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Alpha1 Germany eV.
Our first 2025 edition is published digitally – modern, fresh, and with a clear new layout. Enjoy comfortable reading on your computer, tablet, or smartphone with a practical search function, intuitive navigation, and resource-efficient design – available now as a flipbook for everyone. Feel free to form your own opinion.
Important information for Alpha-1 Antitrypsin Deficiency patients: Access to gene therapy has been improved!
The 2024 annual report of the Federal Office for Social Security (BAS), published in August 2025, contains a clear and guiding message for people with rare diseases such as alpha-1 antitrypsin deficiency: If gene therapy is approved, reimbursable and medically indicated, health insurance companies must not block it for cost reasons.
The Federal Office for Social Security (BAS) emphasizes that gene therapies, within the framework of the statutory orphan drug privilege, should be considered superior to less expensive established therapies. Health insurance companies may not use the argument of cost-effectiveness to deny eligibility for gene therapy.
Why is this so important – also for Alpha-1 patients?
Alpha-1 antitrypsin deficiency is classified as a rare disease – meaning this regulation directly applies to us. Even though gene therapies are not yet widely used, the BAS's position provides an important legal basis and political direction, as it strengthens patients' rights to access innovative therapies.
If gene therapies for Alpha-1 are developed and approved, you and your doctors can invoke this position to pursue claims.
New hope for Alpha-1 patients: Progress with WAVE RNA therapy
Let's stay with the new therapies that are under development: As described from page 53 in the Alpha-1 Journal, there are many approaches that have made it into preclinical research and the first phases of studies.
Due to current events, we would like to discuss WAVE today:
WAVE Life Science has announced the first, very encouraging, results from the Phase 1 and Phase 2 clinical trials announced below. as a press release dated September 3, 2025 and very detailed published as part of an investor conference. According to the report, an intervention at the RNA level*, administered via subcutaneous injections, has successfully stimulated the production of healthy M-AATs and reduced the production of the clumping-prone Z-AAT in patients with ZZ. The aim is to improve lung protection and clear the liver of deposits. No concerning side effects have been observed to date. In one participant who simultaneously developed an infection unrelated to the trial medication, it was demonstrated that the release of healthy M-AATs increased during the inflammatory phase, as expected and anticipated.
Difference between DNA and RNA
DNA is the stable archive of genetic information, in which all the blueprints for the body are stored, while RNA is a short-lived working copy of this information that the cell uses to actually implement the blueprints.
Below we provide you with the following additional information that has been made available to us:
And speaking of Aachen…
Heartfelt Spotlight: Dr. Malin Fromme receives prestigious EKFS Memorial Scholarship
Many of you already know her: We are delighted to introduce you to Dr. Malin Fromme, a young scientist whose enthusiasm and commitment are not only impressive, but have now also been recognized with a significant award: Dr. Fromme from the Department of Internal Medicine III at University Hospital RWTH Aachen received the prestigious Else Kröner Memorial Scholarship in 2025. Congratulations!
We have often experienced firsthand Dr. Fromme's passionate and dedicated research into liver diseases, particularly alpha-1 antitrypsin deficiency, and have had the pleasure of encountering her friendly and competent manner. Another of her research projects focuses on the endoplasmic reticulum (ER) – that delicate cell structure often overlooked but essential for liver health.
The Else Kröner Memorial Fellowships They support outstanding young physicians: They receive two years of financial support to dedicate themselves fully to their research. And for Dr. Fromme, that means: time, resources – and above all, space for creative, in-depth research.
With the scholarship, she will be able to examine the collected AATM patient samples and the inducible pluripotent stem cells (iPSCs) generated from them using state-of-the-art methods – including deep visual proteomics, single-cell proteomics (SCP) and single-nucleus RNA sequencing (snRNAseq).
The goal is to better understand the complex processes of this liver disease, to predict its course more accurately, and to identify predictive biomarkers – thereby offering new hope not only to children but also to adults. Furthermore, their findings could be applied to other liver diseases.
We hope that Dr. Fromme's work will soon bear fruit – be it through new diagnostic tools or therapeutics. And perhaps, who knows, this will mark the beginning of a line of research that could open up new perspectives for many people with liver diseases in the long term.
i-COPD: Better managing everyday life through training
Chronic lung diseases like COPD accompany sufferers throughout their lives. Specialized training helps to better manage the disease. A new video series, produced in cooperation with the German Center for Lung Research, provides helpful information for patients and anyone interested.
And when you talk about COPD, Alpha-1 Germany is never far away. The first part of the webinar explains Alpha-1 antitrypsin deficiency (at 5:26 min) and refers to Alpha1 Germany e. V. (7:43 min). Thanks to the MHH team for the excellent videos!
Upcoming events
And as always, we end the newsletter with our upcoming dates and something to try out:
After we had the opportunity to meet Kathrin Skoupil at a lecture on mindfulness and resilience as part of an AlphaCare (Grifols) event in Mainz, We have linked this topic here for you to try out.
At the bottom of the page you will find an exercise for breath observation.
Autumn is approaching, make the most of the beautiful days outdoors, and remember to check your vaccination status!
Your Alpha1 Germany Team
