The US blueprint for pharma growth: gene therapy, AI and biosimilars

In his latest column, Joseph Pategou reviews the US pharmaceutical market for cell and gene therapies (CGTs), AI and biosimilars.

In 2023, the global pharmaceutical market reached approximately US$1.6 trillion [1], reflecting a US$100 billion increase from 2022, according to Statista [2]. The growing demand for innovative drugs is expected to drive oncology spending to nearly US$370 billion by 2027, almost doubling its current level [3]. By the same year, biotech medicines will account for  35% of global pharmaceutical spending, encompassing breakthrough CGTs as well as a rapidly expanding biosimilars segment [3].

The US remained the world’s largest pharmaceutical market in 2023, contributing 44% of total global pharmaceutical spending. Data from the Centers for Medicare & Medicaid Services (CMS; MD, USA) indicates that US National Health Expenditure (NHE) grew 7.5% to US$4.9 trillion in 2023—equivalent to US$14,570 per person—representing 17.6% of GDP [4]. Over the 2023–2032 period, NHE growth (5.8%) is projected to surpass GDP growth (4.3%), increasing healthcare’s share of GDP from 17.6% in 2023 to 20.3% in 2032 [4].

These figures reinforce the US’s dominance in the global pharmaceutical industry and its pivotal role in shaping the broader healthcare market. This leads to a crucial question: What factors contribute to this leadership, and where do we see opportunities for further growth?

To address these questions, we will explore three key segments of the pharmaceutical market that have been and will continue to be major drivers of innovation and growth: artificial intelligence (AI), CGTs, and biosimilars.

US dominance in gene, cell and RNA therapy

Gene therapy emerged in the late 1970s with the development of recombinant DNA technology. Key American figures, such as Professor Theodore Friedmann and Martin J Cline, played pivotal roles in its conceptualization and early trials. In response to growing interest, the National Institutes of Health (NIH; MD, USA) established the Recombinant DNA Advisory Committee and introduced federal guidelines. A breakthrough came in 1990 when NIH researchers conducted the first human gene therapy trial, successfully treating a girl with ADA deficiency.

The NIH has continued to lead gene therapy advancements, funding key research initiatives such as the Somatic Cell Genome Editing program. In 2023, NIH awarded a US$40 million grant to Yale School of Medicine (CT, USA) for CRISPR-based therapies targeting brain diseases [5].

In late 2024, US startups in gene, cell and RNA therapy secured significant funding. Judo Bio (MA, USA) raised US$100 million, City Therapeutics (MA, USA) secured US$135 million, and March Biosciences (TX, USA) raised US$28.4 million for CAR-T therapy [6]. Leal Therapeutics (MA, USA) also raised US$45 million for antisense oligonucleotide therapies [6]. Other notable rounds included Trace Neuroscience’s (CA, USA) US$101 million for antisense therapies, Portal Biotechnologies’ (MA, USA) US$7 million for gene editing, Tolerance Bio’s (NC, USA) US$20.2 million for iPSC therapies, and GEMMABio’s (PA, USA) US$34 million for gene therapy [6].

The US remains a leader in gene therapy dealmaking, with Poseida Therapeutics (CA, USA) acquired for US$1.5 billion and Kate Therapeutics (CA, USA) securing a US$1.1 billion deal [6]. The sector’s growth was bolstered by significant initial public offerings and public financings, contributing to North America’s US$11.8 billion investment in 2024 [6]. The US also led global CGT revenue, accounting for 65% of the total in 2024, with continued growth projected in the coming years [6].

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US edge in AI: driving drug discovery, trials and logistics

In 2023, US venture capital investment in AI healthcare totaled US$7.2 billion, with projections reaching US$11.1 billion in 2024—a 26% increase [7]. Since 2021, AI-related venture capital funding has been heavily allocated to three key areas: therapeutics and research (US$12.9 billion), administrative functions (US$6.6 billion) and clinical applications (US$12.5 billion) [7]. According to a 2024 GlobalData Intelligence Center survey, 69% of pharmaceutical companies are investing in AI, surpassing investments in cloud computing (47%) and big data (46%) [8].

AI and machine learning are transforming drug discovery by predicting toxicity, identifying promising compounds, and analyzing large datasets to accelerate research. They also enhance clinical trials through more efficient patient recruitment using electronic medical records and social media. AI-driven drug design is also gaining traction—Aulos Bioscience (CA, USA), for example, announced in December 2023 that its AI-designed monoclonal antibody, AU-007, has entered Phase II trials, targeting solid tumors via interleukin-2 (IL-2) [9].

AI-powered drones are streamlining clinical logistics. Zipline, in partnership with WellSpan Health (PA, USA), is using autonomous drones to deliver prescriptions to patients’ homes and transport lab samples between facilities, reducing delivery times up to sevenfold [10]. Similarly, Matternet’s (CA, USA) drone network connects laboratories and hospitals, ensuring rapid transport of critical samples and improving diagnostics [11]. These advancements are fueling the growth of the global medical drone market, which reached US$1.47 billion in 2024 and is projected to expand at a 15.3% compound annual growth rate (CAGR), reaching US$4.68 billion by 2032 [12].

US biosimilar growth, savings and future opportunities

In 2016, the US had only four approved biosimilars. Today, it has emerged as a leader in the space, with 64 biosimilars approved and 42 launched, according to the Center for Biosimilars (NJ, USA). Adalimumab leads with ten approvals and launches, followed by Pegfilgrastim and Trastuzumab with six each.

The market continues to expand, with 97 biosimilars in development. Since the first launch in 2015, biosimilars have generated US$36 billion in savings, including US$12.4 billion in 2023 alone [13]. 60% of total biosimilar-related savings have occurred in the past 2 years, demonstrating accelerated adoption [14]. These products have contributed to 2.7 billion days of patient therapy, with 495 million additional days enabled by increased competition [14].

By 2030, the global biosimilar market is projected to reach US$74 billion, with the US driving much of this growth. Between 2015 and 2021, the US market saw a remarkable 97% CAGR, and while growth is expected to moderate, it is still projected to maintain a 26% CAGR through 2025 [15].

Looking ahead, 118 biologics will lose patent protection from 2025–2034, representing a US$232 billion opportunity for biosimilars [16]. However, as of June 2024, biosimilars are in development for only 12 of these molecules, suggesting untapped potential and the need for further expansion [16].

Conclusion

The US continues to set the global benchmark in pharmaceutical innovation, with AI, CGT, and biosimilars driving transformative advancements. The nation’s dominance in AI healthcare is streamlining drug discovery, clinical trials and logistics, while its leadership in CGT is fueled by pioneering research, record-breaking investments, and strategic acquisitions. Meanwhile, the rapid growth of the US biosimilar market underscores the potential for significant cost savings and expanded patient access.

About the author:

Joseph Pategou has authored over 30 articles in esteemed journals such as Cell and Gene Therapy, The Indian Economist, Labotech.eu, Drug Discovery & Development and others. A former consultant at Boston Consulting Group (MA, USA) in New York, he has held and continues to hold operational roles in biopharma companies. The views expressed are his own. He holds an MBA from New York University (USA) and a master’s degree from HEC Paris (France). You can connect with and follow him on LinkedIn, Instagram, or Twitter.

Sources

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