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Wednesday, June 7 • 13:40 - 15:00
Session 4

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Dominic Hoepfner - Chemogenomics with CRISPR – Old Tricks but New Dogs

Chemogenomic profiling is a powerful and unbiased approach to elucidate pharmacological targets and the mechanism of bioactive compounds. Until recently, genome-wide, high-resolution experiments of this nature have been limited to fungal systems due to lack of mammalian genome-wide deletion collections. With the example of a novel nicotinamide phosphoribosyltransferase (NAMPT) inhibitor and a novel antiviral compound of natural origin, we demonstrate that the CRISPR/Cas9 system enables the generation of transient homo- and heterozygous deletion libraries and allows for the identification of efficacy targets and pathways mediating hypersensitivity and resistance relevant to the compound mechanism of action.

Niclas Nilsson - Enabling scientific exploration using a truly open innovation platform – Pharma collaborations with no strings attached


  • Why the Industry and Academic research mutually benefits from opening up

  • How this is done, exemplified by LEO Pharma’s concrete and unique open innovation platform

  • What opportunities can come from truly changing the way we work together

Connecting targets and compounds - the European Lead Factory experience - Kristina M. Orrling, PhD

The European Lead Factory (ELF) is a public–private partnership supported by the Innovative Medicines Initiative (IMI).It provides researchers in Europe with a unique platform for translation of innovative biology and chemistry into high-quality starting points for drug discovery. It combines high-throughput screening (HTS) infrastructure and hit follow-up capabilities with an exceptional collection of small molecules to advance research projects from both private companies and publicly funded researchers. Established in 2013, its unique collaborative model has so far resulted in >6,000 hit compounds with a defined biological activity from over 100 successfully completed HTS and hit evaluation campaigns, >145,000 novel innovative screening compounds that complement the 327,000 compounds contributed by the big pharma, >30 protein–ligand structures, two patents and two start-up biotechs. Intrinsic to its setup, ELF enables breakthroughs in areas with unmet medical and societal needs, where no individual entity would be able to create a comparable impact in such a short time.

The presentation will reveal some cross-programme trends, further illustrated by a few case studies from the target programme portfolio.

Roger Olsson, Fish and Grasshoppers in the Service of MANkind: exploring new in vivo and in vitro models

The presentation will focus on the use of alternative animal models in drug discovery, phenotypic screening—profiling in zebrafish and evaluation of blood-brain barrier (BBB) permeation and drug metabolism in the brain using an ex vivo model based on grasshoppers. Is the zebrafish model sensitive enough to distinguish between structurally similar compounds with polypharmacological profiles, and is clozapine N-oxide used as an inert ligand in chemical genetics using the DREADD technology really inert, and is it readily penetrating the BBB?



avatar for Per-Anders Enquist

Per-Anders Enquist

Senior research engineer at Department of Chemistry, Chemical Biology Consortium Sweden

avatar for Dominic Hoepfner

Dominic Hoepfner

Novartis Institutes for Biomedical Research
Dominic did his Ph.D. in the lab of Prof. Peter Philippsen at the Biozentrum in Basel, Switzerland followed by a postdoc with Prof. Henk Tabak at the University of Utrecht Netherlands, where he helped to identify the fundamental biogenesis pathway of peroxisomes. Dominic joined... Read More →
avatar for Niclas Nilsson

Niclas Nilsson

Head of Open Innovation Research, LEO Pharma
Niclas is spearheading an initiative to use Open Innovation at the core of drug research with focus on science and external collaborations at LEO Pharma, a pharmaceutical company in Denmark focusing on dermatology and inflammatory skin disease. Prior to setting up the LEO Pharma... Read More →
avatar for Roger Olsson

Roger Olsson

Professor, Chemical Biology & Therapeutics, Lund University
Professor Olsson is a trained organic chemist, that learnt medicinal chemistry in the pharmaceutical industry. However, after returning to academia he is focusing on behavioral neuropharmacology using zebrafish. The main theme in the group is the focus on response profiles of drugs, connecting behaviour with neuronal signalling and drug distribution within the brain, rather than on receptor affinities and target-centered drug discovery... Read More →
avatar for Kristina Orrling

Kristina Orrling

Kristina Orrling has worked more than 15 years with various aspects of drug discovery. Her scientific activities have focused on medicinal chemistry and infectious diseases. She holds a PhD in medicinal chemistry and a MSc Chemical Engineering with drug research specialization from Uppsala University, Sweden, as well as a... Read More →

Wednesday June 7, 2017 13:40 - 15:00
Auditorium 4