2019 Conference

 
 
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RUNX1 ASPEN 2019
3RD ANNUAL SCIENTIFIC CONFERENCE
ASPEN MEADOWS RESORT, COLORADO, OCT 2-4, 2019

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Organizer: RUNX1 Research Program

Our third annual scientific conference took place this fall at the Aspen Meadows Resort in Aspen, Colorado - home of the Aspen Institute.

Recipients of our shared grant programs with Alex’s Lemonade Stand Foundation and the  Leukemia & Lymphoma Society, as well as our independent grant awardees, presented on the status of their research. There was also an update on the state of the RUNX1-FPD Centralized Mutation Database as well as the status of the now-live, NIH RUNX1-FPD Clinic.

The objectives of our scientific meetings are to learn from each other and to encourage the cross-pollination of ideas to advance our program’s research prerogatives quickly and efficiently.

Over the last several years we have been creating a global community of stakeholders tackling RUNX1-FPD, and this meeting offered us an invaluable opportunity to have our community of researchers come together under one roof.

Last year, there was overwhelming positive feedback on the inclusion of guest speakers who are thought leaders in clonal hematopoiesis at our Santa Barbara conference. This year we were honored to have Dr. Matt Porteus of Stanford give a keynote address regarding the advances in gene editing of hematopoietic stem cells.


PROGRAM

Aspen Meadows Resort
845 Meadows Rd, Aspen, CO 81611

Wednesday, October 2

Location: Albright Pavilion; Walter Isaacson Center

  • 17:00 – 18:00 Opening reception and cocktail-hour

  • 18:00 – 18:10 Tim and Monica Babich - Opening remarks

  • 18:10 – 18:25 Katrin Ericson - State of RRP

  • 18:25 - 18:45 Joy Anderson - A patient family story

  • 18:45 – 19:45 Dinner

Session 1. New RRP Awardees and Updates on the Natural History Study and Mutation Database

Moderator: Katrin Ericson

  • 19:45 – 20:00 Zuzana Tothova - Investigating phase separated condensates in FPD to MDS/AML progression

  • 20:00 - 20:05 Q&A

  • 20:05 – 20:20 Martijn Ernst - Deconstructing mesenchymal niche contributions to disease pathogenesis in FPD/AML

  • 20:20 - 20:25 Q&A

  • 20:25 – 20:55 Lea Cunningham/Matt Merguerian/Kathy Calvo - Early results from the RUNX1-FPD NIH natural history study

  • 20:55 – 21:00 Q&A

  • 21:00 - 22:00 Dessert and Networking


Thursday, October 3

Location: Davis Commons ; Walter Isaacson Center

  • 7:00 – 8:15 Breakfast

Location: Lauder Room; Koch Building

  • 8:30 – 8:45 Anna Brown - RUNX1 mutation data aggregation, analysis and sharing

  • 8:45 – 8:50 Q&A

  • 8:50 - 9:05 Ryan Carr - Spectrum of Abnormalities and Clonal Transformation in Germline RUNX1 FPDMM and a Comparative Analysis with Somatic RUNX1 Mutations in Myeloid Neoplasms

  • 9:05 - 9:10 Q&A

Session 2. Targeting Inflammation

Moderator: Grover Bagby

  • 9:10 – 9:25 Nancy Speck - Mouse models of secondary mutations in RUNX1 familial platelet disorder

  • 9:25 – 9:30 Q&A

  • 9:30 – 9:45 Anupriya Agarwal - Role of inflammatory environment in clonal evolution and progression from FPD to AML

  • 9:45 – 9:50 Q&A

  • 9:50 – 10:15 Coffee Break

Session 3. Disease Models

Moderator: Marshall Horwitz

  • 10:15 – 10:30 Len Zon - Barcoding fish with RUNX1 Deficiency

  • 10:30 – 10:35 Q&A

  • 10:35 – 10:50 Lucio Castilla - Modeling human pathogenic RUNX1 mutations in mice

  • 10:50 – 10:55 Q&A

  • 10:55 – 11:10 Eirini Papapetrou - Modeling progression of RUNX1-FPD into MDS and AML using CRISPR-mediated gene editing of patient-derived iPSCs

  • 11:10 – 11:15 Q&A

  • 11:15 – 11:30 Ravi Majeti - Modeling FPD/AML RUNX1 mutations in primary human HSPCs

  • 11:30 – 11:35 Q&A

Session 4. Advancing Ongoing Research Towards a Cure

Moderator: Katrin Ericson

  • 11:35 – 11:45 Introduction to breakout sessions and team lists

Location: Davis Commons ; Walter Isaacson Center

  • 11:45 – 12:45 Lunch

Location: Lauder Room; Koch Building

  • 12:45 – 13:30 Breakout: Individual teams meet and discuss assigned topics

  • 13:30 – 13:45 Team 1 - report back

  • 13:45 - 14:00 Team 2 - report back

  • 14:00 - 14:15 Team 3 - report back

  • 14:15 - 14:30 Team 4 - report back

  • 14:30 - 14:45 Coffee Break

Session 5. Gene Editing

Moderator: Nancy Speck

  • 14:45 - 15:15 Keynote Speaker: Advances in gene editing of HSPCs - Matt Porteus

  • 15:15 - 15:30 Q&A

  • 15:30 - 15:45 Paul Liu - Modeling clonal competition between RUNX1 mutated HSPCs and wild-type HSPCs in rhesus macaque

  • 15:45- 15:50 Q&A

  • 15:50 - 16:15 Group discussion and key takeaways for the day

  • 16:15 - 17:30 Free time

  • 17:30 – 18:30 Hike/SAB meeting

  • 19:00 – 20:00 Dinner


Friday, October 4

Location: Davis Commons ; Walter Isaacson Center

  • 7:00 – 8:15 Breakfast

Session 6. Raising RUNX1 Activity

Location: Lauder Room; Koch Building

Moderator: Paul Liu

  • 8:30 – 8:45 Morty Poncz - Drug screen for RUNX1-FPD therapeutics

  • 8:45 – 8:50 Q&A

  • 8:50 – 9:05 Alan Cantor - Pharmacologic Enhancement of Residual Wild Type RUNX1 Protein Activity in FPD/AML

  • 9:05 – 9:10 Q&A

  • 9:10 – 9:25 Stephen Nimer - Epigenetic modifying enzymes in FPD/AML

  • 9:25 – 9:30 Q&A

  • 9:30 - 9:45 . Coffee Break

Session 7. Targeting Multi-hit Progenitors

Moderator: Len Zon

  • 9:45 – 10:00 Laura Simon - RUNX1 mutations that confer exquisite sensitivity to glucocorticoids

  • 10:00 – 10:05 Q&A

  • 10:05 – 10:20 Waihay Wong - Acquired platelet abnormalities in myelodysplastic syndrome

  • 10:20 – 10:25 Q&A

  • 10:25 – 10:45 Private collection and discussion

  • 10:45 – 11:30 Breakout sessions - driving multi-group collaborations

  • 11:30 - 11:50 Group discussion and key takeaways for the day

  • 11:50 - 12:00 Wrap up and closing remarks

    Location: Davis Commons ; Walter Isaacson Center

  • 12:00 – 13:00 Lunch