Charles River Teams Up with Elly’s Team to Accelerate Gene Therapy for Ultra-Rare NEDAMSS Disorder
Charles River Laboratories International, Inc. (NYSE: CRL), a global leader in early-stage drug development and manufacturing services, has announced a collaborative agreement with Elly’s Team, a parent-led foundation devoted to finding a cure for a devastating rare genetic disorder known as Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS). The partnership centers around Charles River’s role as a contract development and manufacturing organization (CDMO) for plasmid DNA, a critical component in gene therapy development.
This collaboration falls under Charles River’s Cell and Gene Therapy (CGT) Accelerator Program (CAP), a platform designed to support small and emerging biopharma organizations in the development of advanced therapies. Through CAP, Elly’s Team gained access to Charles River’s world-class CDMO capabilities and strategic advisory services, which enabled the rapid production of essential gene therapy materials in preparation for a Phase I clinical trial.
A Deeply Personal Mission
Elly’s Team was founded in 2024 by Michelle and Dan Krueger, parents of a young girl named Elly who was diagnosed with NEDAMSS, a disorder caused by mutations in the IRF2BPL gene. The disease affects fewer than 150 individuals worldwide, making it one of the most ultra-rare conditions in the world. NEDAMSS is characterized by severe neurodevelopmental regression, including the loss of motor abilities, speech, vision, and independent eating, as well as frequent seizures and abnormal movements. Due to the lack of existing treatments, families like the Kruegers are left with few options, motivating them to spearhead efforts that could lead to a potential cure.
“Our mission from the start has been clear: translate emerging scientific knowledge into real treatments for children like Elly as quickly as humanly possible,” said Michelle Krueger, Co-Founder of Elly’s Team. “Charles River’s support and partnership were instrumental in helping us achieve that mission.”
From Diagnosis to Treatment in Just Over a Year
In the face of Elly’s dire prognosis, her parents wasted no time. They quickly assembled a team of leading experts in gene therapy, clinical research, and regulatory affairs, and began funding pivotal studies to understand the biology of NEDAMSS and develop a therapeutic strategy. Elly’s Team pushed forward with multiple workstreams in parallel: conducting preclinical safety and toxicity studies, navigating regulatory pathways, and initiating the manufacturing of the gene therapy candidate.
Charles River’s contribution was central to the rapid development timeline. Through the CGT Accelerator Program, the foundation was able to leverage Charles River’s extensive plasmid DNA manufacturing infrastructure and expertise. Using the company’s eXpDNA™ plasmid platform, which allows for rapid, phase-appropriate production of High Quality (HQ) plasmids following good manufacturing practice (GMP) principles, Elly’s Team was able to secure the critical starting materials needed for regulatory submission.
This expedited development pathway culminated in a significant milestone. In March 2025, the U.S. Food and Drug Administration (FDA) granted Investigational New Drug (IND) clearance to the program. Just weeks later, on April 3, 2025—only 13 months after her diagnosis—Elly became the first child in the world to receive IRF2BPL gene replacement therapy.
“This isn’t just a personal triumph—it’s a breakthrough for the entire NEDAMSS community,” Michelle Krueger said. “Now, when another family receives the same diagnosis, they will have a glimmer of hope. Their physician will be able to say, ‘There is a path forward.’”
Streamlined Manufacturing Through Innovative Platforms
One of the defining aspects of the collaboration is the use of Charles River’s off-the-shelf adeno-associated virus (AAV) Rep/Cap plasmids, designed to streamline gene therapy development. These ready-to-use plasmids offer significant time and resource savings—reducing production complexity and shortening timelines by up to 66%.
Each plasmid product is developed under strict chemistry, manufacturing, and controls (CMC) guidelines and is accompanied by a Certificate of Analysis (COA). This enables regulatory submissions, such as INDs and Clinical Trial Applications (CTAs), to proceed with greater speed and confidence.
By integrating these advanced plasmid solutions into Elly’s gene therapy program, Charles River not only helped accelerate timelines but also contributed to the scientific and regulatory credibility of the project.
“Supporting Elly’s Team aligns perfectly with our mission to help advance potentially life-saving therapies for patients with no treatment options,” said Kerstin Dolph, Corporate Senior Vice President of Global Manufacturing at Charles River. “It has been a privilege to partner with such a passionate, determined organization. We are proud to play a part in their journey.”
A Broader Commitment to Cell and Gene Therapies
Charles River has made significant investments in recent years to enhance its capabilities in the cell and gene therapy space. Through multiple strategic acquisitions and internal expansion efforts, the company has built a robust portfolio of services encompassing plasmid DNA, viral vectors, and cell therapies.
By combining these manufacturing services with its long-standing expertise in drug discovery, safety assessment, and biological testing, Charles River now offers a comprehensive “concept to cure” model that aims to support biopharma innovators from bench to bedside.
This full-spectrum offering is particularly valuable for rare disease foundations and smaller biotech firms that often face barriers accessing scalable manufacturing and regulatory support. Programs like the CGT Accelerator are specifically designed to help these groups overcome resource limitations and accelerate clinical readiness.
A Beacon of Hope for Ultra-Rare Diseases
The collaboration between Charles River and Elly’s Team is more than just a scientific partnership—it’s a story of resilience, hope, and the power of collaboration to drive tangible change for underserved patient communities. By navigating the complex path of gene therapy development in record time, this partnership has not only delivered a potentially transformative treatment to Elly but also opened the door for future patients with NEDAMSS and similar ultra-rare diseases.
Looking forward, Elly’s Team continues to advocate for broader access, additional research, and long-term support for the IRF2BPL community. Meanwhile, Charles River remains committed to leveraging its global infrastructure and cutting-edge technologies to bring more gene therapies to patients who need them the most.
“In every sense, this collaboration exemplifies the future of rare disease research,” said Dolph. “It’s driven by science, fueled by compassion, and grounded in the belief that no patient should be left behind—no matter how rare their condition.”
About Charles River Laboratories
Charles River Laboratories International, Inc. provides essential products and services to help pharmaceutical and biotechnology companies, government agencies, and academic institutions around the globe accelerate their research and drug development efforts. The company supports clients from discovery through preclinical development and into advanced therapy manufacturing.
About Elly’s Team
Elly’s Team is a parent-led nonprofit foundation established in 2024 with the mission of developing treatments for NEDAMSS, a devastating genetic disorder caused by mutations in the IRF2BPL gene. Through partnerships, scientific collaboration, and community engagement, the organization is working to build a brighter future for children diagnosed with this ultra-rare disease.
