About

The Amyloidosis Forum

Formed in 2019, The Amyloidosis Forum has been developed through a public-private partnership between the Amyloidosis Research Consortium (ARC) and the US Food and Drug Administration (FDA). The overarching goal of the Forum is to optimize innovation and accelerate the advancement of new therapeutics by addressing scientific gaps and needs through the conduct of mutually beneficial scientific activities in the pre-competitive domain.

Public Private Partnership with FDA

PPPs and Precompetitive Research

A public–private partnership (PPP) is a cooperative arrangement between two or more public and private sectors, typically of a long-term nature. In 2019, a PPP was formed between the nonprofit Amyloidosis Research Consortium (ARC) and the US Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER). The goal of this PPP is to identify and bridge the scientific gaps that are acting as barriers to drug discovery and development for the treatment of AL amyloidosis. The PPP seeks to leverage expertise and resources of all stakeholders (academia, industry, patients, and regulatory agencies) for the conduct of mutually beneficial scientific activities in the precompetitive domain to support bringing new, safe and efficacious therapies to patients with AL and ATTR amyloidosis.

Steering Committee

Angela Dispenzieri, MD

Consultant, Division of Hematology; Serene M. and Frances C. Durling Professor of Medicine and of Laboratory Medicine and Pathology; Hematology Research Chair, Mayo Clinic College of Medicine

Isabelle Lousada

Founder and CEO, Amyloidosis Research Consortium

James Signorovitch, PhD

Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.

Kristen Hsu

Executive Director of Research, Amyloidosis Research Consortium

Mathew S. Maurer, MD

Cardiologist and Medical Director, The HCM Center at New York-Presbyterian Hospital/Columbia University Medical Center

Mona Fiuzat, Pharm D

Senior Scientific Advisor, FDA; Associate Prof. of Medicine, Duke

Preston Dunnmon, MD, MBA, FACP, FACC

Vice President, Data Science, Cardiovascular/Metabolism & Pulmonary Hypertension, Janssen Research and Development (Retired)

Sarah Cairns-Smith, PhD, MBA

Chairman, Amyloidosis Research Consortium; Senior Partner and Managing Director, Boston Consulting Group

Sharmila Dorbala, MD, MPH

Director of Nuclear Cardiology, Brigham and Women’s Hospital

Vaishali Sanchorawala, MD

Professor of Medicine. Director, Amyloidosis Center, Boston University School of Medicine and Boston Medical Center

Subcommittees

Aliza Thompson, FDA Division of Cardiology and Nephrology
Andrea Baines, FDA Division of Hematologic Malignancies
August Hofling, FDA Division of Imaging and Radiation Medicine
Austin Hu, FDA Division of Cardiology and Nephrology
Charu Gandotra, FDA Division of Cardiology and Nephrology
Cynthia Welsh, FDA Division of Rare Diseases and Medical Genetics
Jenni Li, FDA Office of Surveillance and Epidemiology
Laura Jawidzik, FDA Division of Neurology 1
Matthew Kowalik, FDA Division of Gastroenterology
Mona Fiuzat, FDA CDER Patient Focused Drug Development
Sarrit Kovacs, FDA Division of Gastroenterology
Shuichi Kawarasaki, PMDA (Japan)
Steven Bai, FDA Office of Biostatistics

Adam Castaño, BridgeBio
Ansgar Conrad, Prothena
Brent Warner, Protego
Cristina Quarta, Alexion
Jersey Chen, AstraZeneca
John Vest, Alnylam
Jovian Yu, AbbVie
Lori Baylor, Pfizer
Michelle Lim-Watson, NovoNordisk
Preston Dunnmon, Janssen (Retired)
Ricardo Rocha, Intellia
Spencer Guthrie, Attralus
Teresia Moller, Life Molecular Imaging

James Signorovitch, Analysis Group

Collaborators

AbbVie Inc
Abramson Cancer Center of the University of Pennsylvania
Alnylam Pharmaceuticals
Analysis Group
AstraZeneca
Attralus
Boston Medical Center
BridgeBio Pharma, Inc
Brigham and Women’s Hospital
Cedars-Sinai Medical Center
Cleveland Clinic
Cleveland Clinic Florida
Columbia University
Froedtert and Medical College of Wisconsin
Hospital Universitario Puerta de Hierro-Majadahonda
Intellia Therapeutics
International Society of Amyloidosis
Janssen Pharmaceuticals
Jefferson Health
Johns Hopkins
Mayo Clinic
Mayo Clinic Arizona
Medical University of South Carolina
National Amyloidosis Centre, University College of London
National and Kapodistrian University of Athens
Novo Nordisk
Oregon Health & Science University
Pfizer
Pharmaceuticals and Medical Devices Agency (Japan)
Protego Biopharma, Inc.
Prothena Biosciences
Quality Metric
Royal Free London NHS Foundation Trust
Stanford University Medical Center
Stanford University School of Medicine
The University of Chicago Medicine
Tufts Medical Center
Université De Poitiers
University of Miami Sylvester Cancer Center
University of Pavia
University of Pennsylvania
University of Pittsburgh Medical Center
UT Southwestern Medical Center
Vanderbilt University Medical Center

The Amyloidosis Research Consortium

The Amyloidosis Research Consortium (ARC) is a nonprofit organization dedicated to driving advances in the awareness, science, and treatment of amyloid diseases. ARC’s mission is to improve and extend the lives of those with amyloidosis. ARC is committed to collaborative efforts that accelerate the pace of discovery, expand patient access to the most effective care, and improve short- and long-term outcomes. Working with partners in industry, government, and academia, ARC seeks to spark innovation and to bring promising treatments from labs to clinics. ARC’s outreach and education inform and empower patients, families, caregivers, physicians, and researchers.

Visit the ARC Website