In just eight years, the NIH National Institute of Allergy and Infectious Diseases moved from identifying an ultra-rare genetic disorder, CD55 deficiency, hyperactivation of the complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) to FDA approval of its first treatment option. CHAPLE is an ultra-rare disease affecting fewer than 100 known people worldwide. Patients with CHAPLE disease have two defective copies of the CD55 gene, and the deadly attack complex that ordinarily targets invaders begins to attack the body’s healthy tissues, leading to a cascade of life-threatening health problems.

In 2020, NIAID entered a CRADA with Regeneron to study the safety and efficacy of Regeneron’s proprietary compound pozelimab (REGN 3918) in a Phase II/III trial in patients with CHAPLE. In August 2023, Veopoz™ (pozelimab-bbfg) became the first FDA-approved treatment for CHAPLE disease.

Dr. Michael Lenardo of NIAID, lead inventor on the CHAPLE technology, noted that most individuals diagnosed with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy. “I saw first-hand the transformational clinical improvement that pozelimab achieves in those [living with] CHAPLE. The approval of pozelimab is a milestone to celebrate.”

This technology transfer success story serves as an inspiration—and model—for future initiatives aimed at tackling rare and complex health challenges through meticulous research and strategic public/private partnerships. 

The Federal Laboratory Consortium (FLC) recognized this when awarding NIAID with a 2025 Technology Transfer Impact Award. This award honors FLC member labs whose technology transfer efforts have made a tangible lasting impact on the populace or marketplace ranging from a local to a global scale. Congratulations to both the research/clinical team formerly led by Dr. Michael Lenardo and the team of TTIPO professionals managing the technology portfolio Yogikala Prabhu, PhD; Cecilia Pazman, PhD (formerly at TTIPO); Cosimo Fuda, JD, PhD; and former Branch Chief Richard Williams, PhD.

The CHAPLE Disease Clinical Trial Team also received a 2024 NIH Director’s Award for their exceptional work.