Web Desc
Protein Replacement through Nucleic Acid Therapies
Organizer(s): Pad Chivukula, Jean Bennett and Paloma Giangrande
Date: April 07 - 10, 2019
Location: Steamboat Grand, Steamboat Springs, CO, USA
Sponsored by Moderna, Novo Nordisk A/S and Sarepta Therapeutics
For important information on the coronavirus, please click here
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.
Scholarship Deadline: January 8 2019
Discounted Abstract Deadline: January 8 2019
Abstract Deadline: February 5 2019
Discounted Registration Deadline: February 5 2019
Abstract submission is now closed. Registered attendees may bring a poster onsite. Please contact our office at +1 800-253-0685; +1 970-262-1230 or email info@keystonesymposia.org if you are interested.


Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:
ModernaNovo Nordisk A/SSarepta Therapeutics
We gratefully acknowledge additional support from these exhibitors at this conference:
TriLink BioTechnologies, Inc.
We gratefully acknowledge additional support for this conference from:
American Society of Gene & Cell Therapy (ASGCT)Arcturus Therapeutics, Inc.
We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

Arcturus Therapeutics, Inc.

Burke Bioventures

Moderna

Spark Therapeutics

Translate Bio
We gratefully acknowledge the generous grant for this conference provided by:

National Center for Advancing Translational Sciences (NCATS)
Grant No. 1R13TR002781-01
Funding for this conference was made possible (in part) by 1R13TR002781-01 from the National Institutes of Health. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

Click here to view more of these organizations
Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

Click here to view more of these organizations

Program

Sunday, April 07 | 4:00PM - 8:00PM
Arrival and Registration
Room: Korbel Foyer


Sunday, April 07 | 6:00PM - 8:00PM
Welcome Mixer
Room: Korbel Foyer
No registration fees are used to fund alcohol served at this function.

Monday, April 08 | 7:00AM - 8:00AM
Breakfast
Room: Korbel Grand Ballroom


Monday, April 08 | 8:00AM - 9:00AM
Welcome and Keynote Address
Room: Korbel Grand Ballroom

Speaker 1 of 2
* Pad Chivukula, Arcturus Therapeutics, Inc., USA

Monday, April 08 | 8:00AM - 9:00AM
Welcome and Keynote Address
Room: Korbel Grand Ballroom

Speaker 2 of 2
Pieter Cullis, University of British Columbia, Canada
Effective Non-Viral Vectors for Delivery of mRNA in vivo

Monday, April 08 | 9:00AM - 11:30AM
Design and Delivery
Room: Korbel Grand Ballroom

Speaker 1 of 6
* Paul Burke, Burke Bioventures LLC, USA
LNPs for Delivery of mRNA Therapeutics: Lessons from Translation

Monday, April 08 | 9:00AM - 11:30AM
Design and Delivery
Room: Korbel Grand Ballroom


Coffee Break

Monday, April 08 | 9:00AM - 11:30AM
Design and Delivery
Room: Korbel Grand Ballroom

Speaker 3 of 6
Stephen L. Hart, University College London, UK
Nucleic Acid Therapeutics for Cystic Fibrosis Delivered by Targeted Nanoparticles

Monday, April 08 | 9:00AM - 11:30AM
Design and Delivery
Room: Korbel Grand Ballroom

Speaker 4 of 6
Manish Aneja, ethris GmbH, Germany
mRNA Transcript-Activated Matrix for Bone Regeneration

Monday, April 08 | 9:00AM - 11:30AM
Design and Delivery
Room: Korbel Grand Ballroom

Speaker 5 of 6
John H. Lockhart, University of South Florida, USA
Short Talk: Effective Delivery of Therapeutic mRNA using Peptide-Based Nanoparticles

Monday, April 08 | 9:00AM - 11:30AM
Design and Delivery
Room: Korbel Grand Ballroom

Speaker 6 of 6
Jeffrey Rubin, Mayo Clinic Graduate School of Biomedical Sciences, USA
Short Talk: Enhanced Gene Delivery to the Kidney by Adenovirus and Adeno-Associated Virus

Monday, April 08 | 11:30AM - 5:00PM
On Own for Lunch


Monday, April 08 | 11:30AM - 1:00PM
Poster Setup
Room: Korbel Grand Ballroom


Monday, April 08 | 1:00PM - 10:00PM
Poster Viewing
Room: Korbel Grand Ballroom


Monday, April 08 | 4:30PM - 5:00PM
Coffee Available
Room: Korbel Foyer


Monday, April 08 | 5:00PM - 7:00PM
Protein Replacement with mRNA
Room: Korbel Grand Ballroom

Speaker 1 of 5
* Anton McCaffrey, TriLink BioTechnologies, USA

Monday, April 08 | 5:00PM - 7:00PM
Protein Replacement with mRNA
Room: Korbel Grand Ballroom

Speaker 2 of 5
Pad Chivukula, Arcturus Therapeutics, Inc., USA
Protein Replacement with mRNA

Monday, April 08 | 5:00PM - 7:00PM
Protein Replacement with mRNA
Room: Korbel Grand Ballroom

Speaker 3 of 5
Jeffrey R. Holt, Boston Children's Hospital, USA
Improved Gene Therapy Restores Hearing, Balance, and Secondary Measures in Mice with Genetic Inner Ear Disorders

Monday, April 08 | 5:00PM - 7:00PM
Protein Replacement with mRNA
Room: Korbel Grand Ballroom

Speaker 4 of 5
Keiji Itaka, Tokyo Medical and Dental University, Japan
mRNA Medicine for Introducing Therapeutic Transcription Factors

Monday, April 08 | 5:00PM - 7:00PM
Protein Replacement with mRNA
Room: Korbel Grand Ballroom

Speaker 5 of 5
Jia Tay, Translate Bio, USA
Advancing the Development of mRNA Therapeutics to Restore Protein Function

Monday, April 08 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Korbel Grand Ballroom
No registration fees are used to fund alcohol served at this function.

Monday, April 08 | 7:30PM - 10:00PM
Poster Session 1
Room: Korbel Grand Ballroom


Tuesday, April 09 | 7:00AM - 8:00AM
Breakfast
Room: Korbel Grand Ballroom


Tuesday, April 09 | 8:00AM - 11:00AM
Protein Replacement with Gene Therapy
Room: Korbel Grand Ballroom

Speaker 1 of 5
* Mark A. Kay, Stanford University, USA
Novel Recombinant AAVs for Classical and Genome Editing Applications

Tuesday, April 09 | 8:00AM - 11:00AM
Protein Replacement with Gene Therapy
Room: Korbel Grand Ballroom

Speaker 2 of 5
Beverly L. Davidson, Children’s Hospital of Philadelphia and University of Pennsylvania, USA
Considerations in Gene Therapy for the LSDs

Tuesday, April 09 | 8:00AM - 11:00AM
Protein Replacement with Gene Therapy
Room: Korbel Grand Ballroom


Coffee Break

Tuesday, April 09 | 8:00AM - 11:00AM
Protein Replacement with Gene Therapy
Room: Korbel Grand Ballroom

Speaker 4 of 5
Federico Mingozzi, Spark Therapeutics, Inc., USA
Liver Gene Transfer as Enzyme Replacement Therapy in Lysosomal Storage Diseases

Tuesday, April 09 | 8:00AM - 11:00AM
Protein Replacement with Gene Therapy
Room: Korbel Grand Ballroom

Speaker 5 of 5
Jean Bennett, University of Pennsylvania, USA
Seeing the Light with Retinal Gene Therapy: From Fantasy to Reality

Tuesday, April 09 | 11:00AM - 5:00PM
On Own for Lunch


Tuesday, April 09 | 1:00PM - 10:00PM
Poster Viewing
Room: Korbel Grand Ballroom


Tuesday, April 09 | 4:30PM - 5:00PM
Coffee Available
Room: Korbel Foyer


Tuesday, April 09 | 5:00PM - 7:00PM
Therapeutic Applications
Room: Korbel Grand Ballroom

Speaker 1 of 4
Paloma Giangrande, Moderna, USA
Protein Replacement with mRNA for Inherited Metabolic Diseases

Tuesday, April 09 | 5:00PM - 7:00PM
Therapeutic Applications
Room: Korbel Grand Ballroom

Speaker 2 of 4
* Drew Weissman, University of Pennsylvania, USA
Talk Title to be Announced

Tuesday, April 09 | 5:00PM - 7:00PM
Therapeutic Applications
Room: Korbel Grand Ballroom

Speaker 3 of 4
Adrian R. Krainer, Cold Spring Harbor Laboratory, USA
Targeted Antisense Therapeutics for Modulation of Splicing or NMD

Tuesday, April 09 | 5:00PM - 7:00PM
Therapeutic Applications
Room: Korbel Grand Ballroom

Speaker 4 of 4
Liangliang Hao, Massachusetts Institute of Technology, USA
Short Talk: Tumor Penetrating RNA Delivery for Therapeutic Benefit of Pancreatic Cancer

Tuesday, April 09 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Korbel Grand Ballroom
No registration fees are used to fund alcohol served at this function.

Wednesday, April 10 | 7:00AM - 8:00AM
Breakfast
Room: Korbel Grand Ballroom


Wednesday, April 10 | 8:00AM - 11:00AM
Gene Correction
Room: Korbel Grand Ballroom

Speaker 1 of 5
* Erik Sontheimer, University of Massachusetts Medical School, USA
Enhancing Genome Editing with New Cas9s and Chemically Engineered Guides and Homology-Dependent Repair Donors

Wednesday, April 10 | 8:00AM - 11:00AM
Gene Correction
Room: Korbel Grand Ballroom

Speaker 2 of 5
Annemieke Aartsma-Rus, Leiden University Medical Center, Netherlands
Development of Antisense-Mediated Exon Skipping for Duchenne: It Takes More than an Antisense Oligonucleotide

Wednesday, April 10 | 8:00AM - 11:00AM
Gene Correction
Room: Korbel Grand Ballroom


Coffee Break

Wednesday, April 10 | 8:00AM - 11:00AM
Gene Correction
Room: Korbel Grand Ballroom

Speaker 4 of 5
Patrick D. Hsu, University of California, Berkeley, USA
New Molecular Technologies for Transcriptome Engineering

Wednesday, April 10 | 8:00AM - 11:00AM
Gene Correction
Room: Korbel Grand Ballroom

Speaker 5 of 5
Ranjan Batra, Locana Bio, USA
Reversal of Microsatellite Diseases using RNA-Targeting Proteins

Wednesday, April 10 | 11:00AM - 5:00PM
On Own for Lunch


Wednesday, April 10 | 4:30PM - 5:00PM
Coffee Available
Room: Korbel Foyer


Wednesday, April 10 | 5:00PM - 6:45PM
Current Challenges
Room: Korbel Grand Ballroom

Speaker 1 of 3
Matthew Hirsch, University of North Carolina at Chapel Hill, USA
AAV Gene Therapy Prevents and Reverses MPS1 Corneal Opacity

Wednesday, April 10 | 5:00PM - 6:45PM
Current Challenges
Room: Korbel Grand Ballroom

Speaker 2 of 3
* Luk H. Vandenberghe, Harvard Medical School, USA
Development of Technology to Overcome Translational Hurdles

Wednesday, April 10 | 5:00PM - 6:45PM
Current Challenges
Room: Korbel Grand Ballroom

Speaker 3 of 3
Maria Limberis, University of Pennsylvania, USA
Using Vectors as Vaccines

Wednesday, April 10 | 6:45PM - 7:00PM
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
Room: Korbel Grand Ballroom


Wednesday, April 10 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Spring Creek/Burgess Creek
No registration fees are used to fund alcohol served at this function.

Wednesday, April 10 | 8:00PM - 11:00PM
Entertainment
Room: Spring Creek/Burgess Creek
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.

Wednesday, April 10 | 8:00PM - 11:00PM
Cash Bar
Room: Spring Creek/Burgess Creek


Thursday, April 11 | 10:24AM - 10:24AM
Departure


*Session Chair
†Invited, not yet responded.