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Gene-based Therapeutics for Neurological Disorders
From Basic Science to Clinical Applications
08 Feb - 11 Feb 2014
Cancun, Mexico
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Mimoun Azzouz
The University of Sheffield
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Nicholas Mazarakis
Imperial College London
Early Bird - Expired • Talk Submission - Expired • Poster Submission - Expired • Registration & Payment Deadline - Expired
Synopsis
This is a great opportunity to assemble a rich programme of international speakers to address the latest advances in gene therapy, from basic research of vector design, modelling and pre-clinical testing to clinical applications in CNS disorders such as Parkinson’s, Alzheimer’s, Huntington’s, motor neuron diseases and eye diseases.
The conference will bring together basic and clinical scientists to discuss recent discoveries in genetics and mechanisms of disease (e.g. the role of RNA metabolism and processing in neurological diseases, including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), spinal muscular atrophy (SMA), and repeat expansion disorders) and how these discoveries could help develop novel gene based therapeutics for neurodegenerative diseases. Further topics such as challenges in vector manufacturing and views of regulatory agencies on gene therapy clinical applications will be discussed.
Confirmed Plenary Speakers
Professor Richard Jude Samulski
University of North Carolina at Chapel Hill
Professor Jeffrey H. Kordower
Rush University, Chicago
Professor Ronald G Crystal
Weill Cornell Medical College
Dr Krystof Bankiewicz University of California, San Francisco
Confirmed Invited Speakers
Dr Fraser Wright
Childrens Hospital of Philadelphia
Dr Maria Kinali
Imperial College, London
Bernard Schneider
Ecole Polytechnique Fédérale de Lausanne
Professor Alberto Auricchio
TIGEM
Dr. Hicham Fenniri
QBRI
Prof. Giuseppe Battagli
University College London
Bas Blits
uniQure B.V.
Dr Howard Federoff
Georgetown University
Dr Daniel Lipinsky
University of Oxford
Joost Verhaagen
Netherlands Institute for Neuroscience
Dr Brian Kaspar
Nationwide Children's Hospital, Columbus
Confirmed Speakers
Chairs
Mimoun Azzouz
Professor, The University of Sheffield
Nicholas Mazarakis
Lucas Lee Chair of Molecular BioMedicine, Imperial College London
Plenary Speakers
R. Jude Samulski
Director, Gene Therapy Center, University of North Carolina at Chapel Hill
Ronald Crystal
Chairman, Department of Genetic Medicine, Weill Medical College of Cornell University
Krystof Bankiewicz
Professor, University of California San Francisco
Jeffrey Kordower
Professor, Rush University Medical Center
Invited Speakers
Bas Blits
Head neurobiology, uniQure
Bernard Schneider
Research and Teaching Associate, Ecole Polytechnique Federale de Lausanne
Joost Verhaagen
Prof, Netherlands Institute for Neuroscience
Alberto Auricchio
Director of the Molecular Therapy (MT) Research Programme, TIGETelethon Institute of Genetics and Medicine (TIGEM)
J Fraser Wright
Associate Professor, Director Clinical Gene Therapy Core Facility, Children's Hospital of Philadelphia
Maria Kinali
Consultant paediatric Neurologist and Honorary Senior clinical Lecturer , Chelsea and Westminster Hospital
Howard Federoff
Executive Vice President for Health Sciences and Executive Dean , Georgetown University Medical Center
Olivier Danos
Senior Vice President, Kadmon
Martine Barkats
"Biotherapy of Motor Neuron Disorders" Team Leader, Centre de Myologie, Inserm UMR974, Paris
Brian Kaspar
Associate Professor, The Research Institute at Nationwide Children's Hospital
Nicholas Boulis
Associate Professo rof Neurological Surgery, Emory University School of Medicine
Luis Pereira de Almeida
Prof., CNC - Center for Neuroscience and Cell Biology, University of Coimbra
Mark Tuszynski
Professor, University of California, San Diego
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 |
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11:15 – 11:50 |
Brian Kaspar |
AAV9-mediated SOD1 downregulation as a Future Therapy for Amyotrophic Lateral Sclerosis |
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11:50 – 12:20 |
Bernard Schneider Ecole Polytechnique Federale de Lausanne |
SOD1 Silencing in Motoneurons or Glia Rescues Neuromuscular Function in ALS Mice |
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12:20 – 12:45 |
Maria Grazia Biferi Centre de Myologie, Paris |
Modeling of UBIQUILIN2-Related ALS Using AAV10 Vectors |
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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 Children's 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 |
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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 |
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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 clinician's 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 |
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10:10 – 10:40 |
Ravindra Singh Iowa State University |
The Evolving Structural Map Of Therapeutic Targets For Splicing Correction In Spinal Muscular Atrophy |
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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 |
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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 |
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09:15 – 09:50 |
Krystof Bankiewicz |
Trophic or Dopamine replacement Gene Transfer in PD; Pros and Cons |
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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 |
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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 |
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11:45 – 12:20 |
Nicholas Boulis |
Delivery of Genes That Control Neural Activity in Functional Disorders |
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12:20 – 12:40 |
Denis Cecchin |
Polymer Nanaparticles Mediated Gene Delivery |
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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 |
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17:00 – 17:30 |
Refreshments |
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17:30 – 18:15 |
Jude Samulski University of North Carolina |
AAV: Progress and Challenges |
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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 |
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18:45 – 19:15 |
Joost Verhaagen Netherlands Institute for Neuroscience |
Gene Therapy To Promote Repair Of The Traumatically Injured Peripheral Nerve |
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19:15 – 19:45 |
Joseph C Glorioso University of Pittsburgh |
HSV Vectors and the Treatment of Chronic Pain |
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19:45 – 20:00 |
Discussion and Q&A |
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21:00 – 00:00 |
*Gala Night & Group Photo* |
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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 |
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10:30 – 11:00 |
Luis Pereira de Almeida University of Coimbra Portugal |
Gene Silencing Strategy for MJD |
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11:00 – 11:20 |
Refreshments |
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11:20 – 11:55 |
Alberto Auricchio TIGEM, Italy |
Eye: An Attractive Target for Gene Therapeutics |
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11:55 – 12:30 |
Nicholas Boulis Emory University School of Medicine |
General Review |
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12:30 – 12:45 |
Closing Remarks (Chairs) |
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12:45 – 13:00 |
Farewell & Goodbye (Fusion) |
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Contact usVenue & Location
Fiesta Americana Condesa
This stylish hotel features contemporary Mexican architecture, including one of the largest and most impressive thatched-roof "palapas" in the entire country. There are cultural activities, arts and crafts and sports programs to keep you constantly entertained, plus time to relax and enjoy the Mayan culture, soak up the Caribbean sunshine and revel in the international ambiance that settles in after the sun goes down.
Throughout your stay delegates will enjoy a full meal plan, inclusive of beverages. Take your pick from the aromatic Asian delights at Kaumbu, traditional fare at El Mexicano, delicious international cuisine at Kalmia Buffet or perhaps sample the sumptuous Italian dishes at Rosato. There are also several other dining opportunities such as the Cevichería, Pizzeria, Sushi Corner, pool and lounge bar areas. The Gala Night with either a Mayan or Caribbean theme takes place on the third evening of the conference with a mouth-watering feast of local cuisine, an open bar and amazing local entertainment. We welcome all delegates and their accompanying persons to the Gala Night – a truly fun filled night not to be missed!
Hotel Facilities
- Lagoon Pool
- Extensive Health Club and Spa
- Complimentary Wi-Fi in guest rooms and throughout hotel and conference areas
The Fiesta Americana Condesa, Cancun is the 2015 Winner of the Trip Advisor Certificate of Excellence and also received the Travellers Choice Award in 2014.
The remains of ancient Mayan cities are scattered throughout the Yucatan Peninsula and no trip to Cancun would be complete without a visit to these majestic temples and pyramids set amongst lush tropical vegetation. We will be working very closely with a reputable tour company who will be organising trips to Tulum, Chichen Itza, Coba and Xcaret to name a few, some of which may require a full day. Full day excursions will be arranged pre or post conference and may be on an individual or group booking so we would recommend booking extra nights to extend your stay as early as possible to avoid disappointment should these excursions be of interest to you and/or your party. For further information please contact us.
General Information
Venue Rating
★ ★ ★ ★ ★
Currency
US Dollar (USD)
Address
Fiesta Americana Condesa, Blvd.Kukulkán km 16.5 Zona Hotelera, Cancun, 77500 Mexico
Nearest Airport
Cancun International Airport
Location
Cancun is a delightful combination of natural beauty, islands, ecological reserves and white sandy beaches. However, besides sun, sand and sea, this destination also offers an infinite variety of underwater activities to choose from: the diving, snorkelling and fishing here are outstanding and you will find an undersea world packed with tropical fish that live on the second largest barrier reef in the world. Sports enthusiasts might choose one of the many eco-tourism activities, such as cycling or hiking through the tropical forest or kayaking through mangroves, or something a little more adventuresome, like zip lining through the treetops.
Apart from the more well-known ancient Mayan archealogical sites such as Tulum, Cobá and Chichén Itzá you may like to visit the Aktun Chen caverns, voted one of the Top 10 underwater walks by National Geographic described as a truly magical experience.
Gallery
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Conference Manager
Sarah Trundle
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As a family run business, our dedication runs deep. We’re committed to each other and, even more so, to every attendee’s experience, delivering a level of care and passion that’s truly unmatched.
