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Scientific Meeting & Research Roundtable Agenda
All agendas are tentative. Times, sessions, and speakers are subject to change.
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Thursday, June 26, 2025
8:00 (60 minutes) Continental Breakfast
9:00 (10 minutes) Welcome (Kathleen M. Loomes, MD and Stacey S. Huppert, PhD)
Intro for Keynote
9:10 (15 minutes) Ronald J. Sokol, MD (Children’s Hospital Colorado)
https://som.cuanschutz.edu/Profiles/Faculty/Profile/5796
Keynote: Pediatric Cholestatic Liver Disease: Evolution from the Past to Present State
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Session 1: Deconstructing the Cellular and Physiological Basis of Cholestatic Disease
Description: Key aspects of development that contribute to cholestatic disease such as understanding bile duct development, canalicular development, hepatocyte maturation, mesenchymal-epithelial interactions, ciliogenesis, and bile acids will be covered.
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Discussion Leaders:
Sarah Taylor, MD (Children’s Hospital of Colorado)
https://som.cuanschutz.edu/Profiles/Faculty/Profile/37607
Alexander G. Miethke, MD (Cincinnati Children’s Hospital Medical Center)
https://www.cincinnatichildrens.org/research/divisions/g/gastroenterology/labs/miethke
9:25 (5 minutes) Intro for Session 1 by the Discussion Leaders
9:30 (20 minutes) Marino Zerial, PhD (Human Technopole, Italy)
https://humantechnopole.it/en/
Alterations of hepatocyte polarity and function induced by increased intraluminal pressure in liver disease
9:50 (20 minutes) Adam Gracz, PhD (Emory University)
Building and breaking bile ducts: linking development and regeneration
10:10 (20 minutes) Pieter C. Dorrestein, PhD (University of California, San Diego)
https://dorresteinlab.ucsd.edu/
Scaling The Discovery of New Microbial Bile Acids Through Data Science of Unused Metabolomics Data
10:30 Break (10 minutes)
10:40 (10 minutes) Abstract presentation
10:50 (10 minutes) Abstract presentation
11:00 (10 minutes) Bench to bedside – Clinical and research implications by the Discussion Leaders
Panel discussion
11:10 Break (10 minutes)
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Flash Talk Session: Highlighting the research in the cholestatic disease space
Description: Short talks will be presented to allow speakers to gain visibility to all Scientific Meeting investigators and patient/family attendees. The presentations will represent and provide the broad range of research in the cholestatic disease-related field. The early placement of this session in the program is critical for the audience members to have ample time to interact with the flash talk presenters during the breaks/informal interaction times, and Research Roundtables set aside during the Summit.
Discussion Leaders:
Kathleen M. Loomes, MD (Children’s Hospital of Pennsylvania)
https://www.med.upenn.edu/apps/faculty/index.php/g275/p3872
Stacey S. Huppert, PhD (Cincinnati Children’s Hospital Medical Center)
https://www.cincinnatichildrens.org/research/divisions/g/gastroenterology/labs/huppert
11:20 (5 minutes) Intro for Flash Talk Session by the Discussion Leaders
11:25 – 11:55 10 talks that are 3 minutes each
12:00 (60 minutes) Group Photo and Lunch
Session 2: Condensing Cholestatic Disease to Genes and Pathways
Description: Underpinnings of genetic diagnosis, discovery of genetic modifiers and new biomarkers, as well as platforms for identifying candidates and/or testing causation will be presented.
Discussion Leaders:
Nancy B. Spinner, PhD (Children’s Hospital of Philadelphia)
https://www.research.chop.edu/people/nancy-b-spinner
James E. Squires, MD, MS (Children’s Hospital of Los Angeles)
https://providers.upmc.com/provider/james-squires/1324685
1:00 (5 minutes) Intro for Session 2 by the Discussion Leaders
1:05 (20 minutes) Takuya Sakaguchi, PhD (Cleveland Clinic)
https://www.lerner.ccf.org/inflammation-immunity/sakaguchi/
Forward genetic analysis on biliary system formation in zebrafish
1:25 (20 minutes) Saul Karpen, MD, PhD (Virginia Commonwealth University)
Genetic and developmental contributors to the etiology of biliary atresia
1:45 (20 minutes) Sofia Ferreira Gonzalez, MSc, PhD (University of Edinburgh, Scotland)
https://www.ed.ac.uk/inflammation-research/people/principal-investigators/dr-sofia-ferreira
Cellular senescence: the “Achilles’ Heel” of liver transplantation
2:05 Break (10 minutes)
2:15 (10 minutes) Abstract presentation
2:25 (10 minutes) Abstract presentation
2:35 (10 minutes) Bench to bedside – Clinical and research implications by the Discussion Leaders
Panel discussion
2:45 Break (10 minutes)
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Session 3: Replicating Cholestatic Liver Disease in Experimental Models
Description: Novel approaches and standardization of assays to model and enable recognition of primary versus secondary aspects of cholestatic disease in addition to distinguishing subtypes of cholestatic diseases will be discussed. The presentations will inform commonalities and differences between cholestatic diseases using novel experimental models such as organoids, organs on a chip, and animals. Strengths, limitations and opportunities of simple to complex experimental models – from single cell lineage to multi-cell lineage with and without addition of immune cells and bile acids to investigate trajectory of pathogenesis will be addressed.
Discussion Leaders:
Holger Willenbring, MD, PhD (University of California, San Francisco)
https://willenbringlab.ucsf.edu/
Laura Bull, PhD, RDN (University of California, San Francisco)
https://bms.ucsf.edu/people/laura-bull-phd
2:55 (5 minutes) Intro for Session 3 by the Discussion Leaders
3:00 (20 minutes) Nataliya Razumilava, MD (University of Michigan)
https://www.uofmhealth.org/profile/3858/nataliya-razumilava-md
Preclinical models of extrahepatic bile duct disorders
3:20 (20 minutes) Gregory Underhill, PhD (University of Illinois Urbana-Champaign)
https://underhill.bioen.illinois.edu/
Engineering Microenvironments to Study Liver Differentiation and Notch Signaling
3:40 (20 minutes) Luke Boulter, PhD (University of Edinburgh, Scotland)
https://www.boulter-lab.co.uk/
Beyond scarring – understanding the reciprocal relationships between ductular cells and their regenerative niche
4:00 Break (10 minutes)
4:10 (10 minutes) Abstract presentation
4:20 (10 minutes) Abstract presentation
4:30 (10 minutes) Bench to bedside – Clinical and research implications by the Discussion Leaders
Panel discussion
4:40 Break (10 minutes)
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Session 4 - Developing Therapies Addressing Specific Needs in Cholestatic Disease
Description: Emerging therapeutic strategies will be evaluated, and the potential needs for personalized therapies to address genetic variants, genetic modifiers, cell therapies, and combinations will be communicated.
Discussion Leaders:
Rohit Kohli, MBBS, MS (Children’s Hospital Los Angeles)
https://www.chla.org/research/lab/kohli
Hamed Jafar-Nejad, MD (Baylor College of Medicine)
https://www.bcm.edu/research/faculty-labs/hamed-jafar-nejad-lab
4:50 (5 minutes) Intro for Session 4 by the Discussion Leaders
4:55 (20 minutes) Amita Tiyaboonchai, PhD (Oregon Health and Science University)
https://www.ohsu.edu/people/amita-tiyaboonchai
Allogeneic hepatocyte transplantation without immune suppression
5:15 (20 minutes) Rebecca Adherns-Nicklas, MD, PhD (Children’s Hospital of Philadelphia)
Optimizing gene therapy for hepatic inborn errors of metabolism
5:35 (20 minutes) Eric Lagasse, PharmD, PhD (University of Pittsburgh)
https://mirm-pitt.net/staff/eric-lagasse-pharmd-phd/
Growing a Surrogate Liver
5:55 Break (10 minutes)
6:05 (10 minutes) Abstract presentation
6:15 (10 minutes) Abstract presentation
6:25 (10 minutes) Bench to bedside – Clinical and research implications by the Discussion Leaders
Panel discussion
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6:35 (15 minutes) Binita M. Kamath, MBBChir, MRCP, MTR (Children’s Hospital of Philadelphia)
https://www.chop.edu/doctors/kamath-binita
Keynote: Pediatric Cholestatic Liver Disease: Hope for the Future
Friday, June 27, 2025
7:00 (60 minutes) Continental Breakfast
8:00 (15 minutes) Overview of Scientific Meeting (Kathleen M. Loomes, MD and Stacey S. Huppert, PhD)
Overview and Goals of the Roundtables with academic, industry, patient advocacy groups, patients, families and caregivers to further inform research efforts.
8:15 (45 minutes) Discussion Leaders selected from BARE Inc., PFIC Network, and ALGSA.
15 minutes Introduction
10 minutes ALGS - survey analyses and topics that will be discussed
10 minutes PFIC - survey analyses and topics that will be discussed
10 minutes BA - survey analyses and topics that will be discussed
Collective Roundtables
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9:00 (60 minutes) Round 1
Discussion of multiple topics at different tables
Share highlights of discussion for each topic
10:00 (60 minutes) Round 2
Discussion of multiple topics at different tables
Share highlights of discussion for each topic
11:00 (60 minutes) Round 3
Discussion of multiple topics at different tables
Share highlights of discussion for each topic
NOTE: Breaks will be taken, dependent on flow of discussion.
12:00 (60 minutes) Group Photo and Lunch
Disease Specific Roundtables
1:00
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ALGS
Discussion Leader David A. Piccoli, MD (Children’s Hospital of Pennsylvania)
https://www.med.upenn.edu/apps/faculty/index.php/g275/p13006
PFIC
Discussion Leader Richard J. Thompson, BM, BCh, MRCP, MRCPCH (King’s College London)
https://kclpure.kcl.ac.uk/portal/en/persons/richard.j.thompson
BA
Discussion Leader Sonny Harpavat, MD, PhD (Texas Children’s Hospital) https://www.texaschildrens.org/find-a-provider/sanjiv-harpavat-md-phd
PSC
Discussion Leader Cara Mack, MD (Children’s Wisconsin)
https://childrenswi.org/physician-directory/m/mack-cara-lynn
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