatment, and Marci McCue, the first patient to participate in a CAR-T clinical trial for multiple sclerosis.

Hunt also addressed a critical industry concern that continues to shape perceptions of the field: patient mortality. Across all adeno-associated virus (AAV) gene therapy programs, treatment-related mortality has remained approximately 0.2%, a rate lower than that associated with several comparable, one-time medical interventions.

He highlighted AAV gene therapy programs showing resilience for Rett syndrome, Duchenne muscular dystrophy (DMD), and Danon disease as examples where clinical benefit has consistently outweighed risk.

Friction points that could impact 2026

Despite a more constructive regulatory outlook, Hunt acknowledged several unresolved issues that could slow progress in 2026.

These issues include questions about whether the FDA’s Office of Therapeutic Products (OTP) is adequately staffed, how improved communications can curb unexpected road bumps in the industry, and whether excessive emphasis on methodological purity could limit flexibility for therapies targeting serious diseases.

To bring greater transparency to these concerns, ARM announced it is rolling out a new scorecard designed to track OTP performance against real-world product milestones.

“We want to just have the data on how OTP is performing vis-à-vis some of these major therapies,” Hunt said.