C-Path Welcomes New Advisory Members to Alpha-1 Antitrypsin Deficiency Consortium
C-Path's Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-1) consortium today announced the addition of several...
Founded in 2023, CPA-1 is a public-private partnership comprised of industry partners, academic researchers, regulators, and patient groups working collaboratively to accelerate medical advancements for Alpha-1 Antitrypsin Deficiency (AATD).
AATD silently impacts an estimated 1 in 1,500 to 3,500 individuals of European descent worldwide. In fact, up to 100,000 people in the U.S. alone are affected by this condition. It often manifests as chronic obstructive pulmonary disease at approximately 40 years of age. Infants aren’t spared either; nearly 10% of infants with AATD face abnormal liver functions, posing a threat of future transplants. The intricate genetic facets of AATD cloud our understanding of patient experiences and the nuances of clinical trials. Given these statistics and complexities, there’s an undeniable urgency to recalibrate our approach.
CPA-1 generates solutions for unmet drug development needs in AATD, with a specific focus on addressing its hepatic and pulmonary manifestations. This will be achieved through the collaborative creation of an aggregated database, integrating data from clinical trials, natural history studies, registries, and other relevant sources to inform and support these solutions.
The consortium’s mission is to forge a resilient public-private partnership comprised of industry partners, academic researchers, regulators, and patient groups. Our goal is to work collaboratively to accelerate medical advancements for AATD, ensuring better lives for people affected by AATD.
CPA-1 unites key stakeholders in the AATD sphere. Within this pre-competitive space, CPA-1 fosters alignment among AATD stakeholders, identifying challenges, prioritizing solutions, and forging innovative tools. This new alignment is the catalyst for pioneering new drug development tools tailored to meet the pressing needs of those living with AATD.
CPA-1 embarks on a journey to cultivate a comprehensive knowledge base for AATD, with a focus on achieving the following milestones:
In the realm of AATD, unmet needs and opportunities abound that the CPA-1 consortium will evaluate and prepare regulatory-ready solutions with the hope of:
The journey towards conquering Alpha-1 Antitrypsin Deficiency has been fraught with obstacles, but through CPA-1’s collaborative spirit and unwavering dedication, we aspire to remove obstacles and enhance drug development to improve lives — together.
Alpha-1 Foundation
COPD Foundation
Global Liver Institute
Jon Hagerstrom
Board Member, Alpha-1 Foundation
Professor Noel G. McElvaney
Royal College of Surgeons in Ireland
Marc Miravitlles, MD
Pulmonologist at the Pneumology Department, University Hospital Vall d’Hebron / Vall d’Hebron Research Institute in Barcelona, Spain
Jeff Teckman, MD
Pediatric Hepatologist at Saint Louis University
Alice Turner, MBChB, MRCP, PGCE, PhD
Professor of Respiratory Medicine, University of Birmingham, United Kingdom
Hugo E. Vargas, MD
Professor of Medicine, Mayo Clinic
Andrew A. Wilson, MD
Professor of Medicine and Director of The Alpha-1 Center, Boston University and Boston Medical Center