Join us on Thursday 19th November 2020, 15:00 GMT | 16:00 CET | 10:00 EST, when Dr Paula Salmikangas and Professor Steffen Thirstrup discuss how to keep up with the Fast-Evolving World of ATMPs.
The development of novel advanced therapy medicinal products (ATMPs) is moving fast, as witnessed by the growing number of products appearing in clinical trials. Investments and ATMP developers are also increasing worldwide. Innovative technologies like gene editing have enhanced the design of products to better target the indications – for example, by allowing the move from autologous products to allogeneic, off-the-shelf products.
Advances in virus vector production systems have greatly improved the safety profiles of current gene therapy products. However, the design and engineering of ATMPs are becoming more and more complex, which brings new and possibly unknown risks and regulatory concerns. The availability of non-clinical models for safety testing is limited for these products and it may be that some risks are identified only through human exposure; this requires careful planning of the clinical studies and robust strategies for risk mitigation and long-term pharmacovigilance.
The challenges of persistency and immunogenicity for some products, such as adeno-associated viruses (AAVs), are currently hampering their long-term effectiveness and the overall clinical outcomes. Outstanding efficacy results for approved products like Kymriah®, Yescarta®, and Zolgensma® are strongly encouraging the field towards curative treatments, but the risks related to novel technologies should remain manageable and well-controlled to ensure real benefits for patients.
The key discussion points of this webinar will include:
- An overview of the current status of approved ATMPs and those in clinical trials worldwide
- Recognition of novel manufacturing technologies and their perceived risks
- Understanding the pros and cons of non-clinical studies, including risk identification and mitigation strategies
- An update on recent clinical results and new trial designs, including histology agnostic trials
If you require further information, please do not hesitate to contact us.
We are expecting this webinar to generate a lot of audience interest, so please send in your questions now to ensure the speakers have time to answer them in the Q&A: email our moderator at email@example.com. If your question is for a specific presenter, please include this in your email.
Dr. Paula Salmikangas, Director of Biopharmaceuticals and ATMPs, NDA Group
Paula is the Director of Biopharmaceuticals and ATMPs and has been at NDA since 2017. Her main areas of expertise are biological medicinal products, especially advanced therapy medicinal products and the CMC aspects of biopharmaceuticals. As a former EU regulator, Paula was actively involved in establishing the regulatory guidelines for ATMPs and applies her unique experience to provide companies with technical & strategic advice for their drug development programs.
Professor Steffen Thirstrup, Director NDA Advisory Board, NDA Group
Steffen has been with NDA since 2013 and excels at advising companies on their development strategies to meet the expectations of regulatory agencies around the world. He applies his skills to a broad range of therapeutic areas and has successfully helped numerous clients interact with regulatory agencies throughout the stages of development and during regulatory review.
Moderated by Conor Kavanagh, Journalist, and Editorial Assistant, Pharmafocus