{"id":6829,"date":"2025-09-16T12:36:26","date_gmt":"2025-09-16T10:36:26","guid":{"rendered":"https:\/\/alpha1-deutschland.org\/?p=6829"},"modified":"2025-09-23T09:35:11","modified_gmt":"2025-09-23T07:35:11","slug":"1-2025-newsletter-flippingbook","status":"publish","type":"post","link":"https:\/\/alpha1-deutschland.org\/en\/1-2025-newsletter-flippingbook","title":{"rendered":"1\/2025: Browse digitally now: Our FlippingBook \u2013 plus current news from science & research"},"content":{"rendered":"
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Alpha1 Germany eV.<\/strong><\/p>\n<\/div>\n\n\n\n

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Our first 2025 edition is published digitally \u2013 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 \u2013 available now as a flipbook for everyone. Feel free to form your own opinion.<\/a><\/p>\n<\/div>\n\n\n\n

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Important information for Alpha-1 Antitrypsin Deficiency patients: Access to gene therapy has been improved!<\/span><\/h2>\n<\/div>\n\n\n\n
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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.<\/p>\n

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.<\/p>\n

Why is this so important \u2013 also for Alpha-1 patients?<\/strong>
\nAlpha-1 antitrypsin deficiency is classified as a rare disease \u2013 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.<\/p>\n

If gene therapies for Alpha-1 are developed and approved, you and your doctors can invoke this position to pursue claims.<\/p>\n

Click here for the full report.<\/a><\/p>\n<\/div>\n\n\n\n

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New hope for Alpha-1 patients: Progress with WAVE RNA therapy<\/span><\/h2>\n

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.<\/p>\n

Due to current events, we would like to discuss WAVE today:<\/strong><\/p>\n

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<\/a> and very detailed published as part of an investor conference.<\/a> 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.<\/p>\n

Difference between DNA and RNA<\/strong>
\nDNA 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.<\/p>\n

Below we provide you with the following additional information that has been made available to us:<\/p>\n