Session 8: Clinical translation of gene and cell therapies

Chairs: Stefan Kochanek, Uniklinik Ulm; Ulrike Köhl, Fraunhofer IZI, Leipzig

13:00-13:15

Stefan Kochanek, Uniklinik Ulm, Ulrike Köhl, Fraunhofer IZI, Leipzig

Clinical translation of gene and cell therapies – current status in a nutshell

INV23 13:15-13:45

Frank Staal, Leiden University ⓘ

Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

INV24 13:45-14:15

Thomas Thum, Hannover Medical School ⓘ

(Pre)clinical development of noncoding RNA based therapeutics

INV25 14:15-14:45

Fatima Bosch, Universitat Autònoma de Barcelona ⓘ

Gene therapy for the Mature Onset Diabetes of the Young Type 3 (MODY3)

OR12 14:45-15:00

Tobias Bexte, Goethe University Frankfurt

High antileukemic efficiency of CD19-CAR NK cells engineered with Sleeping Beauty transposon vectors

OR13 15:00-15:15

Karl Petri, Massachusetts General Hospital, Harvard Medical School, Boston

Global-scale CRISPR gene editor specificity profiling by ONE-seq identifies population-specific, variant off-target effects.

OR14 15:15-15:30

Lea-Isabell Schwarze University Medical Center Hamburg-Eppendorf

CCR5-edited CAR T cells for the treatment of HIV-positive cancer patients