Cell and Gene Therapy – Revolutionising Medicine and Offering Hope

For millions facing the challenges of genetic diseases and other complex illnesses, a new era of treatment is dawning. Cell and gene therapy (CGT) is rapidly transforming medicine, offering the potential to cure diseases at their very source – the genes themselves.

This approach goes beyond traditional treatments that manage symptoms. Cell and gene therapies aim to permanently alter faulty genes, add missing ones, or turn genes on or off. This groundbreaking approach holds immense promise for a vast range of conditions, from inherited disorders to cancers.

The field is rapidly expanding. According to GlobalData, as at April 2024, there are currently 2,016 cell therapies in clinical development globally, with 141 of these currently in Phase III clinical trials. In their State of the Biopharmaceutical Industry 2024 report, it was revealed that 18% of healthcare industry professionals believed that cell and gene therapy will dominate as the industry trend to have the greatest impact on the pharmaceutical industry in 2024.

The potential for life-changing treatments is immense. In this insight we look at the transformative power of cell and gene therapy but also some of the challenges being faced as scientists make new discoveries and explore the complexities of developing new therapy treatments.

What is Cell and Gene Therapy?

Cell and gene therapy is an emerging field aiming to treat or manage conditions by adding healthy genes to compensate for those that are missing, mutated or inactive.

It’s a complex task as it’s a very personalised therapy tailored to individuals and genetics, making it hard to standardise finished products. Variation is one of the biggest challenges with very little time from extraction to delivery.

How does Gene Therapy Work?

Gene therapy works by directly modifying cells. It can repair faulty genes, introduce new genes into the body or stopping those genes which are causing harm. By doing so, it offers the potential to restore normal cellular function helping to manage or cure a wide range of diseases.

Cell Therapy Procedures

There are two cell therapy supply chain steps – ex-vivo and in-vivo gene therapy.

Ex-vivo cell therapy is a powerful technique where doctors treat diseases by modifying a patient’s cells outside a patient’s body in a laboratory. This process involves several steps:

  • Cell extraction: Doctors first extract specific cells, often stem cells, from the patient’s body. These are then transferred to a manufacturing site.
  • Genetic modification: In a specialised laboratory, scientists introduce new genetic material or repair faulty genes within these extracted cells. This is often achieved using vectors, such as viruses or plasmids (non-viral method), to deliver the desired genetic changes.
  • Cell expansion: Once modified, the cells are carefully grown and multiplied in a controlled environment to create a sufficient number for therapy.
  • Quality control: Rigorous testing ensures the modified cells are safe and function as intended. Once quality procedures have been satisfied, the cells are harvested and packaged, ready for transfer and delivery to the patient.
  • Reintroduction: Finally, the engineered cells are infused back into the patient’s body where they will be able to target and eliminate diseased cells or produce a missing or deficient protein or hormone which will help cure or enhance a patient’s quality of life.

In-vivo delivers gene therapy directly to a patient’s cells within the body. This eliminates the need to extract and modify cells in a lab. This process is broken into two principal procedures:

  • Delivery of Genetic Material: In-vivo approaches use vectors such as viruses or liposomes to carry therapeutic genes into the target cells inside the body. This can be done through an IV or by local injection to a specific organ.
  • Gene Editing Inside the Body: Once delivered, the vectors introduce the desired genetic material into the patient’s cells. This may involve repairing faulty genes, adding missing genes, or instructing cells to produce a specific protein.

A simpler procedure, in-vivo therapy avoids the complex steps of cell extraction, modification, and reintroduction involved in ex-vivo therapy. This approach offers hope for treating diseases in organs or tissues that are difficult to access for cell extraction.

Key Challenges for Stem Cell Manufacturers

Costs: The manufacturing cost of developing a new cell or gene therapy is exceedingly expensive, requiring significant investment which can be limiting for smaller biotech companies. Venture capital funding has decreased significantly, making it harder to raise money. According to a report by Global Data, the cost of developing a new therapy is estimated at $1.94 billion. Some therapies can be expensive to produce due to facility needs, limited automation and complex cycle management. Combined with unpredictable expenses, this can make budgeting difficult.

Scaling-up: Difficulties can also arise in scaling up production processes and taking small batch sizes to large, commercial quantities. Certain therapies, such as CAR-T therapies, are personalised for each patient and may not be suitable for the large-scale production needed for other common diseases. Lack of suitable equipment available to meet the demand for production can also be a problem.

Based on CPHI’s Annual Industry Report published in 2023, 50% of pharma executives expect that cell and gene therapy manufacturing will experience resource procurement and capacity limitations, leading to “slower approvals within the next 12–18 months” and perhaps a moderate decrease in cell and gene therapy manufacturing contract numbers.

Regulatory hurdles: The regulatory process for cell and gene therapy can be complex. Navigating legal procedures and keeping up to date with regulations as they evolve, can add to the time and cost of development. In addition to this, the detailed and lengthy manufacturing process when dealing with personalised therapies can often prove challenging when trying to achieve consistent specifications. This, in turn, can lead to regulatory or legal issues.

Environmental Issues: The industry generates a significant amount of waste, particularly from plastics used in disposable materials which arise as part of the manufacturing process. Currently there are limited waste disposal options and the waste either has to be incinerated or landfilled, neither of which are environmentally friendly.

In a new initiative1, Cell and Gene Therapy Catapult (CGT Catapult) is working with the Innovation Launchpad Network+ on two projects that aim to assess and reduce the environmental impact of the advanced therapies industry. One project, headed by Dr. Alejandro Sanchex Monsalve, an expert in material sustainability at the University of Bath, is aimed at the development of a concept that would allow for the recycling of waste plastic that has been used by the cell and gene industry to be recycled. The second initiative with Dr Laurence Stamford, an expert in sustainable chemical engineering at the University of Manchester, will seek to identify ways to reduce the environmental impact of AAV production by analysing small and large bath production processes.

Lack of Collaboration:  In a relatively new field, there is not always a deep understanding of how to best manufacture and handle cells.  A lack of collaboration between pharma and biotech companies and CDMOs can hinder efficient end-to-end manufacturing.

Quality Control: There is also a lack of reliable and consistent controls for validating the accuracy of tests used in cell therapy and manufacturing and in some instances essential controls for specific cell therapy markers may not be readily available.

One company trying to address this is Slingshot Bio2 who have launched a first-of-its-kind suite of synthetic cells that express controlled amounts of cell-therapy-relevant markers including BCMA and CD19. Another key addition to this product portfolio is a product containing lymphocyte subsets which is comprised of defined populations of cells relevant to patient characterization standardization. These products enable cell therapy developers and researchers to improve their analytical methodologies by providing consistent, cost-effective controls for accurately profiling patients and evaluating therapies. Designed for use in flow cytometry assays, they improve quality control and assay consistency, accelerating research and development toward more effective cell therapy solutions.

Finding Solutions

In a recent report published by Global Data, upcoming trends and challenges in cell and gene therapy was a main topic at the CPHI Europe 2023 conference in Barcelona. Gathered experts emphasised the need for earlier collaboration and the building of strong partnerships between pharma/biotech, CDMOs and other stakeholders to ensure successful manufacturing from pre-clinical stages and to overcome challenges and accelerate commercialisation.

There was a need for investment in larger facilities by CDMOs to accommodate the growing demand for therapies. The development of common platforms for manufacturing across different therapies can scale-up and streamline production.

The application and impact of AI was a common theme across talks. Highlighting possibilities to positively reshape the landscape, the use of AI algorithms for target identification, gene editing and other aspects has the potential to accelerate drug development.

Cell and Gene Therapy Supply Chain Solutions from Oximio

Oximio has been supporting the dynamic evolution of clinical trials for over 20 years. With state-of-the-art warehouse facilities, our own courier fleet and regulatory industry experts based across Europe, the Middle East, Africa, supported by reliable, global network partners, we are able to support all aspects of cell and gene logistics.

Discover our comprehensive range of clinical trial logistics services or contact us to find out how we can work with you in the fulfilment of your clinical trial requirements.


1: https://ct.catapult.org.uk/news/new-projects-aim-to-improve-environmental-sustainability-of-the-cell-and-gene-therapy-industry

2: https://www.businesswire.com/news/home/20240326777742/en/Slingshot-Unveils-a-Novel-Cell-Therapy-Focused-Portfolio-to-Streamline-Development-while-Improving-Manufacturing-and-Clinical-Trial-Capabilities/?feedref=JjAwJuNHiystnCoBq_hl-RLXHJgazfQJNuOVHefdHP-D8R-QU5o2AvY8bhI9uvWSD8DYIYv4TIC1g1u0AKcacnnViVjtb72bOP4-4nHK5ieT3WxPE8m_kWI77F87CseT