New frontiers in cell and gene therapies: A growing market

This article was co-authored with Jenny Yu, Chemical & Life Sciences Practice Leader for the UK and Ireland at Marsh. As this is a fast moving topic, please note that this article is current as at 22/04/2024. For further information, please contact Paula Margolis, Samantha Silver, or Jenny Yu.

Stem cell and gene therapies (CGTs) are forms of personalised, regenerative medicine, described as some of the most innovative and promising disciplines in modern science. Also known as “advanced therapy medicinal products” (ATMPs), they involve the use of cells, tissues, and/or genes to treat and potentially eliminate a range of complex health conditions and hard-to-treat diseases, ranging from cancer and HIV to Parkinson’s disease and Type 1 diabetes. 

For the life sciences sector, increased funding in the regenerative medicines field has provided unique opportunities for collaboration between government, academia, and industry, paving the way for ground-breaking research and the formation of commercial partnerships. However, the scientifically complex nature of CGTs can give rise to unique legal risks for parties involved in their development, production, and supply.

This four-part “New frontiers in cell and gene therapies” series will consider the current state of the CGT market and provide an overview of some of the most promising scientific advancements. It will also look at the regulatory frameworks governing CGT development and use, the legal and commercial risks facing biotech companies, and the wider impacts on the insurance industry.

CGTs: How do they work?

Stem cell and gene therapies are distinct treatments that can be used independently or in combination with one another. In their simplest form, stem cell therapies aim to modify, repair, or replace damaged cells or tissues, whereas gene therapies aim to replace or repair missing or faulty genes. 

CGTs typically involve extracting cells, protein, or genetic material from a patient or donor for modification or treatment before being reintroduced to the patient.

Some of the main CGTs include:

(i) Stem cell transplantation: This complex procedure involves harvesting healthy stem cells from a patient's body (an autologous procedure) or a donor (an allogeneic procedure). The cells are usually extracted from bone marrow, tissue, or blood, for reintroduction into the recipient . There is a greater risk that a recipient may reject the cells when the cells come from a donor.

(ii) Induced pluripotent stem cell (IPSC) therapy: IPSCs do not exist naturally. They are derived from a patient’s skin or blood cells and then reprogrammed to have embryonic-like properties. This enables the development of the type of human cell required for therapy.

(iii) CAR-T cell therapy: This involves the modification of a patient’s immune cells (T-cells) using genetic material to enable them to better detect and destroy malignant cells.

Market attracts substantial investment

Only a few CGTs have reached clinical use as ATMPs since the first stem cell therapy in 1957. Biotech companies working in the CGT sphere face many hurdles, including the significant costs of CGT development, clinical trial failures, and the challenges of establishing manufacturing processes for commercialisation. Ethical concerns over the modification of cells also continue to have an impact.

However, increased government funding for research and development is encouraging significant investment into the market. For example, a UK Government grant of £9.43 million led to the opening of the Clinical Biotechnology Centre in Bristol in March 2023, which aims to expand the UK’s ability to make clinical grade products to support the research and development of new CGTs.

Advances in new technologies that have increased the scalability of CGT production have also encouraged investment. In particular, the introduction of gene editing technology — known as CRISPR/Cas9 — in stem cell science in 2013 was a fundamental turning point for the industry. It has enabled further research and clinical trials, resulting in the production of cutting-edge treatments.

According to one estimate, the global stem cell therapy market is expected to reach US$21.33 billion in 2026.

Notable advancements 

The development of COVID-19 vaccines using messenger RNA (mRNA) technology propelled cell and gene therapy into the forefront of mainstream news. More recently, noteworthy advancements are treating diseases such as: 

• Leukaemia: In April 2023, the US Food and Drug Administration approved Omisirge®, a substantially modified donor cord blood-based cell therapy to accelerate the recovery of neutrophils in the body and reduce infection risk. 
• HIV: In July 2023, a man dubbed the “Geneva patient” was the latest person to be cured of HIV following a stem cell transplant for cancer treatment. 
• Type 1 diabetes: In October 2023, Vertex Pharmaceuticals presented long-term data on patients dosed with VX-880, a stem cell-derived islet cell therapy for Type 1 diabetics. Following treatment, all patients showed improved glycaemic control, with some patients reported to have achieved insulin independence. 
• Sickle cell and beta thalassemia: In November 2023, the UK’s Medicines and Healthcare products Regulatory Agency approved CASGEVY™, a stem cell therapy that was developed using CRISPR/Cas9 for the treatment of sickle cell disease and beta thalassemia. It is the world’s first approved therapy using the CRISPR/Cas9 technology. US and European regulators followed suit, approving the therapy this year. 

In the next article of our series, we consider the regulatory frameworks governing CGTs, the risks and challenges they present, and how they can help foster innovation in this space.

Read other items in Commercial Brief - July 2024

Part 2: New frontiers in cell and gene therapies: Comparative regulatory frameworks