Programme
Saturday 8th February 2014 |
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08:15 - 10:15 |
Registration & Reception |
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10:15 - 10:30 |
Opening Comments |
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Session Title: Gene Therapy Approaches for Amyotrophic Lateral Sclerosis |
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10:30 – 11:15 |
Plenary: Ronald Crystal |
Gene Therapy For CNS Disorders |
11:15 – 11:50 |
Brian Kaspar |
AAV9-mediated SOD1 downregulation as a Future Therapy for Amyotrophic Lateral Sclerosis |
11:50 – 12:20 |
Bernard Schneider Ecole Polytechnique Federale de Lausanne |
SOD1 Silencing in Motoneurons or Glia Rescues Neuromuscular Function in ALS Mice |
12:20 – 12:45 |
Maria Grazia Biferi Centre de Myologie, Paris |
Modeling of UBIQUILIN2-Related ALS Using AAV10 Vectors |
12:45 – 13:00 |
Discussion and Q & A |
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13:00 – 14:15 |
Welcome Reception/Lunch |
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14:15 – 17:30 |
Free Time |
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Session Title: GMP Manufacturing For Clinical Gene Therapy |
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17:30 – 18:10 |
J Fraser Wright Childrens Hospital of Philadelphia |
Manufacturing And Characterizing Recombinant AAV For Parkinson’s Disease Clinical Trials
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18:10 – 18:40 |
Bas Blitz |
The Use Of AAV Vectors Produced By A GMP-Compliant And Scalable Production Platform For The Treatment Of Neurodegenerative Diseases |
18:40 – 19:10 |
Refreshments |
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19:10 – 20:30 |
Debate: GMP Manufacturing, Progress and Challenges Moderator: Ronald Crystal Panel: J Fraser Wright, Bas Blits, Jude Samulski |
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20:30 – 21:15 |
Cocktails and Networking |
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21:15 – 22:30 |
Dinner & Free Time |
Sunday 9th February 2014 |
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08:20 -08:30 |
Chair Opening |
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Session Title: Gene Therapy For Spinal Muscular Atrophy |
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08:30 – 09:10 |
Maria Kinali Imperial College London |
Spinal Muscular Atrophy: a clinicians perspective on the diagnostic and management challenges
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09:10 -09:40 |
Brian Kaspar |
AAV9-Mediated systemic gene therapy for SMA
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09:40 – 10:10 |
Martine Barkats |
Comparaison of Central and Peripheral scAAV9-SMN Gene Delivery for the Treatment of Spinal Muscular Atrophy |
10:10 – 10:40 |
Ravindra Singh Iowa State University |
The Evolving Structural Map Of Therapeutic Targets For Splicing Correction In Spinal Muscular Atrophy |
10:40 – 11:10 |
Refreshments |
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11:10 – 13:00 |
Round Table Debate: Clinical Gene Therapy for SMA: Hype or Hope? Moderator: Jeffrey Kordower |
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13:00 – 18:00 |
Group Tour: Tulum (To be Confirmed) |
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19:00 – 22:00 |
Dinner & Free Time |
Monday 10th February 2014 |
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08:20 – 08:30 |
Chair Opening |
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Session Title: Parkinson’s Disease |
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08:30 – 09:15 |
Jeffrey Kordower |
Trophic Factor Gene Therapy for Parkinson’s Disease: Promises and Challenges |
09:15 – 09:50 |
Krystof Bankiewicz |
Trophic or Dopamine replacement Gene Transfer in PD; Pros and Cons |
09:50 – 10:25 |
Howard Federoff |
In vitro and in vivo evaluation of a LRRK2G2019S allele-specific shRNA as potential gene therapy for human Parkinson’s disease |
10:25 – 11:00 |
Refreshments |
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Session Title: Gene Therapy Vector Delivery & Emerging Non-Viral Technologies |
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11:00 – 11:45 |
Krystof Bankiewicz |
Influence of Viral Vector Delivery on Outcome of CNS Gene Therapy Trials |
11:45 – 12:20 |
Nicholas Boulis |
Delivery of Genes That Control Neural Activity in Functional Disorders |
12:20 – 12:40 |
Denis Cecchin |
Polymer Nanaparticles Mediated Gene Delivery |
12:40 – 16:15 |
Lunch & Free Time |
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16:15 – 17:00 |
Mark Tuszynski University of California, San Diego |
Discussion with Q&A – Growth Factor Gene Therapy for Alzheimer’s Disease |
17:00 – 17:30 |
Refreshments |
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17:30 – 18:15 |
Jude Samulski University of North Carolina |
AAV: Progress and Challenges |
Session Title: Other Gene Therapy Applications for CNS Disorders |
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18:15 – 18:45 |
Olivier Danos Kadmon |
Intracerebral Administration of AAVrh.10 Carrying Human SGSH And SUMF1 cDNAS In Children With MPSIIIA Disease: Results Of A Phase I/II Trial |
18:45 – 19:15 |
Joost Verhaagen Netherlands Institute for Neuroscience |
Gene Therapy To Promote Repair Of The Traumatically Injured Peripheral Nerve |
19:15 – 19:45 |
Joseph C Glorioso University of Pittsburgh |
HSV Vectors and the Treatment of Chronic Pain |
19:45 – 20:00 |
Discussion and Q&A |
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21:00 – 00:00 |
*Gala Night & Group Photo* |
Tuesday 11th February 2014 |
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09:30 - 9:45 |
Chair Opening |
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Session Title: New Gene Delivery Methods And Emerging Technologies |
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09:45 – 10:30 |
Nicholas Mazarakis |
Lentiviral Vectors: Prospect for Clinical Gene Therapy |
10:30 – 11:00 |
Luis Pereira de Almeida University of Coimbra Portugal |
Gene Silencing Strategy for MJD |
11:00 – 11:20 |
Refreshments |
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11:20 – 11:55 |
Alberto Auricchio TIGEM, Italy |
Eye: An Attractive Target for Gene Therapeutics |
11:55 – 12:30 |
Nicholas Boulis Emory University School of Medicine |
General Review |
12:30 – 12:45 |
Closing Remarks (Chairs) |
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12:45 – 13:00 |
Farewell & Goodbye (Fusion) |