Featured News
Joining Elly’s Team to Drive Rare Disease Drug Development
Supporting Fast Track Gene Therapy Production Targeting Ultra-Rare Neurodevelopmental Disease
Founded in 2024 by Elly's parents, Michelle and Dan Krueger, Elly's Team has a singular goal of translating medical research to treatment for Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS) as quickly as possible. An ultra-rare disease caused by mutations in the interferon regulatory factor 2 binding protein-like (IRF2BPL) gene, fewer than 150 cases have been diagnosed worldwide.
The regressive neurodevelopmental disorder affects the central nervous system and can impact motor skills, speech, eating, and eyesight, among other functions, and can often cause seizures.
Your Guide to Plasmid DNA from Concept to Cure
Explore challenges and considerations to future-proof your program from plasmid selection in drug discovery, navigating the translation to the clinic, and commercialization.
Access Resource
Translating Research to Treatment in Record Time
Having rapidly assembled experts in gene therapy and drug discovery, Elly's Team was able to advance multiple steps simultaneously to shorten timelines. This included funding additional research to understand the disease mechanism and effects on the body, conducting safety and toxicity studies, and simultaneously manufacturing the drug.
The team did this while also navigating the FDA approval process, to get Investigational New Drug (IND) approval to dose Elly in early April 2025, just 13 months after her diagnosis.
"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," stated Michelle Kruger, Elly's mother and Co-Founder, Elly's Team, in a press release announcing the partnership. "Signed off by the FDA in March 2025, on April 3, 2025, Elly became the first child to receive an IRF2BPL gene replacement therapy. 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."
Because Every Moment Matters
Striving to find a cure for this rare genetic disorder, Elly’s Team participated in the Charles River Accelerator Program (CAP) to fast-track the manufacture of critical starting materials for a Phase I clinical trial.
"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. We are honored to work hand in hand with the team as they navigate the next stage of their program development," commented Kerstin Dolph, CSVP, Global Manufacturing, Charles River.
Leveraging the eXpDNA™ plasmid platform to expedite High Quality (HQ) plasmid production, alongside off-the-shelf AAV Rep/Cap plasmids designed to streamline adeno-associated virus (AAV)-based gene therapy programs, the team was able to reduce production time and improve efficacy of the supply chain, ultimately reducing manufacturing efforts by up to 66%.
The ready-to-use plasmid products are manufactured and released with CMC guidance and according to batch production records, with a Certificate of Analysis (COA) to support IND and Clinical Trial Application (CTA) filings.
For more information on our integrated plasmid DNA, viral vector, and cell therapy development and manufacturing platforms and services, please visit Cell and Gene Therapy CDMO Solutions.