VIENNA. October 8 th -10 th 2012

Transcription

1 VIENNA October 8 th -10 th 2012

2 Days of Molecular Medicine 2012 The Translational Science of Rare Diseases: From Rare to Care Dates: October 8 th, 9 th, 10 th, 2012 Venue: Garden Palace of the Liechtenstein family (Palais Lichtenstein), Vienna, Austria ( Meeting Goal: To bring together experts working on a broad spectrum of rare diseases to discuss new insights made possible by improved technologies such as next generation sequencing and the possibilities for translating these advances into new treatment approaches and therapies for patients. Meeting Organizers: Science Translational Medicine, DMM Global Foundation, IMBA, Fondation Ipsen, Karolinska Institutet, Local Organizers: Josef Penninger, IMBA, Vienna, and Christoph Klein, Munich, Germany Days of Molecular Medicine

3 MONDAY OCTOBER 8 th, am: Registration opens Sala Terrena/Foyer of the LM Palace pm Special Session: Career Paths in Translational Medicine Sala Terrena Gallery Room Fixing the MD/PhD Program: A US and European Perspective For MD/PhD, MD and PhD trainees working in translational medicine pm: Opening Remarks Hercules Hall Josef Penninger, Director, IMBA; Helmut Denk, President of the Austrian Academy of Sciences; Prince Maximillian Liechtenstein; Kenneth Chien, DMM Global Foundation and Harvard University; Orla Smith, AAAS/Science Translational Medicine pm Chair: Helmut Denk, President of the Austrian Academy of Sciences Keynote Lecture I Eric Kandel Columbia University, New York, NY, USA. Mice, Men and Mental Illness: Animal Models of Psychiatric Disorders. Days of Molecular Medicine

4 Session 1: Drugs and Targets for Rare Diseases 2.30 pm Peter Mueller Vertex, Cambridge, MA, USA Kalydeco: Targeting the G551D mutation in cystic fibrosis 3.00 pm Susan Lindquist Whitehead Institute, Cambridge, MA, USA Synuclein Pathology: A Unifying Factor In Rare and Common Diseases 3.30pm Coffee Break Sala Terrena/Gardens Session 1: Drugs and Targets for Rare Diseases cont pm Ben Philpot University of North Carolina, Chapel Hill, NC, USA A New Angle on Angelman Syndrome 4.30 pm Michael Hayden UBC, Vancouver, Canada From Mutations in the Few to Drugs for the Many 5.00 pm Lorenz Studer Memorial Sloan Kettering, New York, New York, USA From ips cells to drugs for familial dysautonomia 5.30 pm Opening Reception and Poster Session I Sala Terrena/Gardens and 3 Galleries 5.30 pm Tour of Palace Art Gallery 6 Days of Molecular Medicine 2012

5 TUESDAY OCTOBER 9 th, am: Coffee/Registration Sala Terrena/Foyer of the LM palace pm Hercules Hall Session II: Molecular Insights into Rare Diseases of Muscle and Bone 9.30 am Padraig Wright GlaxoSmithKline, UK Antisense Oligonucleotides for Duchenne Muscular Dystrophy am Josef Penninger IMBA, Vienna, Austria RANK/RANKL, a therapeutic target for bone diseases am Coffee Break Sala Terrena/Gardens pm: Session II continued Hercules Hall am Jon Seidman Harvard Medical School, Boston, MA, USA Genetic Analyses of Inherited Cardiomyopathies am Short Talk pm Short talk pm: Lunch and Poster Session I cont. ala Terrena/3 Galleries Days of Molecular Medicine

6 pm Hercules Hall Session III: Rare Diseases of Blood Chair: Christoph Klein (University of Munich) 2.00 pm Alain Fischer Necker Hospital, Paris, France Primary T cell Immunodeficiencies: From Pathophysiology to Therapy 2.30 pm Christoph Baum Hannover School of Medicine, Hannover, GER Challenges in the Preclinical Development of Gene Therapy 3.00 pm Short talk: 3.20 am Coffee Break Sala Terrena/Gardens 3.45 pm Dan Kastner NIAID, NIH, Rockville, MD, USA Rare autoinflammatory diseases: from genetics to treatment 4.15 pm Amit Nathwani University College London, London, UK Gene Therapy for Hemophilia B: From Bench to Bedside pm Sala Terrena/3 Galleries Light Bites Reception and Poster Session II 5.00 pm Tour of Palace Art Gallery 8.00 pm Speakers Dinner Hotel Sacher/private banquet room 8 Days of Molecular Medicine 2012

7 WEDNESDAY OCTOBER 10 th, am Coffee Sala Terrena/Foyer of the LM palace pm Hercules Hall Session IV: Genomics and ips Cell Technology for Rare Diseases Chair: Kenneth Chien 9.00 am Richard Gibbs Baylor College of Medicine, Houston, TX, USA Tailoring patient therapy with genomics 9.30 am Steven Schwartz UCLA, Los Angeles, CA, USA Stem Cell Derived Retinal Transplantation: The First Human Experience am Short Talk am Coffee Sala Terrena/Gardens am- 1.00pm Hercules Hall Workshop: State of the Art Technologies For Deciphering Rare Diseases Chairs: Josef Penninger and Kenneth Chien am Tony de Fougerolles Moderna Therapeutics, Cambridge, MA Modified Messenger RNA as a New Therapeutic Approach am Kiran Musunuru Harvard University Genome Editing to Generate Human Isogenic Models of Metabolic Disease am Giulio Superti Furga CeMM, Vienna, Austria Molecular Networks in Innate Immunity and Leukemias Noon Panel discussion Days of Molecular Medicine

8 pm Lunch and Poster Session II cont. Sala Terrena/Gardens and 3 Galleries pm Hercules Hall Session V: Rare Diseases of Epithelia 2.00 pm Christoph Klein University of Munich, Munich, Germany From rare to common: What we can learn about IBD from rare monogenic forms of pediatric colitis 2.30 pm Paul Tam Hong Kong University, Hong Kong Hirschsprung s disease: From cells to patients 3.00 pm Anna Wedell Karolinska Institutet, Stockholm, Sweden Inborn errors of metabolism 3.30 pm William Gahl National Institutes of Health, Rockville, MD, USA Medical mysteries and rare diseases: NIH s undiagnosed diseases program 4.00 pm Closing Remarks 6.30 pm Gala Dinner Viennese wine tavern Heuriger 10 Days of Molecular Medicine 2012

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