A new era of cell and gene therapy signals new market opportunities
A new era of cell and gene therapy signals new market opportunities
By Ed Ahn | Published: 2025-10-24 13:53:00 | Source: MedCity News
For many years, cell and gene therapies have been mainly associated with cancer Rare and orphan diseasesthose that affect small groups of patients and lack effective treatment options. However, recent developments in this area, including high-profile acquisitions and major regulatory changes, indicate a paradigm shift. Cell and gene therapy are becoming increasingly commercially viable, with the potential to improve the lives of millions.
As these treatments reach larger populations and offer promise Live longer and betterNew opportunities are likely to emerge in the market.
Aim for broad impact
In June 2025, Eli Lilly announced that it has acquired Verve Therapeutics for $1 billion. Verve develops single-dose gene-editing therapies designed to treat chronic diseases. The company’s flagship treatment, Verve-102, targets PCSK9, a gene vital for controlling cholesterol levels. A phase 1b clinical trial is currently underway.
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 individualized therapy.
cardiovascular diseases, Which includes the conditions Such as heart disease, heart attack, stroke, heart failure, arrhythmia, heart valve problems, It affects approximately half of adults in the United States And he The leading cause of death worldwide. In other words, it’s certainly not rare.
The Lilly acquisition demonstrates that even the largest pharmaceutical companies see cell and gene therapy as no longer limited to rare diseases and niche applications. Moreover, the concept of a single-dose treatment that provides a permanent cure for cardiovascular disease represents a huge leap forward for patients who used to require continuous care.
It is reasonable to conclude that as interest deepens and applications expand, cell and gene therapies could fundamentally change treatment paradigms on a large scale.
Regulatory changes expand access
Regulatory changes are necessary to expand approval of cell and gene therapy, and in June 2025, the US Food and Drug Administration (FDA) specifically decided Eliminate Risk Evaluation and Mitigation Strategies (REMS) Requirements for the administration of chimeric antigen receptor (CAR) T-cell therapy. This move effectively expands patient access to immunotherapy that can treat — And sometimes treatment – Some types of leukemia.
CAR-T cell therapy It involves modifying a patient’s T cells by adding a gene that enhances their ability to fight cancer. Historically, these treatments were only accessible in tightly controlled health care facilities, and patients were required to stay nearby for four weeks after infusion therapy.
Now, CAR-T cell therapy can be administered in a wider range of locations, including healthcare facilities in non-urban areas, and the mandatory observation time after infusion 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 indicates growing confidence in the safety and effectiveness of cell and gene therapies, and is expected to lead to increased confidence in these treatments. Increased uptake of cancer immunotherapyAnd creating strong investment prospects.
Treatment of degenerative conditions
Another factor to consider is the increasing demand for treatments for degenerative conditions, which are often age-related, e.g The global population aged 60 or over has nearly doubled and Life expectancy increases.
Cardiovascular disease is one such condition, although there are others that affect increasingly large populations: hundreds of millions of people are affected worldwide by Alzheimer’s disease, Parkinson’s disease, Chronic lung diseasesDiabetes complications, osteoporosis, and more.
Cell and gene therapies show great potential in treating these conditions. By targeting underlying mechanisms—chronic inflammation, oxidative stress, and poor tissue repair—these treatments 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 point to growth in the cell and gene therapy market and to a future in which people can enjoy longer, healthier lives. In the coming years, we can expect more clinical trials with larger participant numbers, additional high-profile acquisitions, and a regulatory environment that increasingly supports innovative therapies on a broader scale.
As these treatments become more widespread and accessible, the market opportunities will grow. For those who have traditionally associated cell and gene therapy with rare diseases, high costs, and limited access, now is a good time to reconsider that view.
Photo: Withaya Prasongsin, Getty Images
As CEO of the company Medipost Company, Ed Ahn She leads the global expansion of CARTISTEM – the world’s first allogeneic stem cell treatment for knee osteoarthritis (OA) and advanced regenerative medicine innovations. With a deep commitment to translating scientific discoveries into real-world treatments, he guides the company’s efforts to redefine the landscape of treating degenerative diseases.
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