A New Approach to Drive Rare Disease Drug Development

Founded in 2017 and propelled by innovation and an urgency to accelerate the road to therapeutics for FOXG1 syndrome, FOXG1 Research Foundation (FRF) has built a replicable model for rare disease patient advocacy groups. Incorporating pioneering novel AI platforms for patient data to streamline preclinical work, the platform empowers others to take control and drive the development of treatments when no other options exist.

Highlighting the FRF's model to independently drive rare disease drug development through the clinical phase, we recently announced a comprehensive gene therapy contract development and manufacturing organization (CDMO) agreement, inclusive of critical plasmid starting materials and adeno-associated viral vectors (AAV) to advance the research to cure FOXG1 syndrome and related neurological disorders.

"Given the limited investment for rare disease groups like ours, our foundation has created a model that allows us and other patient advocacy groups to operate like a virtual biotech company and independently and efficiently drive drug development," commented Nasha Fitter, FOXG1 Parent, Co-founder, and Chief Executive Officer, FOXG1 Research Foundation.

"The success of FRF's model is not only focused on operating as a highly efficient team, but also partnering with organizations that are equally passionate about bringing treatments to children with the highest unmet need and severe burden of disease. Selecting the right partner is critical and Charles River has demonstrated a deep understanding of our model and commitment to working on our therapies."

What is FOXG1 Syndrome?

FOXG1 syndrome is a rare neurological genetic disorder caused by a mutation in the FOXG1 gene. Linked to autism spectrum disorder (ASD), it significantly impacts brain development and typically causes both epilepsy and physical and cognitive disabilities, and most children with FOXG1 syndrome cannot walk, talk, or take care of their basic needs.

Mutations to the FOXG1 gene are mostly non-inherited and there are currently around 1,000 known diagnoses but no treatments - yet. FRF is dedicated to advancing treatments for FOXG1 syndrome and related disorders while advocating for and supporting patients and families worldwide.

Gene Therapy Manufacturing

Through the collaboration, our CDMO team will provide FRF access to extensive cell and gene therapy expertise and established plasmid and AAV production platforms, generating materials required for the foundation's Phase I-II adeno-associated viral (AAV) vector-based gene therapy clinical trials.

Produced at our plasmid DNA and viral vector CDMO centers of excellence (CoE), we will supply phase-appropriate High Quality (HQ) plasmid starting materials in addition to good manufacturing practice (GMP) AAV9 viral vectors, leveraging an integrated manufacturing and biologics testing portfolio to streamline the path to the clinic.

"Charles River is proud to work with the FOXG1 Research Foundation to advance its gene therapy through clinical trials," said Kerstin Dolph, CSVP, Global Manufacturing, Charles River. "The FOXG1 patient population has an incredible unmet need, and we are looking forward to lending our expertise to FRF as they continue to trailblaze a path toward providing rare disease treatments."

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 .

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