News
FOXG1 Research Foundation ASGCT Recap -> On Track to a FOXG1 Gene Therapy
The FOXG1 Research Foundation is thrilled to share a recap of our participation at the 2024 American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore. This conference marked a significant milestone for our foundation, truly a "coming out party" to Pharma for our commitment to developing a gene therapy for FOXG1 syndrome.
The FOXG1 Research Foundation recaps our participation at the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore!
This conference marked a significant milestone for our foundation, an introduction to Pharma for our commitment to developing a gene therapy for FOXG1 syndrome.
The FOXG1 Research Foundation at the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore, Maryland 2024
Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA
A Pivotal Conversation about FOXG1 syndrome with the FDA
One of the highlights of the conference was the opportunity to speak with Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA. We discussed the FOXG1 Research Foundation's gene therapy program and the upcoming steps in our journey. This conversation was a significant milestone, paving the way for a productive collaboration with the FDA as we move forward.
Setting the Stage for FOXG1 Gene Therapy IND Approval
We are thrilled to announce that we have officially submitted our request for an INTERACT meeting with the FDA. This meeting is a crucial step in the Investigational New Drug (IND) application process, which allows us to initiate clinical trials for our gene therapy treatment. This marks the official beginning of our IND enabling journey!
FOXG1 Research Center of Excellence at the University at Buffalo poster: AAV9-FOXG1, a gene therapy vector, in a mouse model of FOXG1 syndrome
Groundbreaking Posters on FOXG1 Research Presented
The conference also provided a platform for showcasing the latest advancements in FOXG1 research. The Lee lab from the University of Buffalo presented a captivating poster on their research using AAV9-FOXG1, a gene therapy vector, in a mouse model of FOXG1 syndrome. Their findings suggest the potential of this approach in rescuing corpus callosum agenesis and other brain deficits associated with the syndrome. This research aligns perfectly with our mission and adds valuable data to the growing body of evidence supporting gene therapy as a viable treatment option.
New Findings on the Epidemiology of FOXG1 Syndrome
Another impactful presentation came from our advisor, Karen Malone, who addressed the critical challenge of describing the unmet need for treatments in rare diseases like FOXG1 syndrome. Dr. Mallone’s poster showcased innovative strategies for effectively communicating the urgency and importance of developing therapies for this patient population. The conclusion is that FOXg1 syndrome is more prevalent than currently known:
“FOXG1 Syndrome was previously considered an ultra-rare indication potentially occurring in ~1 per million children. Our analysis based on genetic testing demonstrates the FOXG1 patient population is expected to be approximately one third the size of MECP2 patients largely associated with Rett Disease.”
FOXG1 Team Building Connections and Advancing the Field
The ASGCT conference was not only about sharing our progress, but also about forging valuable connections. We had the privilege of interacting with leading figures in the gene therapy field, fostering collaboration and knowledge exchange.
The momentum gained at the ASGCT conference is truly inspiring. We at the FOXG1 Research Foundation are energized and focused on driving successful genetic therapies for FOXG1 syndrome.
Watch FOXG1 Research Foundation’s CEO Nasha Fitter’s ASGCT take-aways…
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COMBINEDBrain Biorepository enrolling FOXG1 participants at conferences across the USA
CombinedBrain invites FOXG1 families to go to any CombinedBrain member’s rare disease conferences to join the Biorepository for exciting Biomarker studies. Read more to find the next conference closest to you.
Principal investigator: FOXG1 Research Foundation in partnership with COMBINEDBrain
About the study:
COMBINEDBrain is a non-profit consortium of over 60 patient advocacy groups for rare, genetic neurodevelopmental disorders. They recognize the need to collect patient samples for researchers to identify biomarkers to be used to treat /develop treatments for our children. They are on a mission to collect ~500 samples from our member organizations over the next 6-8 months and the FOXG1 Research Foundation is one of them. Samples collected from our community will be stored and available to researchers across the world.
Are you or one of your family members interested in participating in this exciting project for FOXG1-related disorders ?
Here are the basics:
Who can participate?
Any participant diagnosed with FOXG1 syndrome and unaffected sibling.
What does participation involve?
COMBINEDBrain will be collecting urine samples and blood samples (processed for plasma and a finger stick) to be stored in the COMBINEDBrain Biorepository and available for select biomarker projects as well as other interested researchers. They will also collect several online surveys to be completed by caregivers.
Where can you participate?
COMBINEDBrain is collecting samples all across the United States this year. Please see the list below of locations to find the closest biorepository collection to you. You may attend ANY of these meetings
Sample size: The goal is to collect 20 samples from each patient group
How to enroll
Please contact [email protected] to determine the best way and location to participate.
Use of Clinical Research ID (CRID)
This study uses a CRID, or Clinical Research ID. This is a unique identification number generated and known only to the participant. This ID number allows researchers to merge data across research projects without any personally identifying information from the participant.
Please obtain a CRID prior to enrolling in this study, which is a simple online process that takes 2-3 minutes.
Then, please add your CRID to your FOXG1 Patient Registry Profile HERE
Biorepository collection locations:
COMBINEDBrain, August 6th, 9am-5pm (Courtyard by Marriott New York Queens/Fresh Meadows
183-15 Horace Harding Expy, Queens, NY 11365)
GLUT1 Deficiency, August 26th, 9am-5pm (Embassy Suites, 13700 Conference Center Drive South, Noblesville, IN 46060)
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RF2BPL Foundation, Sept 22nd-23rd 2023 (Home 2 Suites by Hilton, 7145 Liberty Centre Drive, Liberty Township, OH 45069, 513-644-2207)
KCNQ2 Cure Alliance Conference, Sept 29th-30th 2023 (Hilton Hotel Chicago 300 E Ohio St, Chicago, IL 60611)
NARS1 Conference, October 1st, 9am-5pm (13550 Commerce Blvd Rogers, MN 55374)
Prader-Willi Syndrome/USP7 Foundation, October 5-7 (1672 Lawrence St, Denver, CO 80202)
TBRS Community, Oct 12th-14th 2023 (Morgan’s Wonderland, 5223 David Edwards Dr, San Antonio, TX 78233)
COMBINEDBrain Meeting, Oct 15th-16th 2023 (Washington DC)