11–14 September 2018

Congress Center Basel

Genome editing

Thursday 13 September 2018

10.00 – 16.45

Innovation Forums

Genome editing (CRISPR/Cas9) in drug discovery

Genome editing technologies allow engineering parts of the genome by removing, adding or altering sections of the DNA Sequence. Recent discovery of the CRISPR/Cas9 system as a technology for genome editing, enables the simplest, quickest and most versatile way of genome editing. CRISPR/Cas9 technology is quickly developing and has already found many applications as a drug discovery tool and even as a potential new therapeutic agent in cellular and gene therapy. This forum focuses on relevant applications of CRISPR/Cas9 for drug discovery such as identification of novel targets through genetic screens, generation of relevant genetic disease models (human cells or animal models) for validation of drug targets and for mechanistic and safety studies. Importantly, current standard practices and perspectives of genome editing in drug discovery will be presented. Furthermore, the forum will show novel applications and discuss future challenges and potential pitfalls of the technology.

Filip Roudnicky (Roche, Basel, Switzerland)
Claas Aiko Meyer (Roche, Basel, Switzerland)

Introduction by the chairs
Claas Aiko Meyer (Roche, Basel, Switzerland)
Filip Roudnicky (Roche, Basel, Switzerland)

Therapeutic genome editing in clinically relevant human cells
Toni Cathomen (University of Freiburg, Germany)

Unraveling biology and identifying targets with functional genomics approaches supported by LentiArray CRISPR library screens
J. Chesnut
Research and Development, Thermo Fisher Scientific, Carlsbad, CA, United States

Creation and correction of human disease alleles in iPSCs through microhomology-mediated end joining
K. Woltjen
CiRA, Kyoto University, Kyoto, Japan

Genome editing tools for iPSC-based disease modeling
W. Hendriks
Neurology, Massachusetts General Hospital/Harvard Medical School, Charlestown, MA, United States

Correction of autosomal recessive disorders via CRISPR-associated base editors in adult animals
Gerald Schwank (ETH Zurich, Switzerland)

CRISPR platform development for drug target discovery and validation
Benjamin Haley (Genentech, San Francisco, United States)

CRISPR screening directly in vivo
Randall Platt (ETH Zurich, Switzerland)

Custom CRISPR screens for drug target discovery in immune cells
F. Wermeling
Karolinska Institutet, Stockholm, Sweden

Editing fate changing marks
Stefan Stricker (Helmholtz Center, Munich, Germany)

Cellular indel profiles and dynamics induced by different CRISPR/Cas9 delivery formats: Applying in vitro methodologies ex vivo
Eric Paul Bennett (University of Copenhagen, Denmark)

Editing of neurodegenerative disease mutations with the transient KamiCas9, (CRISPR/Cas9) system
Nicole Deglon (Lausanne University Hospital (CHUV), Switzerland)

Shanghai 1

Main sponsor(s)