A New Era for Cell and Gene Therapy Signals New Market Opportunities

For years, cell and gene therapies were mainly associated with rare and orphan diseases, those that impact small patient groups and lack effective treatment options. However, recent developments in this space, including a high-profile acquisition and key regulatory changes, point to a paradigm shift. Cell and gene therapy is becoming increasingly commercially viable, with the potential to improve the lives of millions.

As these therapies reach larger populations and offer the promise of living longer and better, new market opportunities will likely emerge. 

Aiming for widespread impact 

In June 2025, Eli Lilly announced that it was acquiring Verve Therapeutics for $1 billion. Verve develops single-dose gene editing therapies designed to treat chronic diseases. The company’s leading treatment, Verve-102, targets the PCSK9 gene, which is vital for controlling cholesterol levels. A Phase 1b clinical trial is currently in progress. 

According to Lilly, Verve-102 has the potential to be the first in vivo gene editing therapy for broad patient populations and could shift the treatment paradigm for cardiovascular disease from chronic care to one-and-done treatment.

Cardiovascular disease, which includes conditions like heart disease, heart attack, stroke, heart failure, arrhythmia, and heart valve problems, impacts nearly half of US adults and is the leading cause of death globally. In other words, it’s certainly not rare. 

Lilly’s acquisition makes it clear that even the largest pharmaceutical companies view cell and gene therapy as no longer limited to rare diseases and niche applications. Furthermore, the concept of a single-dose therapy that provides lasting treatment for cardiovascular disease represents a huge leap forward for patients who are accustomed to seeking ongoing care. 

It’s reasonable to infer that as interest deepens and applications expand, cell and gene therapies could fundamentally change treatment models at a large scale.

Regulatory changes broaden access 

Regulatory changes are essential for expanding cell and gene therapy adoption, and in June 2025, the US Food and Drug Administration (FDA) notably eliminated the Risk Evaluation and Mitigation Strategies (REMS) requirement for administering Chimeric Antigen Receptor (CAR) T-cell therapy. This move effectively broadens patient access to an immunotherapy that can treat — and sometimes cure — certain blood cancers.

CAR-T cell therapy involves modifying a patient’s T cells by adding a gene that boosts their cancer-fighting ability. Historically, these treatments were only accessible at highly controlled healthcare facilities, and patients were required to stay nearby for four weeks after an infusion treatment.

Now, CAR-T cell therapy can be administered in a broader range of locations, including healthcare facilities in non-urban areas, and the mandated post-infusion observation time has been reduced to two weeks. With fewer logistical barriers, more patients will have the opportunity to receive a potentially life-changing blood cancer treatment. 

This regulatory easing suggests growing confidence in the safety and efficacy of cell and gene therapies and is expected to increase the uptake of cancer immunotherapy, creating strong investment prospects.

Treating degenerative conditions 

Another factor to consider is the rising demand for treatments for degenerative, often age-related conditions, as the global population of people who are 60 years or older nearly doubles and life expectancy rises. 

Cardiovascular disease is one such condition, though there are others that affect increasingly large population numbers: hundreds of millions of people are impacted globally by Alzheimer’s, Parkinson’s, chronic lung diseases, diabetic complications, osteoarthritis, and more.

Cell and gene therapies show significant potential in treating these conditions. In targeting the underlying mechanisms — chronic inflammation, oxidative stress, impaired tissue repair — these therapies can help patients maintain function and vitality while delaying or eliminating the need for invasive surgeries and improving quality of life. 

Advancing a new era of health and longevity 

Overall, these trends indicate a growing cell and gene therapy market and a future where people can enjoy longer, healthier lives. In the coming years, we can expect more clinical trials with larger numbers of participants, additional high-profile acquisitions, and a regulatory environment that increasingly supports innovative treatments on a larger scale.

As these therapies become more mainstream and accessible, market opportunities will only grow. For those who have traditionally associated cell and gene therapy with rare diseases, high costs, and limited access, now is the time to reconsider that view.

Photo: Witthaya Prasongsin, Getty Images

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