Periodic Reporting for period 1 - AAVolution (Next-generation AAV vectors for liver-directed gene therapy)
Período documentado: 2022-10-01 hasta 2023-09-30
Overall, the AAVolution team is focused on making liver gene therapy better and accessible to more people, potentially changing lives and opening up new possibilities in medicine.
In addition, regarding the studies on AAV capsids (the carrier of therapeutic genes) (WP2), we created diverse libraries of AAV capsids to find the best ones for effective treatment. We used advanced techniques, including DNA shuffling and rational design, resulting in over 3000 variants. These variants underwent rigorous checks to ensure their quality.
In addressing pre-existing immune response challenges (WP3), we also generated important results. Through innovative approaches, we discovered 48 new Ide molecules, which hold promise in reducing immune responses to the therapy, and developed a machine learning flexible pipeline for designing more Ide. This is crucial for reducing immune responses against AAV therapy.
Overall, the AAVolution team is working to improve liver gene therapy better by using more effective gene-editing tools, exploring ways to make the therapy last longer, and creating diverse libraries of carriers for therapeutic genes. Additionally, the project is also addressing challenges related to the body's immune response to the therapy. These advancements bring hope for more effective and accessible gene therapies in the future.
In the strategic planning and regulatory engagement aspects, we submitted a comprehensive Cell and Gene Therapy (CGT) Strategic Plan, outlining objectives and collaborations with other projects. We also actively engaged with regulatory authorities through participation in an EMA webinar and maintained continuous interaction with private investors, showcasing their project in the EIC CGT Symposium.