FOXG1 Research Foundation

Our quarterly newsletter is now available! Discover the latest updates from the FOXG1 Research Foundation, including new developments in FOXG1 syndrome gene therapy, worldwide events, and ways to get involved.

NEWS: The FOXG1 Research Foundation partners with Charles River Laboratories to advance its gene therapy program for FOXG1 syndrome. “We are thrilled to partner with Charles River, a leader in the life sciences industry,” said Nasha Fitter, FOXG1 parent, co-founder, and CEO of FRF. “Their expertise and commitment to our cause align perfectly with our goal of developing life-changing treatments for children with FOXG1 syndrome.” By combining FRF’s deep understanding of FOXG1 syndrome with Charles River’s CDMO capabilities, the partnership aims to expedite the development of gene therapy and bring hope to families affected by FOXG1 syndrome. Read the full press release: https://lnkd.in/ecRResMP #genetherapy #foxg1 #foxg1syndrome

Taking a new approach to drive rare disease drug development, patient advocacy group FOXG1 Research Foundation is collaborating with our CDMO team to advance its gene therapy program. “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,” Nasha Fitter, FOXG1 parent, co-founder, and CEO. Read the press release in full: https://okt.to/KetnUO #genetherapy #concepttocure #raredisease #foxg1syndrome

As the parent of a child with FOXG1 Syndrome, Nasha Fitter is leading the charge to find a treatment for her daughter and other children with ultra-rare diseases. Learn more about the FOXG1 Research Foundation and how it wants to “give kids the life they deserve” in the season five finale of Vital Science. https://okt.to/QHhyXV

Our FOXG1 Research Foundation gene therapy program progresses: We had a highly productive INTERACT meeting with the FDA resulting in a streamlined and efficient nonclinical path to IND. We look forward to our continued collaborative dialog with FDA and work toward our pre-IND meeting. We will continue to document all our learnings into a blueprint for other patient foundations running drug development to learn from! #curefoxg1 #RareAsOne

📣 California families! Please take two minutes to read this post and sign our petition to ensure the California Institute for Regenerative Medicine (CIRM) continues to fund research into rare disease treatments for FOXG1 syndrome and others. CIRM is one of the only funders of rare disease research and without their funding, more than 3 million Californians (more than 50% of them kids) could be left without treatments and without hope. CIRM is currently evaluating its funding priorities ahead of their board meeting on June 27. We urgently need your help NOW to ensure that they continue to fund rare disease research. Please click on the link to sign our petition. 🔗 https://lnkd.in/etRgCEKy Thank you for helping us to improve and save lives!

New Paper: Groundbreaking Results in AVV9-FOXG1 Gene Therapy Studies A new landmark study is out today in Molecular Therapy Methods & Clinical Development. Led by our FOXG1 Research Center’s Dr. Soo-Kyung Lee, Dr. Jae. Lee, and in collaboration with Dr. Kathrin Meyer who is responsible for the successful SMA gene therapy, this study shows remarkable rescue of brain abnormalities using AAV9-FOXG1 gene therapy. In summary: FOXG1 syndrome results in structural abnormalities like missing parts of the brain's connecting bridge, smaller hippocampus, and delayed nerve fiber coating. After injecting AAV9-FOXG1 into the brains of young mice with FOXG1 mutations, they observed significant improvements included: fixing the corpus callosum deficiencies, restoring the structure of the dentate gyrus in the hippocampus, normalizing the number of certain brain cells, and correcting issues with myelination. This breakthrough study suggests that AAV9-based gene therapy can be a promising treatment for FOXG1 syndrome and other similar brain disorders, especially when applied after birth. As next steps, the FOXG1 Research Foundation will be testing the safety of this gene therapy in larger animals before proceeding to human clinical trials. Thank you Soo-Kyung Lee Jae Lee Kathrin Meyer for your work. Stay tuned for further updates! #foxg1research #foxg1 #genetherapy #aav9 #keypaper

I thoroughly enjoyed discussing payment models for ultra-rare diseases with Vice President at CIRM, Abla Creasey, CEO of Rarity PBC, Paul Ayoub and Vice Chairman at H.C. Wainwright & Co. Michael E. Meyers at #LABEST2024. Unfortunately our funding issues with ultra-rare is not just tied to the 'valley of death' but once we are lucky enough to get a gene therapy to market, insurers actually agreeing to pay. How in general will the system (made of taxpayers) support even the conservative estimate of $25.3 billion in gene therapy costs by 2026? It was a great conversation around milestone and value-based reimbursement, the role of better endpoints to measure long-term efficacy and the role of patients in reporting long term outcomes. I'm excited about the role of platforms like Citizen Health to follow rare disease patients who receive gene therapies for their entire lives to showcase the transformative nature of these drugs. FOXG1 Research Foundation #RareasOne

Please consider joining us on Tuesday, June 11th at The Sultan Room for a killer night of music featuring Summerfeet to raise money and awareness for a great cause, the FOXG1 Research Foundation. Hope to see some of your there. Link to the event is below. https://lnkd.in/gUgzb58s

Nasha Fitter will be a featured speaker at the upcoming WCG Patient Forum Focus Session, titled "Voices Transforming the Science and Practice of Inclusive Clinical Care and Research”. The session will explore: - Mentoring clinical caregivers and community members to build a more diverse workforce. - Collaboration to drive a new era of rare disease research through endpoint validation and accelerated approval pathways. - The power of parent-driven preclinical research. - Identifying and overcoming access barriers. Learn more and register today! Link: https://lnkd.in/guZUNwaX