[SCAN] Functional genomics and genetic engineering for improved manufacturing of viral vectors for gene therapy

Seminar

Title: Functional genomics and genetic engineering for improved manufacturing of viral vectors for gene therapy

Speaker: Ana Filipa Rodrigues

Affiliation: Cell Line Development & Molecular Biotechnology Lab – ACT Unit

Abstract:

Gene Therapy is considered one of the revolutions of the 21st century medicine offering therapeutic solutions to previously unmet medical needs including some infectious diseases, cancer and monogenic conditions. In 2013, the European Medicines Agency approved the first Gene Therapy product in the European market, followed by the United States of America Food and Drug Administration in late 2015. Viral vectors, account for 70% of Gene Therapy vehicles, due to their ability to efficiently deliver the therapeutic genes into the target cells. With the expectable growth of Gene Therapy in the next decade and increased pressure for clinical-to-market transition, improved means for the production of these vectors are needed.

Genetic manipulation of producer cells offers real potential to improve the manufacturing of viral vectors, including increase of production yields, better quality of the viral preparations and the possibility to design relevant cell phenotypes for industrial large-scale production. In the past decade we and other have used large-scale functional genomics tools to identify cellular pathways recruited in recombinant viral vector production and the hallmarks underlying high producing phenotypes. Now, leveraged by the most recent possibilities offered by advanced recombinant DNA technologies and genome editing, this knowledge is being explored for tailored design of new and better cell lines for the production of viral vectors for Gene Therapy. These efforts will ultimately help to deliver Gene Therapy viral vectors to those who need them in a more efficient and cost-effective way.