FOXG1 Patient Day at Charles River Laboratories – Rockville CDMO

On February 25, 2026, during Rare Disease Week, our FOXG1 Research Foundation leadership team joined local FOXG1 families at the Rockville, Maryland CDMO site of Charles River Laboratories — the team manufacturing FRF-001— the gene therapy our children will soon receive in clinical trials.

The experience was meaningful beyond words.

We arrived during what the team calls “a run” — the active, month-long manufacturing process for our AAV gene therapy. By extraordinary timing, we were able to witness our actual gene therapy being produced.

Walking through the facility, seeing the layers of safeguards, the precision environments, and the rigorous documentation required at every step made this moment feel very real for our families.

We saw firsthand the expertise and dedication behind plasmid production and adeno-associated viral (AAV) vector manufacturing that power our program. Every quality check. Every controlled space. Every carefully monitored step.

All driven by a shared commitment to children with FOXG1 syndrome.

What made the day even more powerful was the human connection.

Two families shared their FOXG1 stories — the similar long diagnostic journeys, the daily realities, the urgency we live with. In return, we saw the deep purpose behind the scientists and manufacturing teams who show up each day to build something that has never existed before.

This is what patient-led drug development looks like.

Families and scientists, side by side.
Urgency matched with excellence.
Stories fueling science.

For years, we have said that we are building a new path — advancing a gene therapy through patient clinical trials independently, as a parent-led foundation. Standing inside the facility where that therapy is being manufactured brought that path into sharp focus.

This is no longer theoretical.
It is tangible.
It is underway.

We are deeply grateful to the Rockville CDMO team for welcoming our families and for the extraordinary care being put into every vial.

And to our FOXG1 families: this is what your advocacy, your fundraising, and your refusal to accept “no” has made possible.

This is parent-led drug development in action.