Charles River Partners with Elly’s Team to Manufacture Plasmid DNA for Rare Disease Trial

• Charles River and Elly’s Team have entered a CDMO agreement to manufacture plasmid DNA for a Phase I gene therapy trial targeting NEDAMSS.

• The treatment was delivered to the first patient, Elly, only 13 months after diagnosis, with support from Charles River’s CGT Accelerator Program.

Charles River Laboratories International, Inc. and Elly’s Team, a foundation formed by parents of a child with an ultra-rare genetic condition, have entered into a contract manufacturing agreement to support the production of plasmid DNA for a Phase I gene therapy trial. The partnership was announced as part of Charles River’s Cell and Gene Therapy (CGT) Accelerator Program (CAP), which provides access to its gene therapy CDMO capabilities.

Founded in 2024, Elly’s Team was created by Michelle and Dan Krueger with the aim of accelerating treatment development for Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS). The disease is caused by mutations in the IRF2BPL gene and affects fewer than 150 individuals worldwide. With support from Charles River, the team advanced multiple development steps concurrently to deliver a gene therapy dose just 13 months after Elly’s diagnosis.

The therapy was officially administered to Elly on 3 April 2025, shortly after FDA approval in March. “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, Co-Founder of Elly’s Team.

Charles River contributed through its plasmid platform, eXpDNA™, delivering high-quality plasmid DNA with rapid turnaround. The team also used Charles River’s off-the-shelf AAV Rep/Cap plasmids to streamline manufacturing and reduce production efforts by up to 66 percent, according to the press release.