Liebe Leserinnen und Leser,&nbsp;

28. Februar 2026

Tag der seltenen Erkrankungen 2026

Am 28. Februar, dem Tag der seltenen Erkrankungen, lädt Lünen zum Austausch über seltene rheumatische Krankheiten ein. Es erwarten Sie Vorträge von Mediziner:innen, Vorstellung von Selbsthilfe-Angeboten und die Möglichkeit, sich zu vernetzen – denn gemeinsam ist man stärker als allein.

Wann?

Samstag, 28. Februar 2026

11:00 Uhr - 16:00 Uhr

Wo?

Ringhotel am Stadtpark

Kurt-Schumacher-Straße 43, 44532 Lünen

Weitere Informationen

2026

11.-13. Mär

NK & ILC Symposium 2026 | Freiburg

Zur Webseite [ENG]

2026

17. Apr

10. Rare Disease Symposium der Eva Luise und Horst Köhler Stiftung | Berlin

Zur Webseite

2026

02. - 03. Jun

SaxoCell Konferenz | Dresden

2026

09. Jun

GeneNovate Investor's Day | Berlin

2026

25. Jun

GeneNovate Final Event | München

Zur Webseite

2026

08.-11. Jul

ISSCR Annual Meeting | Montréal, Canada

2026

09.-11. Sep

German Stem Cell Network Conference (GSCN) | Berlin

Zur Webseite [ENG]

2026

27. - 30. Okt

European Society of Gene and Cell Therapy (ESGCT) Annual Congress | Hamburg

Zur Webseite [ENG]

Dear Readers,

Together with the GCT community, we look back on an eventful year 2025. A year marked by many achievements and milestones – always aware that there is still much work ahead to further improve access to GCT in Germany.

In this issue of the newsletter, you will learn about new content in the GCT Media Center, the further development and nationwide rollout of our entrepreneurship program GeneNovate, as well as a survey on information needs for the future GCT patient website. As usual, you will also find updates on regulatory topics, film, podcast and reading recommendations, further developments in Germany and Europe, and information on upcoming events.

As the year draws to a close, we wish you much enjoyment in reading and sharing this newsletter. Enjoy restful holidays, an inspiring time with your families and friends – and please stay connected with us in the year ahead.

All the best,
Elke Luger and the team of the National Network Office GCT Germany

Foto ©: NWB

On our own behalf

GCT Germany 2025 — A Year Full of Progress

2025 was an exciting year full of milestones for the GCT Germany National Network Office. Since the launch of the GCT Atlas in July, seven layers with a total of 555 datasets have already gone online—especially the layer of GMP manufacturing facilities, which was eagerly anticipated.
Our GeneNovate program celebrated a successful completion in summer 2025: 180 participants took part, and in 2026 the program will continue across nine regions of Germany.
There was progress on patient involvement as well: Nine patient representatives are actively helping to shape the National Strategy, and the inclusion of patients and stakeholders has been established as a fixed criterion in the funding programs. The strategy website went live in the summer and is continuously being expanded with relevant content.

Thank you for your support and cooperation, and we look forward to 2026!

Click here to visit the website of the GCT Germany National Network Office

Latest lectures in the GCT Media Center

The GCT Media Center is growing and now includes numerous recordings from the lecture series on gene and cell therapies at the University Hospital RWTH Aachen, initiated by Prof. Hannes Klump (Director of the Institute for Transfusion Medicine and Cell Therapeutics, RWTH Aachen).

The lecture series provides in-depth insights into GCT – from the cell types used, gene transfer, and gene editing methods, to clinical development and application, as well as commercialization and ethical considerations. The lectures are in English.

Go to the Media library

Photo: © getty images - free license

EuroGCT Best Practice Workshop in Berlin

Two days full of inspiration and knowledge exchange took place at this year’s Sharing Best Practice Workshop of the European Consortium for Communicating Gene and Cell Therapy Information (EuroGCT) in Berlin. EuroGCT unites 47 partner organizations across Europe with the goal of providing reliable and easily accessible information on the application of gene and cell therapies.
Key topics discussed at the workshop included effective communication with different target groups, the impact of AI on patient information searches, the role of trustworthy information sources in the era of AI-generated content, and more. This year’s host was the German Stem Cell Network.

Photo: © GSCN | Stefanie Mahler

GeneNovate Enters Its Third Round

The GeneNovate entrepreneurship series has entered its third round and will take place in 2025/2026 across nine regions with a total of 195 participants. The program kicks off with December events at each location, where participants meet their mentors for the first time, discuss their concepts, and form teams. The highlight will be the Final Event on June 25, 2026, in Munich, bringing together the entire GeneNovate community with investors, business angels, and more.

A Strong Alumni Network Fosters Exchange and Networking

Many participants from previous GeneNovate cohorts remain connected to the program. To provide them with a platform for exchange and networking, the GeneNovate Alumni Network was launched this year. Over 70 alumni are already active in the associated LinkedIn group. Planned activities include spotlight posts highlighting exciting developments from alumni startups, as well as digital and in-person meetings to strengthen the network. Those not yet involved can join the LinkedIn group at any time and help shape the community.

Photo: © NWB | Arne Sattler

GeneNovate Idea Validation Startup-Coachings

The four winning teams from the 2nd GeneNovate round (2024/25) received individual coaching sessions. The pilot workshop “Idea Validation” took place this Fall. Idea Validation involves testing a product concept early to determine market relevance before significant time and money are invested. The goal is to confirm or refute assumptions through feedback, surveys, or small experiments.

The pilot workshop builds on the GeneNovate Education series and focuses on practical idea validation to further develop innovative concepts in GCT. Winning teams work on their projects with support from individual coaching sessions (e.g. patent attorneys) and experts (e.g. the Regulatory Support Unit).

Photo: © NWB | Celina Mützel

GeneNovate Investors' Day 2026 - Apply by January 16

Following the successful launch this year, we invite you to the 2nd GeneNovate Investors' Day in Berlin on June 9, 2026. Applications are open until January 16, 2026. International startups in the field of gene- and cell-based innovations can apply – this year with a focus on AI-driven approaches in research, development, and production. In 2025, 16 companies from Germany, France, Switzerland, and the UK presented their innovations. Take this opportunity to showcase your innovation to investors, receive valuable feedback, and network with industry experts.

Go to the GeneNovate Investors' Day website.

Photo: © BIH | Ulf Büschleb

GCT Atlas: Seven Layers with 555 Entries Now Available

This year, we have already launched seven layers that strengthen networking within the German GCT ecosystem:

• Patient and self-help organizations
• University hospitals, specialized centers, and research groups
• GMP manufacturing facilities
• Biotech startups
• GeneNovate sites
• BioRegions of Germany
• Members of the Round Table and actors of the National Strategy GCT

The GCT Atlas is a dynamic source of information and thrives on an active community. Would you like to be visible on the GCT Atlas? Register now.

Visit the GCT Atlas and register

Graphic: © NWB

Survey on Gene- and Cell-Based Therapies – Your Perspective Counts!

A new subpage is being created on the National Strategy GCT website to provide patients, caregivers, and patient organizations with comprehensive and understandable information about gene- and cell-based therapies. We would appreciate to hear from you: Which topics are important? What should be considered in the website design?
A survey has been developed for this purpose. It takes approximately 10 minutes and is open until February 6, 2026. Take the survey

We appreciate your participation and sharing the call within your networks!

Graphic: © BIH | Quest Center

News from the strategy

Innovations from Germany: “We need to better leverage excellent knowledge"

In an interview with Tagesspiegel, Prof. Christopher Baum, Chair of the BIH Directorate and Head of the Translational Research Area at Charité - Universitätsmedizin Berlin, spoke during the “Future Medicine Innovation Days” about Germany’s future as a research and innovation hub. He highlighted the role of research institutions and companies in a globalized world and emphasized academic freedom as the foundation for innovation. Prof. Baum cited gene and cell therapy as an example of current challenges and opportunities.

Go to the interview

Graphic: © Getty Images

GCT Expert Dialogue: Political Visibility and Communication

To raise awareness among policymakers about the importance of gene- and cell-based therapies, the first expert dialogue within the National GCT Strategy took place on November 24, 2025. Participants included SPD parliamentarians Matthias Mieves (member of the Health Committee) and Holger Mann (member of the Research Committee) and their staff. Strategy working groups were represented by Dr. Martin Danner (BAG Self-Help), Dr. Thekla Kurz (Miltenyi Biotec), and Prof. Francis Ayuk (UKE). They discussed opportunities, challenges, and necessary political frameworks for GCT. Further expert dialogues are planned for 2026.

Photo: © BIH | Stefanie Schneidmadl

International Exchange on ATMPs in Gothenburg, Sweden

Colleagues from the Berlin Institute of Health (BIH) attended the ATMP Sweden 2025 conference in Gothenburg on November 27–28. Topics covered included clinical trial frameworks, entrepreneurship in ATMPs, and the high medical need for patients with rare diseases.

Christian Gallus, Project Manager of the National GCT Strategy at BIH, shared Germany’s strategic approach to improving the overall GCT ecosystem. A pan-European perspective proved invaluable: cross-border collaboration accelerates knowledge transfer, constructive exchange, and the creation of joint initiatives.

New Tandems and Locations for the GCT Personnel Support Program

The national Translational Tandem Program for gene- and cell-based therapies (nTTP-GCT) enters its next round: 17 teams applied, six tandems were selected, and will receive protected research time, targeted training, and access to a growing network starting in 2026. New locations in Heidelberg, Tübingen, Greifswald, Hannover, and Berlin expand the community nationwide. In total, 30 talents from eight cities will strengthen translational research in Germany.

Go to the Website

GCT Project Funding: Call-2025 Projects Gain Momentum

This autumn, SPARK-BIH celebrated its 10th anniversary, welcoming SPARK teams from around the world, including recipients of the national GCT project funding, for exchange and networking. Parallelly, Track-2 projects for GCT products and associated diagnostics were added under the Call 2025. These projects work on their milestone agreements according to the SPARK method. Track-1 projects in early development began creating strategic milestones, which also define early medical and regulatory requirements. A continuously updated booklet with project descriptions can be viewed here.

Photo: © BIH | Thomas Rafalzyk

For Patients

vfa Short Report on Drug Expenditures – Only 0.7% for ATMPs

“Germany has high availability of approved ATMPs, yet their real-world application remains limited. In 2024, ATMPs accounted for only 0.7% of drug expenditures in both outpatient and inpatient care, demonstrating that they do not disproportionately burden the statutory health insurance system.”

Read the article here

Film Recommendation: "Gene Horizons" – Understanding Gene Therapies

In recent decades, targeted DNA modification for disease treatment has gone from science fiction to life-saving reality. How do gene therapies work, and what can they treat? Learn more in the new short film Gene Horizons, funded by the EU Horizon 2020 program. Featuring five leading researchers and clinicians, with stop-motion animations by Cameron Duguid, the 18-minute film introduces gene therapy to a broad audience and biology students aged 16+. A multilingual toolkit with further educational materials is also available.

Watch the video [Audio in ENG, German subtitles available]

Graphic: © EuroGCT | Cameron Duguid

Podcast “Wirkstoffradio”: Deep Insights into Immune Cells

Priv.-Doz. Roland Schelker, Head of the T-Cell Fitness” Translational Group at the Leibniz Institute for Immunotherapy (LIT) in Regensburg, guides listeners through the different types of immune cells and their functions. Focus is on T cells and the genetic mechanisms underlying their development from T stem cells to memory and effector T cells. You will learn which T-cell characteristics are most relevant for developing T-cell immunotherapies.

Go to the podcast

Photo: © LIT

Transfer of organells: New Approaches for Cancer Immunotherapy

In a recent Nature Reviews Immunology article, Prof. Luca Gattinoni, Head of „Functional Immune Cell Modulation department at Leibniz-Institut for Immuntherapy (LIT), and his team describe how organelles regulate T-cell function and how this knowledge can enhance cancer immunotherapy

The article provides a comprehensive overview of how organelles – including mitochondria, lysosomes, endoplasmic reticulum, and peroxisomes – drive the metabolism, signaling, and persistence of T cells during the antitumor response. Gattinoni and his team had previously reported in Cell that bone marrow stromal cells (BMSCs) can transfer their mitochondria to CD8+ T cells. This process can be used to improve the metabolism of T cells, reduce their exhaustion, and strengthen antitumor immunity.

Go to the article

Photo: © LIT

Regulatory Radar

The Regulatory Support Unit (RSU) of the National GCT Strategy reports on current regulatory developments.

From “Baby KJ” to the New Framework for Personalized Gene Therapies

The case „Baby KJ“ (Musunuru et al., 2025) has become a landmark example of how personalized gene-editing approaches can be evaluated within new, adaptive regulatory frameworks for ultra-rare diseases. As part of this evolving landscape, the U.S. regulatory authority FDA has introduced a new approach, the “New Plausible Mechanism Pathway” (Prasad und Makary, 2025 ), designed to accelerate the development of personalized gene therapies for severe, often ultra-rare conditions.

The approach is based on five scientific criteria: 1) a clearly defined disease-causing mutation, 2) a therapy that directly targets this defect, 3) robust natural history data, 4) evidence that the biological target has been effectively addressed or edited, and 5) early signs of clinical benefit.

For researchers and treating physicians, this new framework highlights the value of precise molecular diagnosis, well-characterized natural history data, and flexible translational platforms. The RSU monitors these regulatory developments to provide optimal guidance, preparation, and regulatory strategy for the teams we support.

EMA Recommends EU Approval for “Waskyra” by Fondazione Telethon

The Committee for Medicinal Products for Human Use (CHMP) of the EMA has issued a positive opinion on the approval of Waskyra (etuvetidogene autotemcel) in the EU. Waskyra, developed by Fondazione Telethon , is an ex vivo lentivirus-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening genetic disorder. Fondazione Telethon is making history as the first non-profit organization to successfully complete the full translation process from academic research to regulatory approval. Waskyra was supported under the EMA pilot program, which provides enhanced scientific guidance to academic and non-profit developers of ATMPs with high medical need. In addition, Waskyra received an “Orphan Designation” from the EMA – a program that promotes the development of therapies for rare diseases through various incentives.

For more information on the requirements for an Orphan Designation, the benefits, and support during the application process or other regulatory questions regarding your project, please contact the Regulatory Support Unit (RSU): reg-support@bih-charite.de

News from Our Partners

A Strong Stem Cell Network – Soon for All of Europe?

Since 2013, the German Stem Cell Network (GSCN) has demonstrated how science, policy, and the public can be effectively connected through education, talent promotion, and international collaboration. With the steady increase in clinical applications of stem cells, the call for a strong pan-European network is growing. This publication highlights why now is the right time to pool these forces – and what opportunities this creates for research, society, and medicine.

Read the publication

SaxoCell Strengthens GMP and ATMP Expertise: Successful Workshop at Fraunhofer IZI

Leipzig, October 28, 2025 – The SaxoCell workshop “Basics in GMP and ATMP” took place with great success at the Fraunhofer Institute for Cell Therapy and Immunology (Fraunhofer IZI). In a three-hour interactive session, participants gained comprehensive insights into the fundamentals of Good Manufacturing Practice (GMP) and its significance for the production of medicines for advanced therapies (ATMPs).
Led by Mirko Müller (Fraunhofer IZI), key topics of GMP regulation were explained clearly and practically, including historical development, basic principles, international frameworks, and core content of EU GMP guidelines. The workshop concluded with a tour of Fraunhofer IZI’s GMP facility, offering participants rare insights into real manufacturing and quality environments.

Through formats like this, SaxoCell supports the expansion of expertise and infrastructure for innovative gene and cell therapies (GCT) in Saxony, creating a vital foundation for their successful translation into clinical applications.

Photo: © SaxoCell

GCT Germany

Primary Tissue Pipeline (PTP): National Infrastructure and Expertise for Human-Relevant Research

The Primary Tissue Pipeline (PTP) at Charité – Universitätsmedizin Berlin provides researchers across Germany with a central point of access to human tissues from clinical routine. The offering supports the planning and implementation of projects based on human-relevant models – for preclinical testing, new therapy development, or validation of cell-based methods. PTP offers:

• Standardized sample collection and quality control
• Advice on ethical, legal, and organizational questions
• Coordination and logistics for tissue provision
• Interfaces to clinical partners and research teams

This promotes reproducible, ethically responsible, and translationally relevant research. The offer is open to all researchers across Germany and is funded by the Volkswagen Foundation.

Visit the Primary Tissue Pipeline website

Photo: © PTP | A. Kühl

Worth Reading

Global Progress of Therapies Based on Human Pluripotent Stem Cells (hPSC)

Kirkeby et al. (2025) provide a comprehensive overview of the global progress of therapies based on human pluripotent stem cells (hPSC). They report on 115 clinical trials involving 83 different hPSC-based products, primarily targeting eye diseases such as age-related macular degeneration (AMD), neurological disorders including Parkinson’s disease, and various cancers. Over 1,200 patients have been treated with more than 10¹¹ cells, with no major safety concerns reported. Advances in differentiation protocols, GMP-compliant production, and regulatory frameworks have driven this development, while long-term efficacy, immune tolerance, cost, and continuous monitoring remain key challenges.

Read the publication

Gene Technology Report – “Focus: Research Barriers and Opportunities in Germany”

The recently published brochure by AG Gentechnologie, “Focus: Research Barriers and Opportunities in Germany,” addresses current challenges in all areas of genetic engineering, highlights the potential of genetic research, and provides recommendations to policymakers. Overregulation and bureaucratization, lack of uniform regulations, and difficulties in translating scientific findings pose hurdles for research with human embryonic stem cells, laboratory research and gene therapy studies, the development of alternative methods such as organoid research, and studies with genetic data. At the same time, genetic technologies can contribute to solving major societal challenges, including long-term health, climate change, nutrition, and animal welfare.

Read the brochure here

Your Contribution to the GCT-Newsletter

A nationwide newsletter is as relevant and diverse as the community contributing to it.
We are happy to report on initiatives from all over Germany and share your news, activities, and events. Feel free to contact us at:

gct-news@bih-charite.de

Save the Date 2026

January 17, 2026

8th Cancer Day Ruhr

In January 2026, the West German Tumor Center Network will host the 8th Cancer Day Ruhr, where experts will provide information on current developments in cancer research through presentations, workshops, and activity programs. Self-help groups and regional organizations will present their work and will be available for conversation and questions. People affected by cancer, their relatives, and anyone interested are warmly invited to exchange ideas and learn about new insights.

When?

Saturday, January 17, 2026

10:00 a.m. - 4:00 p.m.

Where?

Essen - Lehr und Lernzenztrum am Uniklinikum Essen

Virchowstraße 163a, 45147 Essen

Further Information

February 5-6, 2026

12. National Care Conference of Skin Cancer

The National Care Conference on Skin Cancer (NVKH) brings together professional societies, institutions, and patient organizations involved in the prevention and care of skin cancer. On February 5 and 6, 2026, members, project groups, advisory board members, and other interested participants will meet in Berlin for the annual conference under the theme “Skin Cancer in All Its Facets – An Interdisciplinary Challenge.” In addition, the working groups responsible for the action fields and cross-cutting topics will meet on February 5.

When?

Thursday-Friday,
February 5-6, 2026

09:00 a.m. - 4:00 p.m.

Where?

Haus der Land- und Ernährungswirtschaft

Claire-Waldoff-Straße 7, 10117 Berlin

Further Information

February 11, 2026

InfoSereis Cancer: Breast Cancer, online

At the InfoReihe KREBS, experts from the Charité Comprehensive Cancer Center provide information and advice to patients, families, and anyone interested on selected medical-oncological, psychosocial, and psycho-oncological topics, paying special attention to the needs of people with cancer. On Wednesday, February 11, 2026, the topic will be breast cancer. The event is organized by the Psycho-Oncology Department of the Charité Comprehensive Cancer Center in cooperation with SURVIVORS HOME.

When?

Wednesday, 11 February 2026

5:00 p.m. - 7:00 p.m.

Where?

Online

Further Information

February 18–21, 2026

37th German Cancer Congress

At the German Cancer Congress 2026, experts from medicine, nursing, politics, and patient organizations will discuss the future of oncology and patient-centered care. Key topics include the opportunities and limitations of artificial intelligence, questions of equity, and the increasing diversity of those affected. The goal is to implement modern treatment methods effectively, integrate palliative care early, and provide better support for cancer survivors — with people at the center of all efforts.

When?

Wednesday–Saturday,
18–21 February 2026

09:45 a.m. - 7:00 p.m.

Where?

CityCube Berlin

Further Information

2026

Feb 19

RegMed Forum: Clinical Translation of Advanced Therapeutic Approaches | Berlin

February 28, 2026

Day of rare diseases 2026

The 28th of February is traditionally the day when rare diseases receive special attention. On this day, we would like to come together in Lünen to talk about rare rheumatic diseases. There will be presentations by medical experts, we will introduce our self-help services, and we want to build our network. Better together than alone – this also applies to people with rare rheumatic diseases.

When?

Saturday, February 28, 2026

11:00 a.m. - 4:00 p.m.

Where?

Ringhotel am Stadtpark

Kurt-Schumacher-Straße 43, 44532 Lünen

Further Information

2026

Mar 11 - 13

NK & ILC Symposium 2026 | Freiburg

2026

Apr 17

10th Rare Disease Symposium | Berlin

2026

Jun 02 - 03

SaxoCell Conference | Dresden

2026

Jun 09

GeneNovate Investor's Day | Berlin

2026

Jun 25

GeneNovate Final Event | Munich

2026

Jul 08 - 11

ISSCR Annual Meeting | Montréal, Canada

2026

Sep 09 - 11

German Stem Cell Network Conference (GSCN) | Berlin

2026

Oct 27 - 30

European Society of Gene and Cell Therapy (ESGCT) Annual Congress | Hamburg