Gene transfer with the help of adeno-associated viral vectors (AAV) is one of the most promising technologies for the development of novel cell and gene therapies for the treatment of genetic diseases. They can be used as a means of transport both within the body and outside, to introduce genetic material into specific target cells and to correct genetic defects or replace missing proteins. The potential range of applications covers a broad spectrum - from neurological, oncologic, and autoimmune diseases to monogenetic defects. Compared to other viral vectors, AAV are characterized by a high specificity and an improved safety profile.

The first AAV meeting in Leipzig focused on research and development in the field of AAV-based technologies and their transition into clinical applications. More than 160 international guests from science and industry met to discuss current research projects and clinical successes, as well as analysis and production processes for AAV-based therapies.

Scientists of international renown provided insights and highlighted the prospects for the application of AAV-based technologies for in vivo gene therapies (Prof. Dr. Hildegard Büning, Medical University Hannover, Germany), in precision medicine for the treatment of deafness (Dr. Kathrin Kusch, University Medical Center Göttingen, Germany), as well as in the field of oncology (Dr. William Nyberg, Karolinska Institutet, Stockholm, Sweden). Various contributions by industrial companies presented challenges in the production and development of AAV-based drugs and approaches to their resolution.

Fraunhofer IZI, which hosted and organized the conference, also contributed its specific expertise in the process development and GMP production of cell and gene therapeutics for clinical applications.

At present, one of the most modern GMP clean room facilities for the production of vector-based therapeutics is under construction at Fraunhofer IZI. “There is tremendous interest on the part of both science and industry to ensure more comprehensive clinical translation of AAV therapies over the coming years. Fraunhofer IZI wants to build bridges and use its regulatory, technical, and subject-specific expertise to support researchers (as well as companies) en route from the lab to clinical applications,“ explained Dipl.-Biol. Thomas Schmid, head of the GMP Biopharmaceuticals department, who initiated the conference. In view of the success of the first meeting, the second AAV Meeting in Leipzig is now scheduled to take place in spring 2027.

All the information regarding the event is available at www.AAV-Meeting.com.