Written by:
Andrea Jordà, Project Engineer
Jordi Gibert, head of Biotechnology Unit
Advanced therapies, which include cell, gene and tissue engineering therapies, are emerging as a game changer in the treatment of complex diseases. These therapies offer solutions for pathologies that, until recently, had no cure or effective treatment and were considered incurable.
The advance of biotechnology in the pharmaceutical industry has allowed the development of these treatments, with applications such as genetic modification to treat hereditary diseases.
Unlike conventional treatments, which tend to focus on alleviating symptoms, these therapies act directly on the causes of diseases, giving them great potential to improve the quality of life of millions of people. However, despite their advantages, their development and arrival on the healthcare market face major challenges, from production to distribution.
The science behind advanced therapies
The production process for advanced therapies is highly complex and requires specialized facilities and highly regulated processes. Each type of therapy presents different challenges in its production. According to the European Medicines Agency (EMA), medicines based on advanced therapies can be differentiated into [1]:
- Cellular therapies: contain cells or tissues that have been manipulated to change their biological characteristics or that are used for a different function in the body than the original one. An example is the application of stem cells, which have the potential to regenerate damaged tissues.
- Gene therapies: include genes that produce a therapeutic, prophylactic or diagnostic effect. They work by inserting “recombinant” genes into the organism, generally to treat genetic diseases, cancer or chronic pathologies. The most common therapies use viral vectors to introduce the genetic material into the patient’s cells.
- Tissue engineering: combines cells with biocompatible materials to form three-dimensional structures that can repair, regenerate or replace human tissue.
One of the biggest challenges in the production of these therapies is that they still rely heavily on manual processes. Although manual methodology is valid in the early stages of development, it hinders scalability, process standardization and cost control. In addition, a high degree of manual handling increases the risk of variability, contamination and human error.
Clinical trials and approved therapies
The number of clinical trials related to advanced therapies has increased significantly in recent years. According to the American Society for Gene and Cellular Therapies (ASGCT), there are currently more than 4,000 gene and cell therapies in development. However, despite this growth, only 100 have been approved globally for clinical use. As for tissue engineered products, only 8 have received marketing authorization [2].
This gap between innovation and approval is mainly due to high regulatory standards, high research costs and lengthy development time. However, despite these obstacles, FDA and EMA approvals reflect the enormous progress made in recent years and the fundamental role of these therapies in modern medicine.
Barriers to availability and accessibility
Despite advances in research and development, and the opportunities offered by these therapies, there are several obstacles that limit their access to the population.
One of the main challenges is their high cost per dose. The production of these therapies requires specialized facilities, high-quality materials and complex, highly manual manufacturing processes, which drive up the price of the final product. The price of advanced therapies can be several million dollars per patient, raising questions about their long-term availability for many patients and health systems.
These high costs also affect the companies that develop these therapies. An example is the case of bluebird bio, which went from having three therapies valued at $10 billion to selling them for $30 million [4], reflecting the difficulty of recovering investment in this sector.
In addition, the facilities needed for this type of production are expensive and require highly specialized technology and personnel. The lack of adequate infrastructure and the limited capacity to produce these treatments on a large scale can delay their availability to patients.
Finally, the infrastructure for the distribution and administration of advanced therapies remains insufficient, especially in regions outside the most developed countries. This prevents part of the world’s population from having access to these therapeutic innovations.
Reducing costs and facilitating access
To overcome these obstacles and ensure greater accessibility to these revolutionary therapies, it is necessary to implement various strategies:
- Production automation: Incorporating automation technologies in the production of cells and biological materials could reduce manufacturing time and costs. It would also reduce the risks associated with manual handling.
- Design of specialized plants: Currently, the production of advanced therapies is limited to a small number of specialized plants or depends on the outsourcing of services, which increases their price. It is also important to note that a large part of advanced therapies, such as CAR-Ts, are produced in specialized infrastructures within hospitals and research centers, making it difficult to scale them up to the industrial level. With more private funding in this sector, the global expansion of the production infrastructure could be ensured and a greater availability of these drugs could be guaranteed.
- Public-private collaboration: Greater cooperation between governments, companies and academia in research and development of these therapies could reduce production and distribution costs.
- International collaboration in infrastructure: Establishing global production centers and harmonizing international regulations would facilitate access to these therapies in different regions of the world.
Conclusiones
Advanced therapies represent one of the greatest promises for the treatment of serious and complex diseases. However, high production costs, scalability challenges and regulatory barriers continue to limit their global availability.
The future of these therapies will depend on the development of new automation technologies, innovative financing models and an adequate infrastructure for their production and distribution. With advances in science and technology, it is hoped that these therapies will cease to be a privilege and become an accessible standard for all those who need them.
Bibliography:
- [1] Advanced therapy medicinal products: Overview: European Medicines Agency (EMA). (n. d.). European Medicines Agency (EMA). https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview
- [2] Tissue Engineered Products with a valid marketing authorisation – Paul-Ehrlich-Institut. (n. d.). https://www.pei.de/EN/medicinal-products/atmp/tissue-engineered-products/tissue-engineered-products node.html?cms_gts=166826_list%253DdateOfIssue_dt%252Basc
- [3] 10 of the Most Expensive Drugs in the U.S. (2024, April 1). Drugs.com. https://www.drugs.com/article/top-10-most-expensive-drugs.html
- [4] Mast, J. (2025, February 21). Bluebird Bio sells itself to Carlyle, SK Capital for less than $30 million. STAT. https://www.statnews.com/2025/02/21/bluebird-bio-sells-itself-to-carlyle-sk-capital-for-less-than-30-million/#:~:text=Bluebird%20Bio%20announced%20Friday%20it%20would%20sell%20itself,deal%20that%20lets%20the%20beleaguered%20biotech%20avoid%20bankruptcy.
- [5] 2024 advanced therapy industry report(n. d.). Deloitte.com. https://www2.deloitte.com/us/en/pages/life-sciences-and-health-care/articles/cell-gene-therapy-survey-2024.html
