The workshop was composed of several informative sessions focusing on topics such as pediatric liver disease, the regulatory approach to rare and orphan diseases, liver fibrosis and biopsy, ideal target and current pipeline of Alpha-1 Antitrypsin Deficiency (AATD) liver drugs, and more. The program included presentations followed by open discussion. There was a wide range of attendees, including researchers, clinicians, government representatives, patients, donors, industry, biotech, and academia.
Click here to view 23rd GLS Program Book
“It was inspiring to see the different experts putting their knowledge together to foster the development of therapeutics for alpha1-antitrypsin-associated liver disease,” said Dr. Strnad.
The outcome of this workshop will be a white paper and our proposed collaboration with the regulatory agencies on the issuance of guidance. By bringing together a group of leading experts, we will discuss the use of liver histology and non-invasive markers for clinical trial endpoints.
“It is exciting to see new liver therapies are being developed to address this unmet medical need, and I know the alpha-1 community will rise to the challenge,” said Dr. Clark.
The agenda included presentations from experts in the field including:
Mark Brantly, MD– University of Florida College of Medicine
Manal Abelmalek, MD, MPH– Duke University School of Medicine
Nicola Brunetti-Pierri, MD– Telethon Institute of Genetics and Medicine
Virginia Clark, MD, MS- University of Florida
James Irving, PhD- University College London
Rohit Loomba, MD, MHSc- University of California San Diego
Don Rockey, MD- Medical University of South Carolina
Michael Schilsky, MD, FAASLD– Yale University
Jeffrey Teckman, MD– Saint Louis University School of Medicine
The Alpha-1 Foundation is aware that there are many questions outstanding with regard to the development of drug therapy for alpha-1 related liver disease. With exciting new developments in therapies, we intend to have future workshops that focus on new developments and innovative procedures for liver disease. The Foundation has requested permission from the speakers to present their videos publicly after the conference and they will be available for viewing on the Foundation’s website.
“The 23rd GLS was a great success,” concluded Dr. Brantly, Alpha-1 Foundation Scientific Director. “Outstanding speakers and the co-chairs brought into sharp focus both challenges and pathways to drug approval in a new era of drugs to treat Alpha-1-Antitrypsin Deficiency related liver disease.”
Thank you our 23rd GLS Workshop Sponsors: AlphaNet, CSL Behring, Grifols, Takeda