FOXG1 Research Foundation

Patient Advocacy Groups are ushering in a new model of drug development—and the industry is taking notice. At today’s Charles River Laboratories Cell & Gene Therapy Summit, Gai Ayalon, Chief Drug Development Officer of FOXG1 Research Foundation—and a proud Citizen Health partner—showed how #PAGs are no longer waiting for drug developers to come to them. They are becoming the developers. By breaking down barriers and re-imagining what efficiency looks like, patient groups are combining lived experience with real-world data, natural history studies, and patient-driven registries to accelerate every stage of the process. Gai made one thing clear: If there is no natural history study for your disease, you have to create one. This is why digital natural history studies and platforms designed for patient communities are not just helpful—they are essential. #PatientEntrepreneurs, biotech, and pharma are no longer in separate lanes—they are building the future of drug development together. #CellAndGeneTherapy #AcceleratingCures #PatientDrivenResearch #PatientEntrepreneurs #NaturalHistoryStudies #FOXG1

Our CEO and Co-Founder, Nasha Fitter, was chosen to participate in the SCOPE's keynote panel! SCOPE is the premier conference that is predominantly focused on larger biotech and pharmaceutical companies. Nasha spoke on the FOXG1 Research Foundation's innovative model for drug development and the importance of putting patients at the center. Our chief drug development officer, Gai Ayalon, attended SCOPE as well and engaged in key meetings to drive us along with the FOXG1 gene therapy roadmap. #genetherapy #raredisease #SCOPEsummit

Did you know that FOXG1 is one of the first genes activated during early brain development? This essential gene helps lay the groundwork for how our brains are structured and function. When FOXG1 doesn’t work as it should, it can lead to FOXG1 syndrome, impacting movement, communication, and daily life. Our dedicated researchers are working hard to understand this gene and explore new treatment possibilities. Learn more about our journey and how you can stay informed by visiting our website. #foxg1syndrome #foxg1awareness #foxg1research #foxg1center #raredisease #rarediseaseawareness #geneticresearch

FOXG1 patients - Citizen Health and Global Genes are providing free information sessions to understand your genetics report. It's first come first serve so sign up quickly!

Thank you Eqvista for featuring our cofounder and CEO and the work we are doing to accelerate treatments for FOXG1 syndrome and other rare diseases! #curefoxg1 #founderspotlight

The FRF team is hitting the conference circuit in 2025. As we advance toward clinical trials for the FOXG1 AAV9 gene therapy, we will be meetings with the experts in the area of clinical operations at the SCOPE conference in Orlando. If you’ll be at SCOPE, please let us know and say hello! About Scope: Clinical trials, digital health, and clinical research are essential for advancing medical knowledge, improving patient care, and developing new treatments and therapies for the patients who need them. Execution of this vital work requires collaboration, innovation, and strategic decision-making. Now in its 16th year of fostering these joint efforts to be inclusive of all stakeholders, SCOPE Summit 2025 will take place February 3-6, 2025, in Orlando, FL. https://lnkd.in/e_xbfAX

Japan, Taiwan, Russia, Pakistan, Israel, Poland, France, Italy, Spain, Brazil, Canada and all around the United States. Parents from all over the world flew to Fort Lauderdale, Florida for our FOXG1 Parents Conference November 15-17. There are no words adequate enough to describe how meaningful this weekend was. FOXG1 children and adults, with their parents and siblings joined leading FOXG1 clinicians, scientists, rare disease leaders, and donors to learn - among many things- about the FOXG1 gene therapy that we are on track to bringing to human clinical trials by 2026. Without Pharma stepping in to help us get treatments to our children, we're doing this on our own and our goal is accelerated approvals. While I was on stage in Florida, Josie was in an ambulance without me. She's ok now, but this suffering has to stop - and we know gene therapy is our best chance to give our kids a healthy life. Our preclinical data is incredibly promising—groundbreaking in fact. And our work has far-reaching implications as we're disrupting the model for rare disease drug development - cutting the cost and time in more than half. Josie turned 13 this past Saturday. Please consider clicking this link below and giving her a birthday gift donation towards a treatment that can drastically improve her life. https://lnkd.in/edaEnTdQ Thank you for supporting in any way. We take Crytpo too! https://lnkd.in/eEvFmVQG If there is anyone you know that would consider a major gift, please connect us so I can present our work. Stay tuned for the entire conference on our FOXG1 Research Foundation YouTube channel. Nasha Fitter Effie Parks Soo-Kyung Lee Terry Pirovolakis Ryan Phillips Brianna Fleming Elli Brimble Angela Van Wingerden, DES

Last week, our Chief Clinical Data Officer, Elli Brimble was invited to speak at the invite-only Chan Zuckerberg Initiative Science in Society event. Elli presented the innovative work the FOXG1 Research Foundation has done around utilizing patient data from our registry and Citizen Health to speed up drug development for our gene therapy program. We’re so proud to be at the forefront of innovation in rare disease research! #FOGX1 #GeneTherapy #PatientData #CZI #ScienceInSociety #RareDiseaseResearch

Today's article in Bloomberg (featuring the cutest 12-year-old girl I know) offers an accurate insight into the unfortunate economic landscape for promising gene therapies for rare diseases. It underscores the critical role of philanthropy. There is substantial reward in saving human lives, but not for Wall Street. As Jorge Conde from Andreessen Horowitz aptly states, "It's unfortunate, because the promise here is substantial." Yes, it is substantial, and as a parent-led rare disease patient organization, we must raise the funds to bring life-changing treatments to our children. However, we also need the government to step in—because these treatments won't just help our children live healthier lives, they will also lower healthcare costs by alleviating the economic burden of the 30 million people in the U.S. alone who live with a rare disease. Parents simply won't give up on programs that can save our kids lives, which is why we are the exact groups that government should support. Yesterday I had to announce to our community that a beautiful 17-year-old FOXG1 girl passed away on Sunday. A few hours later, another FOXG1 dad wrote to me to let me know his son is currently fighting for his life at St. Louis Children's. Yes, parent-led patient organizations will do whatever it takes to drive these treatments to children. It might not be lucrative for Wall Street, but the ROI is our children's lives. FOXG1 Research Foundation FOXG1 Research Center Nasha Fitter Ryan Phillips Amber N. Freed Terry Pirovolakis Gerry Smith #genetherapy https://lnkd.in/giVPbemM