Wednesday 4 th May 2016

Transcription

1 Mitochondrial Medicine: Developing New Treatments for Mitochondrial Disease Wellcome Genome Campus Hinxton, Cambridge, UK 4-6 May 2016 Draft Conference Programme Wednesday 4 th May :30-12:50 Registration with lunch Wellcome Genome Campus Conference Centre 12:50-13:00 Welcome and Introductions Francis Crick Auditorium 13:00-14:45 Session 1: Novel mechanisms of mitochondrial disease (molecular mechanisms not directly related to oxidative phosphorylation and ATP synthesis) Chair: Marni Falk 13:00 How CLUH regulates mitochondrial function Elena Rugarli University of Koln, Germany 13:30 Mechanisms of mitochondrial homeostasis in Parkinson s disease - lessons from Drosophila Alex Whitworth 14:00 Short talk-submitted abstract 14:30 Short talk-submitted abstract 14:45-15:15 Round table 1: Molecular mechanisms in mitochondrial diseases: challenging the old dogma 15:15-15:45 Afternoon Tea 15:45-17:30 Session 2: Precision medicine for mitochondrial disorders (whole genome sequencing and diagnostic biomarkers, including metabolomics)

2 Chair: Massimo Zeviani 15:45 Targeting mitochondrial disease: a genomics approach Vamsi Mootha Harvard Medical School, USA 16:15 Title tbc Holger Prokisch Technische Universität München, Germany 16:45 Deciphering mitochondrial disease s impact beyond mitochondria: Novel therapeutic targets lie in the nutrientsensing signaling network and basic cellular processes that regulate proteotoxic stress Marni Falk 17:15 Short talk-submitted abstract 17:30-18:00 Round table 2: State of the art in clinical diagnostics. 18:00-19:00 Poster Session I (odd numbers) with drinks reception 19:00-21:00 Dinner 19:00-23:00 Cash Bar Hinxton Hall Bar and Wellcome Genome Campus Conference Centre Thursday 5 th May :30-09:00 Breakfast 09:00-10:45 Session 3: Targeted treatments for mitochondrial disease 1 (small molecules, antioxidants, inducing biogenesis) Chair: Vamsi Mootha Massachusetts General Hospital, USA 09:00 Experimental therapy for mitochondrial disease, a work in progress. Massimo Zeviani Keeping mitochondria in shape: a matter of life and death Luca Scorrano University of Padova, Italy

3 10.00 Title tbc Valeria Tiranti Foundation IRCCS-Neurological Institute C. Besta, Italy 10:30 Short talk-submitted abstract 10: Morning Coffee 11:15-13:00 Session 4: Targeted treatments for mitochondrial disease 2 (cell therapies, gene editing, novel approaches to disease prevention) Chair: Massimo Zeviani MRC Mitochondrial Biology Unit, UK 11:15 Manipulating mtdna heteroplasmy with designer TAL- Effector nuclease technology Carlos Moraes University of Miami Miller School of Medicine, USA 11:45 Can reproductive technologies prevent transmission of mitochondrial DNA disease? Mary Herbert Newcastle University, UK 12:15 Manipulating mtdna heteroplasmy with designer zincfinger nuclease technology Michal Minczuk 12:45 Short talk: submitted abstract 13:00-13:30 Round table 3: What are the most promising new treatment approaches? 13:30-14:30 Lunch :45 Session 5: Work in progress: measuring clinical progression 1 (natural history) Chair: Anu Suomalainen 14:30 Outcome measures for mitochondrial disease Saskia Koene 15:00 Natural history of adult mitochondrial disease Douglas Turnbull Newcastle University, UK 15:30 Short talk: submitted abstract

4 15:45-16:15 Round table 4: Challenges and advances in studying natural history 16:15-16:45 Afternoon Tea 16:45-18:00 Session 6: Work in progress: measuring clinical progression 1 (biomarkers) Chair: Jan Smeitink 16:45 Elucidating mechanisms of mitochondrial disease provides biomarkers and targets for intervention Anu Suomalainen 17:15 Assessing mitochondrial function in vivo Michael Murphy 17:45 Short talk: submitted abstract 18:00-18:30 Round table 5: Clinically relevant biomarkers for mitochondrial disease 18:30-19:30 Poster Session II (even numbers) with drinks reception 19:30 Conference Dinner 19:30-23:00 Cash Bar Hinxton Hall Bar and Wellcome Genome Campus Conference Centre

5 Friday 6 th May 07:30-09:00 Breakfast 09:00-10:30 Session 7: Work in progress: current active trials Chair: Marni Falk 09:00 Current treatment trials in mitochondrial diseases 09:30 Mitochondrial drug development: the saga of KH176 Jan Smeitink 10:00 Short talk: submitted abstract Short talk: submitted abstract 10:30-11:00 Round Table 6: What are the most promising new treatments? 11:00-11:30 Morning Coffee 11:30-12:30 Session 8: Late breaking news Chair: Anu Suomalainen 11:30 Short talk: submitted abstract 11:45 Short talk: submitted abstract 12:00 Short talk: submitted abstract 12:15 Short talk: submitted abstract 12:30 Conference Summary 12:45 Conference Closure & Lunch 13:30 Complimentary shuttle to Cambridge train station, Cambridge City Centre, London Stansted Airport and London Heathrow Airport