Past Conference

Gene-based Therapeutics for Neurological Disorders

From Basic Science to Clinical Applications

Date

08 Feb - 11 Feb 2014

Location

Cancun, Mexico

  • Mimoun Azzouz

    The University of Sheffield

  • 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

Mimoun Azzouz

Professor, The University of Sheffield

Nicholas Mazarakis

Nicholas Mazarakis

Lucas Lee Chair of Molecular BioMedicine, Imperial College London

Plenary Speakers
R. Jude Samulski

R. Jude Samulski

Director, Gene Therapy Center, University of North Carolina at Chapel Hill

Ronald Crystal

Ronald Crystal

Chairman, Department of Genetic Medicine, Weill Medical College of Cornell University

Krystof Bankiewicz

Krystof Bankiewicz

Professor, University of California San Francisco

Jeffrey Kordower

Jeffrey Kordower

Professor, Rush University Medical Center

Invited Speakers
Bas Blits

Bas Blits

Head neurobiology, uniQure

Bernard Schneider

Bernard Schneider

Research and Teaching Associate, Ecole Polytechnique Federale de Lausanne

Joost Verhaagen

Joost Verhaagen

Prof, Netherlands Institute for Neuroscience

Alberto Auricchio

Alberto Auricchio

Director of the Molecular Therapy (MT) Research Programme, TIGETelethon Institute of Genetics and Medicine (TIGEM)

J Fraser Wright

J Fraser Wright

Associate Professor, Director Clinical Gene Therapy Core Facility, Children's Hospital of Philadelphia

Maria Kinali

Maria Kinali

Consultant paediatric Neurologist and Honorary Senior clinical Lecturer , Chelsea and Westminster Hospital

Howard Federoff

Howard Federoff

Executive Vice President for Health Sciences and Executive Dean , Georgetown University Medical Center

Olivier Danos

Olivier Danos

Senior Vice President, Kadmon

Martine Barkats

Martine Barkats

"Biotherapy of Motor Neuron Disorders" Team Leader, Centre de Myologie, Inserm UMR974, Paris

Brian  Kaspar

Brian Kaspar

Associate Professor, The Research Institute at Nationwide Children's Hospital

Nicholas Boulis

Nicholas Boulis

Associate Professo rof Neurological Surgery, Emory University School of Medicine

Luis Pereira de Almeida

Luis Pereira de Almeida

Prof., CNC - Center for Neuroscience and Cell Biology, University of Coimbra

Mark Tuszynski

Mark Tuszynski

Professor, University of California, San Diego

Programme

Saturday 8th February 2014

08:15 - 10:15

Registration & Reception

 

10:15 - 10:30

Opening  Comments

 

Session Title: Gene Therapy Approaches for Amyotrophic Lateral Sclerosis
Session Chair Ronald Crystal & Nicholas Mazarakis

10:30 – 11:15

Plenary: Ronald Crystal
Weill Cornell Medical College

Gene Therapy For CNS Disorders

11:15 – 11:50

Brian Kaspar
The Research Institute at Nationwide Children's Hospital

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

13:00 – 14:15

Welcome Reception/Lunch

14:15 – 17:30

Free Time

Session Title:  GMP Manufacturing For Clinical Gene Therapy
Session Chair Jude Samulski

17:30 – 18:10

J Fraser Wright

Children's Hospital of Philadelphia

Manufacturing And Characterizing Recombinant AAV For Parkinson’s Disease Clinical Trials

 

18:10 – 18:40

Bas Blitz
uniQure

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

19:10 – 20:30

Debate: GMP Manufacturing, Progress and Challenges

Moderator: Ronald Crystal

Panel: J Fraser Wright, Bas Blits, Jude Samulski

20:30 – 21:15

Cocktails and Networking

21:15 – 22:30

Dinner & Free Time

Sunday 9th February 2014

08:20 -08:30

Chair Opening

Session Title: Gene Therapy For Spinal Muscular Atrophy
Session Chairs: Brian Kaspar and Mimoun Azzouz

08:30 – 09:10

Maria Kinali

Imperial College London

Spinal Muscular Atrophy: a clinician's perspective on the diagnostic and management challenges

 

09:10 -09:40

Brian Kaspar
The Research Institute at Nationwide Children's Hospital

AAV9-Mediated systemic gene therapy for SMA

 

09:40 – 10:10

Martine Barkats
Centre de Myologie, Paris

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

11:10 – 13:00

Round Table Debate: Clinical Gene Therapy for SMA: Hype or Hope?

Moderator: Jeffrey Kordower

13:00 – 18:00

Group Tour: Tulum (To be Confirmed)

19:00 – 22:00

Dinner & Free Time

Monday 10th February 2014

08:20 – 08:30

Chair Opening

Session Title: Parkinson’s Disease
Session Chair Krystof Bankiewicz

08:30 – 09:15

Jeffrey  Kordower
Rush University

Trophic Factor Gene Therapy for Parkinson’s Disease: Promises and Challenges

09:15 – 09:50

Krystof Bankiewicz
UCSF

Trophic or Dopamine replacement Gene Transfer in PD; Pros and Cons

09:50 – 10:25

Howard Federoff
Georgetown University

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

Session Title: Gene Therapy Vector Delivery & Emerging Non-Viral Technologies
Session Chair Alberto Auricchio & Mimoun Azzouz

 

 

11:00 – 11:45

Krystof Bankiewicz
UCSF

Influence of Viral Vector Delivery on Outcome of CNS Gene Therapy Trials

11:45 – 12:20

Nicholas Boulis
Emory University

Delivery of Genes That Control Neural Activity in Functional Disorders

12:20 – 12:40

Denis Cecchin
UCL London

Polymer Nanaparticles Mediated Gene Delivery

12:40 – 16:15

Lunch & Free Time

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

17:30 – 18:15

Jude Samulski

University of North Carolina

AAV: Progress and Challenges

Session Title: Other Gene Therapy Applications for CNS Disorders
Session Chair Jeffrey Kordower & Jude Samulski

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

   

21:00 – 00:00

*Gala Night & Group Photo*

Tuesday 11th February 2014

09:30 - 9:45

Chair Opening

Session Title: New Gene Delivery Methods And Emerging Technologies
Session Chair Nicholas Boulis

09:45 – 10:30

Nicholas Mazarakis
Imperial College London

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

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)

12:45 – 13:00

Farewell & Goodbye (Fusion)

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Venue & 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.

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Conference Manager

Sarah Trundle

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