Featured News
Rare Disease Patient Spotlight: FOXG1 Research Foundation
A Rare Disease Community Rewriting What's Possible for Their Children
During Rare Disease Month, families from across the FOXG1 community gathered at our Viral Vector CDMO Center of Excellence in Rockville, MD, to share their stories and to witness something extraordinary: the manufacturing of a gene therapy designed for their own children. It was a day filled with gratitude, vulnerability, and a shared belief that together, families, foundations, and scientists are reshaping the future of rare disease.
Beth Tebeau, Site Director, captured the significance of the moment in her opening remarks: "Having the opportunity to interact with families and the foundation is not something that happens every day for us…which makes today even more special."
A Decade of Determination
For Nasha Fitter, cofounder and CEO of the FOXG1 Research Foundation and mother to ten-year-old Amara, standing inside the facility was almost indescribable: "It's hard to explain what it feels like to be at the place where a drug is being made that is going to go into our own children’s brains."
Eight years earlier, when Nasha and Nicole Johnson, fellow co-founder and mother to Josie, first received their daughters' diagnoses, FOXG1 syndrome was barely understood. There were no models, no natural history studies, and little consensus on symptoms.
Parents stepped in where industry could not, and through sheer persistence and a fierce refusal to accept the standard pace of rare disease drug development, they built a path to a treatment from scratch.
Cooper's Story: A Mother's Intuition and Search for Answers
For Mercedes, Cooper's mother, the journey began with concerns no one seemed to take seriously. At three months, Cooper wasn't tracking or meeting milestones, but despite reassurances, mother's intuition told her that something was wrong.
After months of pushing for evaluations, genetic testing revealed FOXG1 syndrome, but information was scarce. Mercedes recalled finding a page on social media, and Nicole immediately reaching out to suggest a video call, offering comfort and mutual understanding.
Through years of seizures, hospital trips, therapies, and hard-won progress, Cooper's spirit has never faded.
"Everything that Nasha and Nicole have done is amazing; we're so thankful for everybody on our team who works hard every single day for these children, some of them they never even met. They're busting their butts for them, and I'm so thankful for that."
Gianna's Story: Finding Answers and a Community
For Courtney and Zach, parents to six-year-old Gianna, early signs were subtle but worrisome: no social smile, a head circumference below the first percentile, missed milestones. Courtney, a doctor herself, was overwhelmed by uncertainty and fear.
COVID lockdowns exacerbated their isolation, stretching long nights of inconsolable crying into months. Then came the moment of clarity: a long-awaited genetic result confirming FOXG1. Courtney remarked, "That day was the happiest day of my life since she was born. We finally had an answer."
Within 24 hours, Nicole got in touch, and they gained a new FOXG1 family, one they didn't know they needed, which welcomed them in immediately.
"We owe a great debt of gratitude to everyone at the Foundation who has poured their lives and souls into making this gene therapy a reality this year against all odds…and on top of this incredible parent-led team, we are especially grateful to all of you at Charles River who are working tirelessly to make this gene therapy possible in 2026."
FOXG1 Case Study Webinar
Join Dr. Gai Ayalon, Chief Drug Development Officer, FOXG1 Research Foundation, and James Cody as they discuss AAV Production for Rare Disease Therapies: From Challenge to Solution.
Watch Here
Working Hand-in-Hand to Make History
FOXG1 Research Foundation has built models, run tox studies, advanced manufacturing, and secured FDA Fast Track designation, helping to cross the notorious clinical "valley of death" by creating a new economic and scientific model, one driven not by profit, but by parents unwilling to wait.
Behind the emotional stories lies something unprecedented in rare disease: FOXG1 is on the verge of becoming the first patient-led global gene therapy trial in history.
Gai Ayalon, Chief Drug Development Officer, described the essence of our partnership: "Our return on investment is lives…We needed a partner who cared, and Charles River is basically part of the team. We are operating as one team."
This alignment of purpose, with parents and scientists working together as equals, enabled the program to accelerate at an unprecedented pace.
Meeting with families face-to-face, the Rockville team was honored to see firsthand the meaning behind their meticulous work, and Ahmad Hussin, VP of CDMO, offered a poignant reminder: "Behind every task, there's a family counting on us to deliver."
Parents echoed that sentiment; Courtney spoke of dreaming not of extraordinary achievements for Gianna, but of ordinary childhood moments, and Mercedes reflected on waking each morning to her son's smile.
This is what rare disease innovation looks like, not just cutting-edge science, but families, foundations, and partners joining forces to rewrite what's possible for children who have been waiting far too long.
FOXG1 Research Foundation CEO Nasha Fitter shares milestone updates: FDA Fast Track, IND clearance, and the first FOXG1 gene therapy trial now in reach.
Listen to the Podcast
Bringing Life-Changing Treatments to Patients, Faster
As a cell and gene therapy CDMO committed to serving clients globally, our team has partnered with the FOXG1 Research Foundation since 2024, providing scientific guidance, alongside critical plasmid starting materials and AAV to support this groundbreaking program.
Did you know? The nAAVigation® AAV production platform accelerates the development of viral vector gene therapies, reducing a program’s timeline to GMP by up to 55% compared to traditional AAV manufacturing workflows.
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.