WILMINGTON, Mass. — Charles River Laboratories (NYSE: CRL) has announced a partnership with Elly’s Team, a parent-led, faith-based foundation committed to accelerating treatment for the ultra-rare genetic disorder NEDAMSS (Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures).
Under a new contract development and manufacturing organization (CDMO) agreement, Charles River is supporting Elly’s Team through its Cell and Gene Therapy (CGT) Accelerator Program. The collaboration provides the foundation with essential CDMO services and scientific guidance to produce plasmid DNA — a key component for initiating a Phase I clinical trial of a novel gene therapy.
“The opportunity to work with Elly’s Team aligns closely with our core mission: supporting the development of potentially curative treatments for Elly and other patients suffering from ultra-rare diseases with no known treatment,” said Kerstin Dolph, Corporate Senior Vice President of Global Manufacturing at Charles River. “We are honored to work hand in hand with the team as they navigate the next stage of their program development.”
Founded in 2024 by Michelle and Dan Krueger after their daughter Elly was diagnosed with NEDAMSS, the foundation has moved with exceptional speed. Just 13 months after Elly’s diagnosis, the group secured FDA approval to administer the first IRF2BPL gene replacement therapy. Elly received the experimental treatment on April 3, 2025, becoming the first child in the world to undergo this gene therapy targeting the IRF2BPL gene.
NEDAMSS is caused by mutations in the IRF2BPL gene and affects fewer than 150 individuals globally. The disorder impacts the central nervous system and can impair speech, movement, vision, and eating, often leading to seizures and neurological regression.
Through rapid coordination with experts in gene therapy, drug development, and regulatory strategy, Elly’s Team has advanced key milestones in parallel — including basic research, safety studies, drug manufacturing, and regulatory submissions — all with the goal of bringing a treatment to patients as fast as possible.
“We want to express our deepest gratitude to all members of Elly’s Team who made this incredible treatment a reality, including Charles River whose partnership and expertise has undoubtedly contributed to the advancement of our gene therapy program,” said Michelle Krueger, Elly’s mother and Co-Founder of Elly’s Team. “This achievement marks a major milestone, not only for our family but also for the entire IRF2BPL community. In the future, another family will sit in the hospital and receive the same diagnosis, but their doctor will tell them there is a path to treatment.”
