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

Thank You to Our Supporters

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