Gene therapy reached a turning point in 2026. New approvals and global investments of around USD 12 billion make it clear that gene therapies are no longer a niche technology, but a central growth area within the Life Science industry. As so-called Advanced Therapy Medicinal Products (ATMPs) – innovative medicines that act at the genetic, cellular, or tissue level – they open up treatment options that cannot be achieved with conventional medicines. However, this development also brings increasing requirements for Manufacturing, Regulatory Affairs, Supply Chain, and the professionals responsible for these processes. This article explains what the current state of gene therapy means for companies and careers in the industry.
What Is Gene Therapy?
Gene therapy refers to medical procedures in which genetic material is used in a targeted manner to treat diseases at their biological root cause – rather than merely alleviating their symptoms. Unlike many symptom-oriented treatments, gene therapies directly target genetic or cellular mechanisms, thereby opening up entirely new therapeutic possibilities.
This approach is particularly relevant for diseases caused by genetic changes or for conditions in which conventional therapies have reached their limits – for example, rare hereditary diseases, certain types of cancer, or neurological disorders. In some cases, a single treatment can produce a long-term or even permanent effect – representing a paradigm shift compared with lifelong therapies.
How Does Gene Therapy Work?
Basically, two approaches can be distinguished: In in-vivo gene therapy, the genetic material is introduced directly into the body – for example, via viral vectors that serve as transport means and deliver the therapeutic gene to the target cells. In ex-vivo gene therapy, cells are first removed from the body, genetically altered outside the body, and then returned – an approach that is particularly used in combined cell therapies such as CAR-T. Additionally, a technology for targeted DNA modification (so-called genome editing) has been established with CRISPR-Cas9, which allows for precise correction, replacement, or inactivation of faulty gene segments. These methods significantly expand the therapeutic spectrum and are the basis of many current development programs.
For companies, understanding these approaches is not only scientifically relevant – it largely determines which production processes, quality requirements, and regulatory paths are necessary. In the DACH region, gene therapies are classified as ATMPs.
In Germany and Austria, as EU member states, the EU ATMP regulations apply; in Switzerland, Swissmedic is responsible under its own Swiss law. The competent national authorities are the Paul-Ehrlich-Institut (PEI) in Germany and the AGES Medical Market Surveillance in Austria – each closely coordinated with the EMA for centrally approved products, with the final market authorization granted by the European Commission.
What Are ATMPs and How Is the Approval Landscape Developing in 2026?
ATMPs – short for Advanced Therapy Medicinal Products – include gene therapies, cell therapies, and tissue products. In the EU, the Committee for Advanced Therapies (CAT) of the EMA evaluates these products and prepares a scientific recommendation – the final market authorization is granted by the European Commission. Gene therapies are a subcategory of ATMPs and are therefore subject to their own demanding regulatory framework. The approval landscape will evolve significantly in 2026: with Waskyra (Wiskott-Aldrich Syndrome), Adstiladrin (bladder cancer), and a positive CAT recommendation for Itvisma (spinal muscular atrophy) in April 2026, the list of approved ATMPs has grown to 22 products – and is expected to keep increasing. Many other products are also in the approval process, focusing on rare diseases, oncology, and genetically based conditions.
There is also significant regulatory progress: As part of the EU pharmaceutical legislative reform, which was passed in December 2025, a closer alignment between CAT and CHMP is planned – aiming to streamline approval processes for companies in the long term and create more consistency across therapeutic areas. Global investment in cell and gene therapy reached approximately 12 billion USD in 2025 – an increase of 30 percent compared to 2023. This trend continues into 2026 and signals that the industry is growing not just scientifically, but also economically with significant dynamism.
Which Therapeutic Areas Are in Focus in 2026?
The current development shows a clear concentration on specific areas of application. Particularly active in 2026 are development programs in the following fields:
- Oncology: CAR-T cell therapies and other gene therapeutic approaches for the treatment of blood cancer and increasingly also solid tumors remain a central field of research and development.
- Rare genetic disorders: Conditions like hemophilia, spinal muscular atrophy, or Wiskott-Aldrich syndrome are classic indications for gene therapies, where one-time treatments are expected to have long-term effects.
- Neurological disorders: Gene therapeutic approaches for diseases like Huntington’s are increasingly being clinically tested – however, an approved therapy is not yet available.
- Ophthalmology: Retinal gene therapies are among the most developed fields of application and provide important insights for further indications.
For companies, this development means: The question is no longer whether cell and gene therapy will become relevant – but how quickly and to what extent it will change their own portfolio and organization.
How Is the Growing ATMP Pipeline Changing Organizational Structures?
With each new ATMP approval, not only does the therapeutic offering expand – the demands on the companies developing, manufacturing, and marketing these therapies also increase. Gene therapy is hardly comparable to classical drug development in its complexity. Each phase of the value chain presents specific requirements:
- Research & Clinical Development: New platform technologies, ranging from viral vectors to CRISPR-based genome editing to mRNA approaches, which may be relevant depending on the application in the ATMP context, require specialized scientific expertise that is only limitedly available in the market.
- Regulatory Affairs: ATMPs are subject to their own regulatory framework. With an increasing number of approval processes, the need for experts who understand EMA procedures, CAT requirements, and international regulations from practice also rises.
- Manufacturing: The production of gene therapeutics is significantly more complex than that of conventional drugs. GMP requirements, specialized production facilities, and patient-specific processes make manufacturing a strategic bottleneck factor.
- Quality Assurance & Control: Seamless traceability, strict release processes, and specialized analytical methods are indispensable – and require significant resources.
- Supply Chain: Time-critical transport processes, temperature control, and depending on the type of therapy, Chain-of-Identity and Chain-of-Custody requirements make the supply chain an integral part of the therapeutic success.
As the Aristo Half Year Report 2026 shows, these are exactly the areas where pharmaceutical and biotech companies in the DACH region currently see the greatest need for action.
What Specific Challenges Arise From New Approvals?
New approvals are not just a success for companies – they bring a range of operational and strategic challenges.
- Scaling Production: What works in clinical trials must be made reproducible and economically viable for the market. The tech transfer from development to commercial manufacturing is considered one of the most critical steps in the entire gene therapy process.
- Capacity Bottlenecks: Specialized production facilities for viral vectors or autologous cell therapies are limited in availability. Many companies therefore rely on Contract Development and Manufacturing Organizations (CDMOs), to gain flexibility – although CDMOs also bring their own requirements for quality management and technology transfer.
- Regulatory Monitoring: After approval, the work does not stop – it changes. Post-Market Surveillance, long-term follow-up, lifecycle management, and regulatory adjustments due to process changes require sustained specialized expertise.
- Pricing and Market Access: Gene therapies are costly to produce. At the same time, they are increasingly under political pressure, for example from AMNOG procedures in Germany or HTA assessments at the EU level. Reimbursement eligibility and price negotiations with payers become as critical success factors as the approval itself.
Which Professionals Are in Demand in Gene Therapy?
The growing gene therapy pipeline fundamentally changes the demand for professionals. Not just pure specialists from gene therapy are sought – profiles from pharmaceuticals, biotechnology, and medical technology are needed, bringing experience in regulated environments and the ability to transfer these competencies to new therapy platforms.
Currently, especially in demand are profiles in the following areas:
- Process Development & Scale-up for biological and gene therapeutic processes, e.g. Process Development Scientist Gene Therapy or Tech Transfer Manager ATMP
- GMP Manufacturing with experience in the production of viral vectors or cell therapies, e.g. GMP Manufacturing Specialist Cell & Gene Therapy or Vector Production Specialist
- Regulatory Affairs with ATMP-specific knowledge and EMA experience, e.g. Regulatory Affairs Manager ATMPs
- Quality Assurance & Qualified Person (QP) in regulated biotech environments, e.g. QA Manager ATMP or Qualified Person (QP) Biotech
- Clinical Operations for complex, often international study programs, e.g. Clinical Trial Manager Gene Therapy
- Supply Chain Management focused on temperature-sensitive and patient-specific logistics, e.g. Supply Chain Manager Advanced Therapies
Candidates who combine scientific depth with regulatory or operational practice, i.e., interface profiles, are particularly in demand – a combination that is only limitedly available in the market.
Gene Therapy as a Driver of Structural Change
In 2026, gene therapy has made the transition from scientific promise to clinical and economic reality. New approvals, growing investment, and an expanding range of indications demonstrate that cell and gene therapy have become an established part of clinical and commercial development. The growing number of approved ATMPs is not only changing therapeutic care. It is also forcing companies to rethink their structures, processes, and capabilities. Companies that actively shape this transformation and invest early in specialized expertise can secure a decisive advantage. According to current forecasts, the market will continue to develop dynamically over the coming years.
As a specialized Recruitment Agency for the Life Science industry, Aristo Group supports companies and professionals in identifying and attracting precisely the talent that makes a difference in gene therapy.
FAQ: Frequently Asked Questions About Gene Therapy 2026
1. What Is Gene Therapy in Simple Terms?
Gene therapy is a medical approach in which genetic material is used therapeutically to treat diseases at their source — rather than just alleviating symptoms.
2. What Are ATMPs?
ATMPs (Advanced Therapy Medicinal Products) are innovative medicines based on genes, cells, or tissues. In the EU, ATMPs are scientifically evaluated by the Committee for Advanced Therapies (CAT) of the EMA—the final market authorization is granted by the European Commission.
3. Which Diseases Can Be Treated With Gene Therapy?
Currently, gene therapies are mainly used to treat rare genetic diseases, certain types of cancer (e.g. blood cancer with CAR-T), neurological disorders such as Huntington’s, and eye diseases.
4. How Many ATMPs Are Approved in the EU?
The number of approved ATMPs is continuously growing. As of April 2026, there are 22 ATMPs approved in the EU – this year saw the addition of Waskyra and Adstiladrin among others. The EMA regularly publishes updated lists of all approved ATMPs.
5. How High Is Investment in Cell and Gene Therapy?
Global investment in cell and gene therapy reached around 12 billion US dollars in 2025 – a 30 percent increase compared to 2023. Forecasts suggest that this trend will continue into 2026.
6. Which Authority Is Responsible for Gene Therapies in Germany?
In Germany, the Paul-Ehrlich-Institut (PEI) is the national expert authority for ATMPs – for centrally approved products, the European Commission grants final market authorization following scientific assessment by the EMA. In Austria, the AGES Medical Market Supervision is responsible, while in Switzerland, it is Swissmedic – according to Swiss law.
7. What Career Opportunities Are Available in Gene Therapy?
There is particularly high demand for professionals in Process Development, GMP Manufacturing, Regulatory Affairs, Quality Assurance, and Supply Chain Management with experience in regulated life science environments.
