NIAID Team Honored for First FDA-Approved Drug for Ultra-Rare Genetic Disease
NIH’s National Institute of Allergy and Infectious Diseases (NIAID) recently received a 2025 Impact Award from the Federal Laboratory Consortium (FLC). The award honors laboratories whose technology transfer efforts have made a tangible, lasting impact on the populace or marketplace.
The award honors NIAID researchers who, in 2016, were the first to identify CHAPLE disease—a rare genetic disorder leading to deficiency in the complement protein CD55. In 2024, coordinated technology transfer efforts between the public and private sectors have resulted in FDA approval of the first treatment option for the disease, Pozelimab (Veopoz®) by Regeneron.
Dr. Michael Lenardo, former chief of molecular development at NIAID’s Laboratory for Immune System Biology and lead inventor on the new technology, noted that most individuals diagnosed with CHAPLE disease are children who face severely debilitating symptoms and life-threatening complications.
“I saw firsthand the transformational clinical improvement that pozelimab achieves in those [living with] CHAPLE,” he said. “The approval of pozelimab is a milestone to celebrate.”
Following initial discovery of the disease by Lenardo’s lab in 2017, multidisciplinary experts at the Technology Transfer and Intellectual Property Office (TTIPO) worked with the research team to file a patent application covering methods for the diagnosis and treatment of CHAPLE. TTIPO also worked with the team to navigate the next crucial phase of development—a 2019 collaboration with Marmara University to investigate the off-label use of eculizumab in 16 people with CHAPLE.
Although CHAPLE disease affects fewer than 100 known people worldwide, this success story serves as an inspiration—and model—for future initiatives aimed at tackling rare and complex health challenges through meticulous research and strategic partnerships.
Along with the clinical research team led by Lenardo, the dedicated specialists at TTIPO managing the technology portfolio will be formally honored at a special ceremony during the 2025 FLC National Meeting in May.