Progress in implementing the objectives of the National Strategy

• Why this is important: Establishing a permanent organizational structure that regulates the procedures, processes, and responsibilities of the actors involved is a prerequisite for the consistent implementation and further development of the strategy.
• Current status: A proposal for a joint organizational structure (governance structure) has been developed with the German Society for Gene Therapy (DG-GT). The plan is to continue existing efficient formats for cooperation between the National Strategy GCT and the DG-GT, such as working groups, round tables, and the International Scientific Advisory Board.
• Next steps: Elaboration of the specific details and implementation steps in collaboration with the DG-GT. Coordination with the BMFTR and development of a long-term financing model.

Based on the requirements of Topic I, Objective 1: "Coordinated implementation of the measures of the National Strategy."

• Why it matters: A central overview of important elements related to gene and cell-based therapies, such as research groups, infrastructure, services, and activities creates transparency, facilitates networking, and enables targeted access to information for research, industry, and patients. This allows relevant stakeholders to access the necessary resources quickly and efficiently, creates synergies, and accelerates the development of gene and cell-based therapies.
• Current status: The GCT Atlas has already been launched as a central information platform. It shows a variety of stakeholders on an interactive map – from research institutes, clinics, and GeneNovate locations to patient and self-help organizations. Each map layer provides relevant information about the respective institutions, their areas of focus, and contact details. The matchmaking function allows the map layers to be displayed simultaneously, stacked on top of the other.
• Next steps: The next layers are in preparation. The content is regularly reviewed for quality and gradually expanded.

Based on the requirements of Topic I, Objective 3: "Strengthening national networking structures" and Topic VIII, Objective 1: “Informing society about GCT by providing reliable and target group-specific information.”

Use of existing infrastructure by start-ups

• Why this is important: The targeted use of existing infrastructure makes it easier for early stage companies to get started in GCT development. This supports the development and growth of start-ups in the field of gene and cell-based therapies and enables innovation potential to be exploited efficiently.
• Current status: A legal opinion is currently being prepared to clarify the legal conditions for the use of existing infrastructure by start-ups.
• Next steps: Based on the report, guidelines will be developed that describe how this use can be implemented in practice.

Based on the requirements of Topic III, Objective 3: "Facilitating the exploitation of the social and/or economic potential of scientific results." 

• Why this is important: Standardized contract templates for the purchase and delivery of gene and cell therapy products simplify and accelerate negotiations , subsequent implementation, and thus patient care. They provide transparency, reduce administrative efforts, and support clinics, pharmacies, industry partners, and start-ups alike.
• Current status: The working group has gathered needs and drew up initial drafts of the model contracts with the support of external legal advice.
• Next steps: The model contracts will now be further refined, taking into account additional feedback from all participating subgroups and relevant departments such as pharmacies and internal legal departments. The goal is to create a mutually agreeable, practical template for use in all relevant projects. 

Based on the requirements of Topic VII, Objective 3: “ Streamline contract design procedures”

• Why this is important: Qualified personnel are a key factor in the highly complex production of gene and cell-based therapies in accordance with Good Manufacturing Practice (GMP) guidelines.
• Current status: Collection of existing training and further education programmes. Key obstacles to recruiting and retaining personnel have been identified.
• Next steps: Integration of the programmes into the GCT Atlas and development of proposed solutions for further optimization of the working environment .

Based on the requirements of Topic V, Objective 2: “Ensuring the necessary personnel capacities and expertise for GCT manufacturing and quality control.”

• Why this is important: A method-specific registry enables the assurance and improvement of GCT, creates a better evidence-basefor new therapeutics  andenables registry-based research. Furthermore, it supports public health planning and policy by providing systematic data on the use and benefits of GCT in routine care.
• Current status: Based on the findings of the INTEGRATE-ATMP innovation fund project, the National Registry for Gene and Cell Therapies Germany e.V. was founded in June 2025. The project aims to contribute to the development of binding framework conditions for the quality-assured application of existing and future GCTs in Germany, thereby ensuring optimal care for patients.
• Next steps: Conceptualization and implementation of sustainable financing for the registry. 

Based on the requirements of Topic VII, Objective 4: “Optimization and establishment of the data landscape for the purpose of versatile usability in research and tracking of long-term GCT data.”

• Why this is important: Analyses of value creation potential – particularly in the areas of research and development, clinical translation, manufacturing, regulatory processes, training and qualification of skilled personnel, technology transfer, commercialization, and market launch – create a strong foundation for political decisions. They highlight opportunities for improvement and efficiency gains so that existing resources can be used in a targeted manner and value chains can be strengthened.
• Current status: Preparation of content, involvement of stakeholders from the the National GCT Strategy, recruiting of external experts for a scientific advisory board, structuring of work packages
• Next steps: Approval of the concept for analyzing possible value creation potential with the spokespersons of the National Strategy GCT working groups and with the advisory board. 

Based on the needs of Topics I-VIII

• Why this is important: The establishment of interdisciplinary therapy recommendation boards ensures structured, quality-assured and multidisciplinary decision-making for gene and cell therapies (GCT). The aim of the measure is to facilitate the identification of patients who can be offered gene and cell-based therapies as a treatment option. The therapy recommendation boards are intended to make treatment proposals that are interdisciplinary, expert-based and evidence-based, thereby enabling to offer the optimal personalised therapy to patients. This enhances the safety, efficiency and standardisation of GCT development and application in Germany.
• Current status: Preparation of a proposal on quality requirements for therapy recommendation boards, which is intended to be integrated into the ATMP quality assurance guideline of the G-BA. This is currently being further refined by the entire working group.
• Next steps: Discussion of the draft with the G-BA on steps for possible implementation. 

Based on the requirements of Topic VII, Objective 1: " Establish interdisciplinary therapy decision boards as the gold standard in GCT diagnostics” 

The Berlin Center for Gene and Cell Therapies is an innovative public-private joint project that combines research, development, and production of gene and cell therapies under one roof. The initiators are Bayer AG, Charité – Universitätsmedizin Berlin, and the Berlin Institute of Health at Charité. With an incubator for start-ups that can accommodate 15 to 20 companies and a GMP-certified production facility, the centre provides optimal conditions for the translation of novel therapies from the laboratory to clinical application. Funding from the State of Berlin and the Federal Ministry of Research, Technology, and Space (BMFTR) supports the expansion of the infrastructure and ensures the long-term quality and safety of the pharmaceutical products. ProBioGen AG operates the GMP production facility and ensures compliance with international quality standards, while iQ spaces, as project developer, is responsible for the construction of the building on the Bayer campus in Berlin. The completion of the building is scheduled in 2028.

• Why this is important: The early involvement of politicians and decision-makers ensures support, visibility, and funding for GCT measures that could benefit patients.
• Current status: Communication materials have been created, and contacts have been established with parliamentary committees. In addition, specific expert dialogues are being planned in which members of the Parliament (Bundestag) will engage in dialogues with experts from business, health, and research, as well as from patient organizations.
• Next steps: Conducting expert dialogues (Q4 2025), discussing strategic priorities, and securing further political support.

Based on the requirements and objectives of Topic I, Objective 2: "Strengthening political responsibility for the national future topic of GCT" and Topic VIII, Objective 2: "Supporting/advising decision-makers by strengthening interaction with politics and establishing/maintaining an open-ended humanities/social discourse."

• Why this is important: The active involvement of patients and their representatives from the very beginning ensures that projects and measures of the National Strategy for Gene and Cell-based Therapies (National Strategy GCT) are designed with the patient in mind. This enhances the quality of the developed measures and promotes trust, acceptance, and relevance of the therapies designed.
• Current status: Patients (and patient organizations) are represented in many working groups of the National Strategy GCT. One example is the intensive collaboration in a subgroup of Topic 8, "Interaction with Society." Currently, patient-centered information on gene and cell-based therapies will be compiled and published on this page soon. 

• Next steps: Further measures to integrate the patient perspective into all phases of GCT development will be prepared and implemented in order to further strengthen the patient-centered focus of the National Strategy GCT.

  The project "Patient & Stakeholder Engagement (PSE) in the National Strategy for Gene and Cell-Based Therapies" is supported by the National Network Office GCT Germany and the PSE team at the BIH QUEST Center.

Based on the requirements of Topic I, Objective 4: "Establishment and expansion of national and international networking activities," Measure 2: Patient information, and Topic VIII, Objective 1: "Informing society about GCT by providing reliable and target group-specific information," Measure 1: Establishing a central communication platform with a web presence to provide information for the various relevant target groups"

• Why this is important: International cooperation increases visibility, enables the exchange of experience, and promotes synergies in the research and development of gene- and cell-based therapies.
• Current status: Many contacts have been established with partner initiatives in France, the UK, and Scandinavia. Initial exchanges in different formats have already taken place as part of various projects of the National Strategy GCT. Here, specific starting points for cooperation have been identified, for example, in clinical trials, GMP infrastructures, or in the area of data and transparency (e.g., GCT Atlas).
• Next steps: The details of the collaborations with the individual partner initiatives is now being specified. For example, the exchange on translation and entrepreneurship is being deepened using the GeneNovate programme, work is being done with European partners on GMP production capacities, and an overview of relevant research groups is being created via the GCT Atlas. The next step includes the exchange of ideas with the partners on the specific approach and the development of joint initiatives.

Based on the requirements of Topic I, Objective 4: "Establishment and expansion of national and international networking activities" 

• Why it is important:
  Regulatory requirements relating to the early, non-clinical development stages of gene and cell therapies (GCT) are critical for later clinical application and often represent a major challenge for product developers. The complexity and rapid development in the field of GCT also requires continuous monitoring with the associated regulatory aspects. For this reason, the Regulatory Support Unit (RSU) offers academic GCT teams in Germany expert support on regulatory matters that is, confidential, and free, thereby facilitating the path to clinical application—more efficiently and significantly faster. 

• Current status:
  The RSU was established in 2024 at the Berlin Institute of Health at Charité (BIH) as part of the National Strategy for GCT. Within the first year, there was already a considerable need for regulatory advice, with numerous inquiries from all over Germany. The RSU provided support on a wide range of product-specific queries relating to regulatory strategy and in preparation of scientific consultations at the Paul Ehrlich Institute (PEI). In addition, the RSU participates in the GeneNovate programme, which was also initiated as part of the National Strategy for GCT – the first Germany-wide entrepreneurship programme in the field of gene and cell therapies. The aim of the programme is to provide scientists and medical professionals with in-depth knowledge on relevant topics in the field of entrepreneurship, to support them in the development of innovative therapies, and thus to strengthen the business location Germany. 

  The RSU also contributes to strengthening new GCT developments through its participation in international networks, such as by supporting the EU project Join4ATMP and participating in the international IRDiRC Taskforce-project to improve the treatment and diagnosis of rare diseases. 

• Next steps: Expansion of the advisory network, further increase the visibility within the GCT community, as well as the transfer of regulatory knowledge and clarification of the relevance of early interactions with regulators for the development of new and innovative therapies. 

Based on the needs of Topic IV, Objective 3: "Increasing the availability of low-threshold regulatory advice."

Status: October 6, 2025