Encompassing EATRIS vision and mission “to make translation of scientific discoveries into medical products more effective to improve human health and quality of life”, the course aimed to provide a deeper understanding of the gene therapy development process, from basic research to clinical applications.
The Haapaniemi lab aims to translate their CRISPR-Cas9-based T cell editing platform work to the clinic to improve therapies for patients with rare immune diseases. The week featured interactive and high-quality talks and participating in the course broadened Frida’s perspective on gene therapy development while gaining valuable knowledge about the challenges and bottlenecks in the field of gene therapy development process development, scaling up to GMP-compliant manufacturing, and navigating regulatory aspects.
Frida’s highlights
A standout moment was speaking with an EMA representative about her project in the Haapaniemi lab, gaining valuable clarifications into the regulatory landscape relevant for their project. She particularly appreciated the opportunity to discuss with the speakers between sessions, which allowed for deeper, targeted conversations. Furthermore, the connections that were made have the potential to turn into future collaborations, which could be valuable for our ongoing and future research endeavours.
Visiting UCB Pharma, where the delegates not only attended insightful talks but also toured the newly constructed facility, designed to support the manufacturing of future gene therapies offered a concrete insight into the practical application of concepts related to analytics and manufacturing of gene therapies.
Connecting with the other participants, most of whom were PhD students at various stages working in the gene therapy space was very rewarding. The week began with participant presentations, sparking engaging discussions, and the group dynamic continued to grow through inspiring conversations during the day and social gatherings with dinners and even karaoke in the evenings. The sense of camaraderie and shared passion for this field made the week truly memorable!
Frida’s attendance to the workshop was at a reduced fee due to EATRIS membership and was funded by the Haapaniemi group.
About EATRIS
EATRIS is the European Infrastructure for Translational Medicine. It’s a non-profit organisation that brings together resources and services for research communities to translate scientific discoveries into benefits for patients. Users are given access to a vast array of expertise and facilities from over 150 top-tier academic centres across Europe. EATRIS focuses on improving and optimising preclinical and early clinical development of drugs, vaccines and diagnostics, and overcoming barriers to health innovation. Find out more about EATRIS here: www.eatris.eu