Cholestatic Liver Disease Summit

Friday, June 27, 2025

All agendas are tentative. Times, sessions, and speakers are subject to change.

*Please note the afternoon sessions run simultaneously with both the Scientific Roundtable Meeting and Family Conference taking place at the Hyatt Regency. See below for times and details.

Research Roundtable Meeting @ Hyatt Regency (Aurora I & II)

The Research Roundtable Meeting is designed for all stakeholders (patients, families, researchers, clinicians, and industry) to come together for a broad discussion of patient research priorities followed by working group discussions on specific topics. Each working group must consist of patients, caregivers, clinicians, researchers, and industry (biotech/pharma) representatives. These discussions are meant to deepen the connections with stakeholder groups leading to increased knowledge and potential paths forward to meet the research needs in cholestatic liver disease as well as PFIC, Biliary Atresia, and Alagille Syndrome as specific disease states.

7:00am -8:00am BREAKFAST (Aurora I & II)

8:00am - 8:05am Welcome

8:05am - 8:20am Scientific Meeting Summary

Dr. Stacey Huppert; Dr. Kathleen Loomes

8:20am - 9:05am Research Roundtable Overview

9:05am - 10:05am Roundtable Session 1 - Topics to be announced

10:05am - 10:20am BREAK - Attendees change tables

10:20am - 11:20am Roundtable Session 2 - Topics to be announced

11:20am - 11:35am BREAK - Pick up lunch

11:35am - 12:10pm Working Lunch - Attendees complete Roundtable worksheets

12:10pm - 1:00pm Lunch continues - Kids arrive for lunch

12:30 - Start of Scientific Roundtable Meeting (see below_

1:00pm Break for families until 2:00pm

Scientific Research Roundtable Meeting @ Hyatt Regency (Conf Room IV)

The Scientific Research Roundtable Meeting is a chance for scientists and researchers working or interested in cholestatic liver disease to come together for a dynamic discussion in a separate location. This meeting is primarily for scientists, researchers.

12:30pm - 1:30pm ALGS

Discussion Leader

Binita M. Kamath, MBBChir, MRCP, MTR (Children’s Hospital of Philadelphia)

https://www.med.upenn.edu/apps/faculty/index.php/g275/p13006

1:30pm - 2:30pm 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

2:30pm - 3:30pm BA

Discussion Leader

Sanjiv Harpavat, MD, PhD (Texas Children’s Hospital)

https://www.texaschildrens.org/find-a-provider/sanjiv-harpavat-md-phd

3:30pm - 4:30pm PSC

Discussion Leader

Cara Mack, MD (Children’s Wisconsin)

https://childrenswi.org/physician-directory/m/mack-cara-lynn

CLD Family Conference @ Hyatt Regency

The Cholestatic Liver Disease Summit Family Conference provides presentations, panel discussions, expert talks, and fellowship time for Alagille Syndrome, PFIC, Biliary Atresia, and other cholestatic liver disease families. Sessions are appropriate for all stakeholder groups including patients, caregivers, clinicians, researchers, and industry.

**All sessions appropriate for all disease states unless otherwise specified.**

2:00pm - 2:05pm Welcome (Aurora I & II)

2:05pm - 2:35pm Presenting Sponsor IPSEN

Our Focus on Liver Disease is Rare, Dr. Andrew Coop, Senior Vice President, Head of North America Medical Affairs (Aurora I & II)

2:35pm - 2:45pm General Session: Patient Registries

How patient registries and patient repositories aid drug discovery and drug development, Catherine Chen, PhD - NCATS/NIH - Associate Group Leader, Therapeutics Development Branch (Aurora I & II)

2:45pm - 3:15pm Importance of Patient Data and Patient Registries & Recruitment Panel

Moderator: Emily Ventura - PFIC Network; Panelists: Quinn Rabenau-McDonnell - GALA, Melissa Kochanowsky - PFIC Network, Jen Lau - BARE, Inc., Alyssa Mendel - Coordination of Rare Diseases at Sanford (CoRDS) (Aurora I & II)

3:15pm - 3:45pm Presenting Sponsor Mirum Pharmaceuticals

Advancing Research and Treatments for Patients Living with Cholestatic Liver Disease, Joanne Quan, MD – Chief Medical Officer, Doug Mogul, MD, PhD – Executive Director, Scientific Affairs (Aurora I & II)

3:45pm - 4:00pm BREAK

4:00pm - 4:10pm General Session: Advocacy (Aurora I & II)

Advocacy that Matters to Cholestatic Liver Disease Patients Speaker: Chris Porter, Vice President, Government Affairs & Policy, Travere Therapeutics

4:10pm - 4:50pm Advocacy Initiatives by Organization-Pass the Mic (Aurora I & II)

Moderator: Chris Porter, Travere Therapeutics; Panelists: Cher Bork - ALGSA, Emily Ventura - PFIC Network, Jen Lau - BARE

5:00pm - 5:45pm Breakout Sessions - room locations below. *You can attend any breakout session that interests you regardless of disease state.

Alagille Syndrome - Unlocking the Code: How Genetics Shapes Alagille Syndrome

Facilitators: Nancy Spinner, PhD, FACMG and Melissa Gilbert, PhD - Children's Hospital of Philadelphia (Conference Room VII)

Alagille Syndrome - Cardiac Involvement, preparing for cardiac surgery, new novel research

Facilitators: Dr. Greg Adamson, Dr. Noelle Ebel and Dr. Elisabeth Martin, Stanford Medicine (Conference Room VIII)

PFIC - PCORI-Community Engagement in Research: Project IMPACT & Next Steps

Facilitators: Dr. Jim Squires, Alex Perez, Melissa Kochanowsky, Dr. Gitta Lubke (Conference Room I)

Biliary Atresia - Early Detection/Newborn Screening

Facilitators: Dr. Sanjiv Harpavat/Dr. Tebyan Rabbani, Dr. Saeed Mohammad

Guest Presenter: Ashley Upton - A New Approach to BA Diagnostics (Conferencee Room II)

5:45 pm DISMISS for the evening. Dinner on your own. Please refer to your Summit booklet for local

restaurants within walking distance.

**Please pick up/sign out children from Kids Camp by 6:00pm.