Keystone Symposia

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.

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No registration fees are used to fund entertainment or alcohol at this conference

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

16:00—20:00
Arrival and Registration

Korbel Foyer
18:00—20:00
Welcome Mixer
No registration fees are used to fund alcohol served at this function.

Korbel Foyer

MONDAY, APRIL 8

07:00—08:00
Breakfast

Korbel Grand Ballroom
08:00—09:00
Welcome and Keynote Address

Korbel Grand Ballroom
* Pad Chivukula, Arcturus Therapeutics, Inc., USA

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

09:00—11:30
Design and Delivery

Korbel Grand Ballroom
* Paul Burke, Burke Bioventures LLC, USA
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

Manish Aneja, ethris GmbH, Germany
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

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

11:30—17:00
On Own for Lunch

11:30—13:00
Poster Setup

Korbel Grand Ballroom
13:00—22:00
Poster Viewing

Korbel Grand Ballroom
16:30—17:00
Coffee Available

Korbel Foyer
17:00—19:00
Protein Replacement with mRNA

Korbel Grand Ballroom
* Anton McCaffrey, TriLink BioTechnologies, USA

Pad Chivukula, Arcturus Therapeutics, Inc., USA
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

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

Jia Tay, Translate Bio, USA
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.

Korbel Grand Ballroom
19:30—22:00
Poster Session 1

Korbel Grand Ballroom

TUESDAY, APRIL 9

07:00—08:00
Breakfast

Korbel Grand Ballroom
08:00—11:00
Protein Replacement with Gene Therapy

Korbel Grand Ballroom
* Mark A. Kay, Stanford University, USA
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

Coffee Break

Federico Mingozzi, Spark Therapeutics, Inc., USA
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

11:00—17:00
On Own for Lunch

13:00—22:00
Poster Viewing

Korbel Grand Ballroom
16:30—17:00
Coffee Available

Korbel Foyer
17:00—19:00
Therapeutic Applications

Korbel Grand Ballroom
Paloma Giangrande, Moderna, USA
Protein Replacement with mRNA for Inherited Metabolic Diseases

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

Adrian R. Krainer, Cold Spring Harbor Laboratory, USA
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

19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Korbel Grand Ballroom

WEDNESDAY, APRIL 10

07:00—08:00
Breakfast

Korbel Grand Ballroom
08:00—11:00
Gene Correction

Korbel Grand Ballroom
* Erik Sontheimer, University of Massachusetts Medical School, USA
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

Coffee Break

Patrick D. Hsu, The Salk Institute for Biological Studies, USA
New Molecular Technologies for Transcriptome Engineering

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

11:00—17:00
On Own for Lunch

16:30—17:00
Coffee Available

Korbel Foyer
17:00—18:45
Current Challenges

Korbel Grand Ballroom
Matthew Hirsch, University of North Carolina at Chapel Hill, USA
AAV Gene Therapy Prevents and Reverses MPS1 Corneal Opacity

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

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

18:45—19:00
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)

Korbel Grand Ballroom
19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Spring Creek/Burgess Creek
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.

Spring Creek/Burgess Creek

THURSDAY, APRIL 11

 
Departure


*Session Chair †Invited, not yet responded.



We gratefully acknowledge support for this conference from:


Directors' Fund


These generous unrestricted gifts allow our Directors to schedule meetings in a wide variety of important areas, many of which are in the early stages of research.

Click here to view all of the donors who support the Directors' Fund.



Keystone Symposia thanks our Sponsors for generously supporting this meeting:

Moderna Novo Nordisk A/S
Sarepta Therapeutics
 

We gratefully acknowledge additional support from these exhibitors at this conference:

TriLink BioTechnologies, Inc.
Please stop by to meet these exhibitors during the conference.


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


National Institutes of Health
National Center for Advancing Translational Sciences (NCATS)
National Eye Institute (NEI)

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 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 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


If your organization is interested in joining these entities in support of Keystone Symposia, please contact: Sarah Lavicka, Director of Corporate Relations, Email: sarahl@keystonesymposia.org,
Phone:+1 970-262-2690

Click here for more information on Industry Support and Recognition Opportunities.

If you are interested in becoming an advertising/marketing in-kind partner, please contact:
Nick Dua, Senior Director, Communications, Email: nickd@keystonesymposia.org,
Phone:+1 970-262-1179