Steamboat Grand Floorplan

Registered Attendees


Registered attendees (and speakers, organizers, etc.) will have access to the following items from their Account page:

  • Abstracts from speakers and poster sessions, including the joint meeting abstracts, available 30 days prior to the meeting (You can edit your own abstract from My Account page as well)

    NOTE: Abstract authors/submitters may choose to not have their abstract available online and in the secure mobile app until a week before the meeting.

  • Full participant list, including joint meeting participants
  • Printable Invoices and Invitation Letters
  • Scholarship Information
  • Lodging Information

Login to My Account page

Protein Replacement through Nucleic Acid Therapies (L3)

Scientific Organizers: Pad Chivukula, Jean Bennett and Paloma Giangrande


April 7—10, 2019

Steamboat Grand, Steamboat Springs, Colorado, USA


Sponsored by Moderna, Novo Nordisk A/S and Sarepta Therapeutics


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.
BEWARE of booking lodging through companies unaffiliated with Keystone Symposia. Click here for more details.

Keystone Symposia is committed to maintaining a positive and respectful environment at its meetings. To this end, please review and comply with our policies.

DEADLINES:
*All deadlines end at 11:59 PM US Mountain Standard Time
Scholarship Deadline: Jan 8, 2019 [details]
Discounted Abstract Deadline: Jan 8, 2019 [details]
Abstract Deadline: Feb 5, 2019 [details]
Discounted Registration Deadline: Feb 5, 2019 [details]

If you have missed a deadline due to the US government shutdown, please contact our office.

Click here to view Cancellation Policy

Register Yourself Deadlines and Fees

Rates and Deadlines listed below are for the 2019-L3 "Protein Replacement through Nucleic Acid Therapies" conference only.

*All deadlines end at 11:59 PM US Mountain Standard Time

Deadline Date Cost
Discounted Abstract Deadline
More details
Before and On January 8, 2019 100.00 USD*
- 50.00 USD discount
Abstract Deadline
More details
After January 8, 2019 until February 25, 2019 100.00 USD*
* 50.00 USD of Abstract fee deducted from your registration fee when you register for this meeting
Scholarship
More details
January 8, 2019 n/a
Discounted NON-STUDENT Registration
More details
Before and On February 5, 2019 795.00 USD
(includes 200.00 discount)
Regular NON-STUDENT Registration
More details
After February 5, 2019 995.00 USD
Discounted STUDENT Registration
More details
Before and On February 5, 2019 570.00 USD
(includes 200.00 discount)
Regular STUDENT Registration
More details
After February 5, 2019 770.00 USD

* Changes in payment type that result in a refund are subject to a processing fee.
* Currency exchange gains/losses are the responsibility of the registrant and the Keystone Symposia will accept no liability for money loss through bank charges, transfers or currency fluctuations.




Discounted Abstract Deadline: January 8, 2019

*All deadlines end at 11:59 PM US Mountain Standard Time

Abstract Submission Fee: 100.00 USD*
Submission by this date receives 50.00 USD discount.
*50.00 USD will be applied to Registration Fee when you register

Abstract Details: It is best to submit your abstract early. Abstract and registration spaces are limited and may fill prior to the abstract or discounted registration deadlines. Submitting an abstract does not constitute or guarantee registration.

Submitting your abstract early allows us to:
  • submit your abstract to organizers to be considered for a short talk
  • accept your abstract online
  • include your abstract on our website and in our secure mobile app
  • reserve your space at the meeting for a poster presentation.**
**Submitting an abstract does not constitute or guarantee registration.
(The Abstract Deadline: is February 5, 2019)

Global Health Travel Award Deadline:

*All deadlines end at 11:59 PM US Mountain Standard Time


Global Health Travel Award Details: You MUST complete your Global Health Travel Award appplication by the Global Health Travel Award Deadline and complete the other required steps (verify your contact information and submit a recommendation letter) to be considered for a travel award.


Click here to learn more about Global Health Travel Awards.

Scholarship Deadline: January 8, 2019

*All deadlines end at 11:59 PM US Mountain Standard Time


Scholarship Details: You MUST submit your abstract by the Scholarship Deadline and complete the other required steps (submit a scholarship application and submit a mentor letter) to be considered for a scholarship.

Click here to learn more about Scholarships.

Abstract Deadline: February 5, 2019

*All deadlines end at 11:59 PM US Mountain Standard Time


Abstract Submission Fee: 100.00 USD*
*50.00 USD will be applied to Registration Fee when you register

Details: It is best to submit your abstract early. Abstract and registration spaces are limited and may fill prior to the abstract or discounted registration deadline. Submitting an abstract does not constitute or guarantee registration.

Submitting your abstract by the Abstract Deadline allows us to:

  • submit your abstract to organizers to be considered for a short talk
  • include your abstract on our website and in our secure mobile app
  • reserve your space at the meeting for a poster presentation.**
**Submitting an abstract does not constitute or guarantee registration.
Abstracts submitted after the Abstract Deadline will NOT be considered for a short talk.

(Discounted Abstract Deadline: is January 8, 2019)

Discounted Registration Deadline: February 5, 2019

*All deadlines end at 11:59 PM US Mountain Standard Time


Registration Fee: 795.00 USD* (includes 200.00 USD discount)
Student Registration Fee: 570.00 USD* (must complete student verification form)

*Includes 50.00 USD of your abstract submission fee

After the Discounted Registration Deadline:


Registration Fee: 995.00 USD*
Student Registration Fee: 770.00 USD* (must complete student verification form)

*Includes 50.00 USD of your abstract submission fee

Registration spaces are limited and may fill prior to discounted registration deadline.

Please be advised that some meeting participants have received calls and emails from a number of different housing services, including "Exhibitors Housing Services," “Expo Housing Services,” "Global Housing," etc. THESE COMPANIES ARE NOT AFFILIATED WITH KEYSTONE SYMPOSIA and are not authorized by Keystone Symposia to contact you. These companies may tell you that the meeting is filling, without any actual knowledge to that effect, and pressure you into giving them your credit card number to make a lodging reservation with them.

We highly recommend that you not make a reservation with these companies or any other housing service! These companies' representations to participants have been misleading. Please report any calls or correspondence you might have received to Keystone Symposia and book only through lodging links in your Keystone Symposia account, an established travel agency or directly with the hotel(s) listed on our website.
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

Spring Creek/Burgess Creek
08:00—09:00
Welcome and Keynote Address
Registered attendees can view abstracts starting on 03/07/2019

Korbel Grand Ballroom
Pieter Cullis, University of British Columbia, Canada
Effective Non-Viral Vectors for Delivery of mRNA In Vivo

09:00—11:30
Design and Delivery
Registered attendees can view abstracts starting on 03/07/2019

Korbel Grand Ballroom
Paul Burke, Burke Bioventures LLC, USA
LNP Delivery

Coffee Break

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

Christian Plank, Technical University Munich, 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

Spring Creek/Burgess Creek
13:00—22:00
Poster Viewing

Spring Creek/Burgess Creek
16:30—17:00
Coffee Available

Korbel Foyer
17:00—19:00
Protein Replacement with mRNA
Registered attendees can view abstracts starting on 03/07/2019

Korbel Grand Ballroom
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.

Spring Creek/Burgess Creek
19:30—22:00
Poster Session 1

Spring Creek/Burgess Creek

TUESDAY, APRIL 9

07:00—08:00
Breakfast

Spring Creek/Burgess Creek
08:00—11:00
Protein Replacement with Gene Therapy
Registered attendees can view abstracts starting on 03/07/2019

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 The 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

Spring Creek/Burgess Creek
16:30—17:00
Coffee Available

Korbel Foyer
17:00—19:00
Therapeutic Applications
Registered attendees can view abstracts starting on 03/07/2019

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.

Spring Creek/Burgess Creek

WEDNESDAY, APRIL 10

07:00—08:00
Breakfast

Spring Creek/Burgess Creek
08:00—11:00
Gene Correction
Registered attendees can view abstracts starting on 03/07/2019

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
Registered attendees can view abstracts starting on 03/07/2019

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)
Registered attendees can view abstracts starting on 03/07/2019

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)

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 Development, 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:
Yvonne Psaila, Director, Marketing and Communications, Email: yvonnep@keystonesymposia.org,
Phone:+1 970-262-2676