Global trends in advanced therapies

Global trends in advanced therapies 1080 608 Julia Haro

The advanced therapies are medicines for human use based on gens (gene therapy), cells (cell therapy) and fabrics (tissue engineering), or combinations of these products.

They aim to deliver personalized treatments restoring, improving or replacing damaged or diseased functions of the body, and thus being able to prevent or treat diseases for which there is currently no known effective treatment.

Genes are small segments of DNA that contain the information needed to generate the proteins that make up and maintain our bodies. Genes can have errors (usually called “mutations”) and are inherited or may appear for the first time during a person's life. Any of these variations can interrupt the production of proteins, making them defective or non-existent, leading to health problems or diseases such as cancer, cystic fibrosis, cardiovascular or neurological diseases, among others (1) .

Gene Therapy(GT) aims to treat, cure or prevent affects by modifying problems in genetic information. This technique works by transferring, altering or correcting genetic information, or modulating the expression of genes. Some types of gene therapy use a vector that allows access to the inside of the target cell to introduce the gene or modified genetic material (2).

The basis is a good understanding of how genes work and how modifying them can influence people's health. Researchers around the world are studying in depth the different types of advanced therapies to be able to respond to all these diseases (1).

Both gene therapy and cell therapy fall under the umbrella of advanced therapies, but they work differently, so it's important not to confuse them.

Cell therapyit involves the use of living cells to treat diseases. Cell therapy consideration, as opposed to traditional cell transplantation—such as bone marrow transplantation— is provided by substantial modification of the cells, for example, by expanding or modifying them, and / or use with a different function or place than they originally had in the organism (3).


Figure 1. Cell therapy. Source: Advanced Therapies Report: Cell therapy and gene therapy. Roche Institute (4).

Gene therapyit consists of modifying or replacing defective or absent genes in an individual's genome, or doing so in a cell to improve its action or therapeutic capacity. This may involve the use of viral vectors to introduce desired genes into the body or direct gene editing with techniques such as CRISPR-Cas9, therapeutic DNA/RNA-based technologies or chimeric therapies, as is the case with CAR-Ts ( 3).

Some therapies use a combination of the two. These work by altering genes in specific cells and introducing them once modified into the patient's body (4).

The different types of gene therapy can be classified according to the objective and the methodology used (5):

  • Type of target cell, germ or somatic. When GT is applied to germ cells (sperm or eggs), they cause changes that will affect the individual's future descendants. On the contrary, when GT is used in somatic cells, it only affects the treated individual, without affecting the cells involved in reproduction, and therefore the modifications are not inherited.
  • Strategy: ex vivo and in vivo. In the strategy ex vivo, the target cells are removed and reintroduced into the same patient after being modified in the laboratory. In the strategy in vivo, the administration of the correcting or modifying genetic material is carried out directly to the patient.
    • To insert genetic material into the cells, both in the strategies ex vivo as in in vivo, elements known as “vectors” can be used that act as safe and effective carriers of gene therapy. This vector can be viral (when it comes from a virus) or non-viral (like liposomes).
    • Gene therapies can also be done without using vectors, in which the genetic material can be introduced into the cell by direct injection or by using physical methods such as electroporation, which facilitates the entry of nucleic acids into the cell.
  • Approaches: additive and substitutive. In additive GT, the most widely used and advanced, the genetic material is incorporated with its own regulatory elements, while in substitutive GT, the genetic material remains in its natural context, and is subjected to the expression controls of the cell in which it is introduced.

Figure 2. Different ways to administer gene therapy. Source: Report Advanced therapies: Cell therapy and gene therapy. Roche Institute (4).

The following table specifies the factors that can represent a threat or opportunity for gene therapies, in the different areas of PESTLE (political, economic, social, technological, legal and environmental).

Taulí 1. Key factors in the different areas of PESTLE

Political factors
  • Involvement by governments to modify the current care system.
  • Government grants and incentives to encourage your research.
  • Involvement in legislation and regulation.
Economic factors
  • Very expensive treatments.
  • Public-private collaboration to be able to pay them.
Social factors
  • Possibility of curing diseases that currently have no treatment.
  • Generate trust and reliability in treatments.
Technological factors
  • They involve complex technological processes.
  • Specialized facilities (white rooms) are required.
  • Advances in personalized medicine technology.
Legal factors
  • Modify and adapt the laws for these therapies.
  • Facilitate the increase in the pace of research and technological development.
Environmental factors
  • Significant consumption of material resources for its production (customized production, not on a large scale).

The global gene therapy market, in terms of revenue, generated $7,54 trillion by 2022 and is expected to reach the 17,2 trillion by 2027, which represents a compound annual growth rate (CAGR) of 22% from 2023 to 2030 (6).

Figure 3. Global gene therapy market. Source:

Certain factors may help the expected growth of the global market (7):

  • Gene therapy approvals are on the rise.
  • Investment in gene therapy research and development is on the rise.
  • Expansion of the area of ​​advanced therapies and technologies for the administration of genetic material.
  • Biotech companies' investments in acquisitions, mergers or collaborations as a key strategy to increase internal expertise.
  • The increase in the prevalence of cancer
  • The increase in the visibility of minority diseases of genetic cause.
  • The increase in the size of the population.

However, the high cost of gene therapy is restraining the growth rate of this market.

The gene therapy market can be segmented based on vector type, therapy type, applications, and region (8).

  • Vector type: divided between viral or non-viral vector. The segment of viral vectors, in turn, can be divided according to the type of virus used: retroviruses, lentiviruses, adenoviruses, herpesviruses, poxviruses and others. The non-viral vector segment can be classified into several categories based on their properties and delivery methods; plasmid vectors, gene gun, electroporation, lipofection, nanoparticles, ultrasound and others. The non-viral vectors segment has the largest size within the gene therapy market, and is estimated to have the peak growth during 2027. In the viral vectors segment, retroviruses have experienced a revenue growth of more than 20 % during 2022.

Figure 4. Gene therapy market size and trend segmented by vector type. Source:

  • Type of therapy: The market is classified between therapies ex vivo i in vivo. The therapies segment in vivo, had a higher market share in 2020. Moreover, it is a minimally invasive and effective method, which promotes the growth of the segment and thus promotes the growth of the market.

Figure 5. Size and trend of the gene therapy market segmented by type of therapy (ex vivo / in vivo). Source:

  • Applications: The gene therapy sector has developed mainly in oncology, minority diseases, cardiovascular, neurological and infectious diseases. The oncological disorders segment holds the largest share of the gene therapy market. On the other hand, spinal muscular atrophy dominated the segment by 2022 as a revenue share of 43,4%. Although it is a rare disease, it is one of the most common fatal hereditary diseases in childhood. Hereditary retinal diseases are expected to experience a compound annual growth rate till 2027. The increase in R&D activities focused on the treatment of eye diseases, has boosted this segment.

Figure 6. Gene therapy market size and trend by application. Source:

  • Region: The gene therapy market is located in North America, Europe and Asia-Pacific. North America dominated the market during 2022 and accounted for more than 65% of global revenue. This region is expected to become the most important manufacturer of gene therapies, considering the number of approvals and revenues they will generate in the coming years. Asia-Pacific is estimated to be the regional segment with the highest increase in growth between 2023 and 2030. This is attributed to the large population with unmet medical needs and the growing demand for new technologies for the treatment of rare diseases It may also be due to the local presence of the main companies in the sector and the increase in investment by governments.

Figure 7. Gene therapy market size and trend by application. Source:

Advanced therapies are currently a field in medicine with a high growth potential, thanks to the positive results that provide the opportunity to have more effective treatments for complex diseases. Thus, advanced therapies represent a sector of high strategic interest, and its market can imply a strong economic boost.

However, these therapies present some challenges, discussed in more detail in the next section, such as the high cost of production, which drives small biotech and public sector companies to seek strategic collaborations with manufacturers and large pharmaceutical companies. This also helps to boost the market expansion of these therapies. In addition, investments in this field are growing a lot, which boosts the market even more.

At present, the main players are focusing on the study of new gene therapy products to take advantage of the market growth opportunity. Most of the products are in the preclinical phase, although some are already in the clinical phase awaiting approval in the coming years. This point is important, as regulation in these trials is stricter than regulation in trials with other therapies, to ensure maximum safety. Trials in advanced therapies are also different in that they require specialized spaces, such as biological safety cabinets, for the preparation of medicines.

Figure 8. Number of clinical trials in advanced therapies existing worldwide. Source: Map of capabilities in advanced therapies of biotechnological companies of AseBio.

In the last twelve years, the European Medicines Agency has given green-light to eight advanced therapy drugs. In Spain, currently, there is a high number of clinical trials with this type of therapy, a fact that reflects the growth trend and opportunity of this market, and confirms the need to continue with public-private collaboration for keep moving forward

Advanced therapies are very active areas of development in medicine, which involve some challenges in different areas such as clinical, economic, human resource management, scientific-technical and regulatory, more detailed below:

  • High cost of treatments, since the development and research of these therapies requires a significant investment of time, resources and human capital, in order to guarantee the effectiveness and safety of the treatment.
  • Demonstrate evidence and safety in order to generate trust and for these therapies to be accepted by the population.
  • Production capacity and complex processes, as cell manipulation, genetic modification and the production of biological products require specialized facilities (white rooms) and sophisticated equipment. These specific technologies also impact the high cost of these therapies.
  • Regulation and approval, as advanced therapies require strict regulations and must go through a rigorous approval process, with relevant clinical trials and quality studies, by the relevant regulatory agencies.
  • Collaboration between groups (both private and public) and countries to increase their use.

There are numerous companies involved in the gene therapy market, as listed below. Some of them have drugs that are already approved and can be used for the treatment of genetic disorders, and others have drugs in the preclinical and clinical stages that are waiting to be approved by regulatory authorities.

  • Novartis AG: development of gene and cellular therapies, focused on genetic disorders and cancer (link).
  • Gilead Sciences, Inc: development of cellular therapies, in the fields of virology, oncology and inflammation (link).
  • Spark Therapeutics, Inc: they use adeno-associated viral vectors to treat genetic diseases that affect the retina, the liver and the central nervous system (link).
  • Roche: develops gene therapy for the treatment of Hemophilia A, spinal muscular atrophy and Parkinson's disease. It is also a pioneer in the use of CAR-T cells for oncological treatment (link).
  • Jazz Pharmaceuticals, Inc: development of cellular therapies for the treatment of hematological diseases and solid cancers (link).
  • GlaxoSmithKline plc: development of gene and cellular therapies for various diseases, such as hemophilia, sickle cell anemia, Parkinson's disease, spinal muscular atrophy and HIV (link).
  • Spartea Therapeutics: development of gene therapies for rare neuromuscular diseases (link).
  • Sibiono Gene Tech Co., Ltd.: Its star product is Gendicine, a gene therapy based on a recombinant adenovirus, for the treatment of head and neck cancer (link).
  • Orchard Therapeutics: development of ex vivo gene therapies for rare genetic diseases that affect the immune system, the central nervous system and the blood (link).
  • Bluebird Bio, Inc: Some of its products in development are Zynteglo, a gene therapy for transfusion-dependent beta-thalassemia, and bb2121, a CAR-T cell therapy for multiple myeloma (link).
  • REGENXIBO Inc: development of gene therapies based on AAV for genetic and metabolic diseases (link).
  • Sangamo Therapeutics, Inc: development of cell therapy with CAR-T (link).
  • Intellia Therapeutics, Inc: development of in vivo and ex vivo gene editing therapies for genetic diseases and other diseases (link).

Taulí 2. Competitive landscape of public gene therapy companies, according to technique and development phase (9).

Figure 9. Competitive landscape of public companies that develop gene therapies. Source:

Taulí 3. Most relevant institutions in gene therapy in the United States and Europe (10).

Institution Activity Enllaç
Clinical research in the areas of stem cell transplantation, cell therapy and gene therapy. here
Research focused on the molecular biology of adeno-associated virus with the goal of developing an efficient viral vector system for use in human gene therapy. here
Research in the field of gene therapy. The Vector Center manufactures several recombinant viral vectors. here
Research focuses on cardiovascular, neurodegenerative and metabolism-related diseases. The main target genes are vascular growth factors and the LDL receptor in the cardiovascular field. here
They use gene therapy and oncolytic viruses to improve the treatment of cancers that currently have no effective treatment. here

Taulí 4. Main journals on gene therapy.

Magazine Impact factor Enllaç
Human Gene Therapy 4,2 here
Journal of Molecular Medicine 4,7 here
The Journal of Gene Medicine 3,5 here

Gene therapy legislation aims to protect humans, the general public and the environment. Carrying out gene therapy clinical trials with genetically modified organisms, as vectors, raises unique safety issues. This area is governed by a series of laws and guidelines, some unique to this field, as well as others relevant to any clinical setting.

The main regulatory institutions in Europe and the United States are the following (10):

  • Center for Biologics Evaluation and Research (FDA – CBER) – link
  • European Union – Biotechnology (EU) – link
  • The European Medicines Agency (EMA) – link

In Spain, the authorities involved in the authorization of clinical trials and aspects related to genetically modified organisms (OMG) are theSpanish Medicines and Health Products Agency and the Interministerial Council ofOMG. The aspects related to the OMG of clinical trials with medicines for human use, which contain or consist of OMG, are regulated by the intentional release framework. Requests for authorization in the context of clinical trials and in the context of OMG are not linked (that is, the applicant can decide when to submit the applicationOMG) (11).

Spain is one of the reference countries in advanced therapies, both for the high level of science and publications, and for the number of clinical trials, in all types of advanced therapies (more than 100 per year).

The following tables show some of the companies, hospitals and research centers that are part of the ecosystem.

Taulí 5. Interesting Spanish companies that do research and innovation in gene therapy.

Company Description Enllaç
Develops and manufactures lentiviral vectors. European leader working under EMA and FDA regulations. here
Factory of vectors for clinical trials on gene therapy treatments. here
It develops a gene therapy based on the expression of the telomerase gene, intended to treat diseases related to aging. here
Exploit protein splicing to develop the next generation of gene therapies. here

Taulí 6. Spanish hospitals that carry out research and innovation in gene therapy.

Hospital Description Enllaç
Multidisciplinary team to cover the different phases of development and application of innovative therapeutic strategies. here
They have a research group in gene and cell therapy, and different clinical trials with these treatments. here
They have different clinical trials with advanced therapies to be able to offer the drugs that are in the research phase. here
The Advanced Therapies Unit deals exclusively with cell therapies against cancer and other diseases of the immune system. here

Taulí 7. Interesting Spanish research centers that carry out research and innovation in gene therapy.

Centro  Description Enllaç
It wants to facilitate the introduction of new advanced therapies in the healthcare system, making the BST's white rooms available to researchers. here
Research, development and innovation in advanced therapies. here
They work with the clinical professionals of Clínic Barcelona so that the new CAR-T therapies developed reach the patient as soon as possible. here
The Mixed Advanced Therapies Unit carries out translational research for the development of new advanced therapies. here

In recent years, interest in advanced therapies has grown significantly in Catalonia. There is a significant number of organizations, from research and development centers, entrepreneurial fabric, industry, investors and public administration, which form a very dynamic and constantly growing ecosystem.

There are also new strategic initiatives, such as Center for Advanced and Emerging Therapies of Catalonia (Hub), an infrastructure that will be responsible for guaranteeing the connection between the generation of advanced therapies, within the framework of the public health and research and innovation systems, and the industrial fabric. This project is promoted by the Government of the Generalitat of Catalonia and coordinated by Biocat (12).

The main goals of the future Hub of Advanced Therapies are based on bringing these therapies to the patients who need them as soon as possible. For this reason, they plan to work in the field of research, in infrastructure and manufacturing and with the business fabric. In the Action Plan presented by Biocat, you can find detailed information about the future Hub.

The Parc Taulí University Hospital is committed to advanced therapies, this being one of its strategic lines to which it dedicates resources and facilitates its growth. In this sense, it actively participates in clinical trials, it has promoted the Advanced Therapies Platform - of which the Center for Genomic Medicine, the Clinical Trials and Pharmacy Support Unit are part - in order to participate and collaborate in the implementation of these therapies, and is in contact with the CBATEG (Centre for Animal Biotechnology and Gene Therapy) of the Autonomous University of Barcelona, ​​to be able to carry out gene therapy studies together. The Parc Taulí Research and Innovation Institute (I3PT) also supports research and innovation in advanced therapies.

Currently, the Parc Taulí University Hospital participates in 4 clinical trials on advanced therapies:

  1. Randomized, placebo-controlled, double-blind, multicenter phase IIb study to evaluate the efficacy and safety of HepaStem in patients with acute and chronic liver failure (ACLF) – DHELIVER. Promoter: Promethera Biosciences. IP: Jordi Sánchez Delgado
  2. Open-label, multicenter study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7248824 in participants with Angelman Syndrome. Promoter: Roche Farma. IP: Ana Roche Martínez
  3. Phase I/II, open-label, multiple-dose, step-escalation clinical trial to evaluate the safety and tolerability of GTX-102 in pediatric patients with Angelman Syndrome (AS). Promoter: Ultragenyx Pharmaceutical Inc. IP: Ana Roche Martínez
  4. Implantation of skeletal muscle-derived cells for the treatment of faecal incontinence: a phase III, randomized, controlled, double-blind, two-treatment-arm clinical study. Promoter: Innovacell AG. IP: Laura Mora López.

In the coming years, research and innovation focused on this area is expected to increase significantly, to be in line with the future of personalized medicine.

Julia Haro

Doctorate in Biomedicine by the URV, specialized in nutrition and metabolism. I want to continue contributing my grain of sand to improve the health and quality of life of patients. Currently involved in innovation projects in the Innovation Unit of the I3PT.

All stories by: Júlia Haro

Leave a Reply

Your email address Will not be published.

Privacy preferences

When you visit our website, your browser may store information about specific services, usually in the form of cookies. Here you can change your privacy preferences. Please note that blocking certain types of cookies may affect your experience on our website and the services we offer.

Enable / disable Google Analytics tracking code
Enable / disable Google Fonts
Turn Google Maps on / off
Enable / disable embedded videos
This website uses cookies, mainly from third party services. You can edit your privacy preferences and / or accept the use of cookies.