Steamboat Grand Floorplan
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This meeting took place in 2019
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Protein Replacement through Nucleic Acid Therapies (L3)
Organizer(s) Pad Chivukula, Jean Bennett and Paloma Giangrande
April 7—10, 2019
Steamboat Grand • Steamboat Springs, CO USA
Discounted Abstract Deadline: Jan 8, 2019
Abstract Deadline: Feb 5, 2019
Scholarship Deadline: Jan 8, 2019
Discounted Registration Deadline: Feb 5, 2019
Sponsored by Moderna, Novo Nordisk A/S and Sarepta Therapeutics
Summary of Meeting:
Protein replacement therapy based on recombinant protein has so far been limited to genetic diseases in which the mutated protein acts extracellularly. Nucleic acid therapies such as gene therapy and messenger RNA enable replacement of intracellular proteins, or extracellular proteins too complex to manufacture. This opens up the potential to treat many previously unapproachable diseases. After early setbacks in gene therapy, a new generation of therapeutics are showing progress in the clinic. Novel messenger RNA and RNA delivery technologies are also in development, the potential of which is only beginning to be demonstrated. These represent exciting areas of therapeutic development which also touch on fundamental questions about regulation of gene expression, protein production, and immunity. This conference brings these communities together to discuss common challenges and complementarities, providing an opportunity for cross-fertilization.
View Scholarships/Awards
Protein replacement therapy based on recombinant protein has so far been limited to genetic diseases in which the mutated protein acts extracellularly. Nucleic acid therapies such as gene therapy and messenger RNA enable replacement of intracellular proteins, or extracellular proteins too complex to manufacture. This opens up the potential to treat many previously unapproachable diseases. After early setbacks in gene therapy, a new generation of therapeutics are showing progress in the clinic. Novel messenger RNA and RNA delivery technologies are also in development, the potential of which is only beginning to be demonstrated. These represent exciting areas of therapeutic development which also touch on fundamental questions about regulation of gene expression, protein production, and immunity. This conference brings these communities together to discuss common challenges and complementarities, providing an opportunity for cross-fertilization.
View Scholarships/Awards
No registration fees are used to fund entertainment or alcohol at this conference
The meeting will begin on Sunday, April 7 with registration from 16:00 to 20:00 and a welcome mixer from 18:00 to 20:00. Conference events conclude on Wednesday, April 10 with a closing plenary session from 17:00 to 19:00, followed by a social hour and entertainment. We recommend return travel on Thursday, April 11 in order to fully experience the meeting.
SUNDAY, APRIL 7
MONDAY, APRIL 8
TUESDAY, APRIL 9
WEDNESDAY, APRIL 10
THURSDAY, APRIL 11
Conference Program Print | View meeting in 12 hr (am/pm) time
The meeting will begin on Sunday, April 7 with registration from 16:00 to 20:00 and a welcome mixer from 18:00 to 20:00. Conference events conclude on Wednesday, April 10 with a closing plenary session from 17:00 to 19:00, followed by a social hour and entertainment. We recommend return travel on Thursday, April 11 in order to fully experience the meeting.
SUNDAY, APRIL 7
18:00—20:00
Welcome Mixer
No registration fees are used to fund alcohol served at this function.
08:00—09:00
Welcome and Keynote Address
*
Pad Chivukula,
Arcturus Therapeutics, Inc., USA
Pieter Cullis,
University of British Columbia, Canada
Effective Non-Viral Vectors for Delivery of mRNA in vivo
Effective Non-Viral Vectors for Delivery of mRNA in vivo
09:00—11:30
Design and Delivery
*
Paul Burke,
Burke Bioventures LLC, USA
LNPs for Delivery of mRNA Therapeutics: Lessons from Translation
LNPs for Delivery of mRNA Therapeutics: Lessons from Translation
Coffee Break
Stephen L. Hart,
University College London, UK
Nucleic Acid Therapeutics for Cystic Fibrosis Delivered by Targeted Nanoparticles
Nucleic Acid Therapeutics for Cystic Fibrosis Delivered by Targeted Nanoparticles
Manish Aneja,
ethris GmbH, Germany
mRNA Transcript-Activated Matrix for Bone Regeneration
mRNA Transcript-Activated Matrix for Bone Regeneration
John H. Lockhart,
University of South Florida, USA
Short Talk: Effective Delivery of Therapeutic mRNA using Peptide-Based Nanoparticles
Short Talk: Effective Delivery of Therapeutic mRNA using Peptide-Based Nanoparticles
Jeffrey Rubin,
Mayo Clinic Graduate School of Biomedical Sciences, USA
Short Talk: Enhanced Gene Delivery to the Kidney by Adenovirus and Adeno-Associated Virus
Short Talk: Enhanced Gene Delivery to the Kidney by Adenovirus and Adeno-Associated Virus
17:00—19:00
Protein Replacement with mRNA
*
Anton McCaffrey,
TriLink BioTechnologies, USA
Pad Chivukula,
Arcturus Therapeutics, Inc., USA
Protein Replacement with mRNA
Protein Replacement with mRNA
Jeffrey R. Holt,
Boston Children's Hospital, USA
Improved Gene Therapy Restores Hearing, Balance, and Secondary Measures in Mice with Genetic Inner Ear Disorders
Improved Gene Therapy Restores Hearing, Balance, and Secondary Measures in Mice with Genetic Inner Ear Disorders
Keiji Itaka,
Tokyo Medical and Dental University, Japan
mRNA Medicine for Introducing Therapeutic Transcription Factors
mRNA Medicine for Introducing Therapeutic Transcription Factors
Jia Tay,
Translate Bio, USA
Advancing the Development of mRNA Therapeutics to Restore Protein Function
Advancing the Development of mRNA Therapeutics to Restore Protein Function
19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.
08:00—11:00
Protein Replacement with Gene Therapy
*
Mark A. Kay,
Stanford University, USA
Novel Recombinant AAVs for Classical and Genome Editing Applications
Novel Recombinant AAVs for Classical and Genome Editing Applications
Beverly L. Davidson,
Children’s Hospital of Philadelphia and University of Pennsylvania, USA
Considerations in Gene Therapy for the LSDs
Considerations in Gene Therapy for the LSDs
Coffee Break
Federico Mingozzi,
Spark Therapeutics, Inc., USA
Liver Gene Transfer as Enzyme Replacement Therapy in Lysosomal Storage Diseases
Liver Gene Transfer as Enzyme Replacement Therapy in Lysosomal Storage Diseases
Jean Bennett,
University of Pennsylvania, USA
Seeing the Light with Retinal Gene Therapy: From Fantasy to Reality
Seeing the Light with Retinal Gene Therapy: From Fantasy to Reality
17:00—19:00
Therapeutic Applications
*
Drew Weissman,
University of Pennsylvania, USA
Talk Title to be Announced
Talk Title to be Announced
Adrian R. Krainer,
Cold Spring Harbor Laboratory, USA
Targeted Antisense Therapeutics for Modulation of Splicing or NMD
Targeted Antisense Therapeutics for Modulation of Splicing or NMD
Liangliang Hao,
Massachusetts Institute of Technology, USA
Short Talk: Tumor Penetrating RNA Delivery for Therapeutic Benefit of Pancreatic Cancer
Short Talk: Tumor Penetrating RNA Delivery for Therapeutic Benefit of Pancreatic Cancer
19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.
08:00—11:00
Gene Correction
*
Erik Sontheimer,
University of Massachusetts Medical School, USA
Enhancing Genome Editing with New Cas9s and Chemically Engineered Guides and Homology-Dependent Repair Donors
Enhancing Genome Editing with New Cas9s and Chemically Engineered Guides and Homology-Dependent Repair Donors
Annemieke Aartsma-Rus,
Leiden University Medical Center, Netherlands
Development of Antisense-Mediated Exon Skipping for Duchenne: It Takes More than an Antisense Oligonucleotide
Development of Antisense-Mediated Exon Skipping for Duchenne: It Takes More than an Antisense Oligonucleotide
Coffee Break
Patrick D. Hsu,
University of California, Berkeley, USA
New Molecular Technologies for Transcriptome Engineering
New Molecular Technologies for Transcriptome Engineering
Ranjan Batra,
Locana Bio, USA
Reversal of Microsatellite Diseases using RNA-Targeting Proteins
Reversal of Microsatellite Diseases using RNA-Targeting Proteins
17:00—18:45
Current Challenges
Matthew Hirsch,
University of North Carolina at Chapel Hill, USA
AAV Gene Therapy Prevents and Reverses MPS1 Corneal Opacity
AAV Gene Therapy Prevents and Reverses MPS1 Corneal Opacity
*
Luk H. Vandenberghe,
Harvard Medical School, USA
Development of Technology to Overcome Translational Hurdles
Development of Technology to Overcome Translational Hurdles
19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.
20:00—23:00
Entertainment
Entertainment is not subsidized by conference registration fees nor any U.S. federal government grants. Funding for this expense is provided by other revenue sources.
*Session Chair †Invited, not yet responded.
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