2023 Impact Report

2023 marked six years of the FOXG1 Research Foundation. We are a parent-driven, global foundation with the mission to help improve the lives of every person affected by FOXG1 syndrome worldwide. We are immensely proud of the organization we have built and the resources we have made available to our community since 2017.

We are known in the industry as innovators focused on three equally critical areas: FOXG1 science, FOXG1 patient data, and FOXG1 patients and community. From advancing our gene therapy program, to building our own bioinformatics platform, to helping guide parents through the rare disease medical caregiver journey, this year has been a year of upward progress in all three areas.

Our community is mourning the loss of 8 children in 2023 alone. We must stop this from happening. We are grateful for this incredible team working collectively to advance this goal, and most importantly, to our donors who enable this work.

Here are some of the FOXG1 Research Foundation highlights from 2023:

• Raised >$1.5M ($7.5M to date)

• Concluded our gene therapy preclinical work to upregulate the FOXG1 gene, rescue symptoms, and pass preliminary safety measures 

• Finalized characterization of key FOXG1 animal models, which are critical for drug screening

• Hired Chief Drug Development Officer, Dr. Gai Ayalon from Neumora Therapeutics to lead us successfully to a gene therapy clinical trial with the FDA and other international regulatory agencies

• Named Dr. Soo-Kyung Lee our Chief Scientific Officer, a FOXG1 mother and esteemed neuroscientist  who leads the FOXG1 Research Center of Excellence at the University at Buffalo with a team of >20 members focused solely on FOXG1 syndrome

• Built FOXG1 bioinformatics platform where all raw scientific, genetic and clinical data is normalized and cleansed enabling drug discovery and data for accelerated drug development from novel AI tools 

• Published paper on FOXG1 syndrome from our FOXG1 patient registry; 3 more publications underway

• Submitted paper to Journal of Neurology on epidemiology of FOXG1 syndrome meta-analysis results 

• Joined pharmaceutical-funded Biomarker studies with CombinedBrain

• Implemented FOXG1 Parent Support Team with monthly support zooms and resourceful webinars

• Connect newly diagnosed families to resources, medical professionals, and other families

• Announced the first FOXG1 patient to our advisory board, bringing a FOXG1 patient’s voice to our community

• Remain sought-after thought leaders/ speaker on podcasts and conferences

Here are some of the things the FOXG1 Research Foundation is working on in 2024:

• Moving our gene therapy program from preclinical to clinical stages with a focus on GMP manufacturing of product and toxicology studies 

• The 2024 FOXG1 Science Symposium and Parents Conference in November in Florida, USA

• Continue preclinical projects around small molecule, RNA and antisense oligonucleotide (ASO) drug screenings while continue studying the FOXG1 gene’s impact on the brain 

• Undertaking a large body of pre-work for clinical trials: deciding clinical trial endpoints, publishing a Disease Concept Model, meetings with clinical trial sites, meetings with the FDA and international agencies 

• Collaborations with biopharma companies as potential partners for our drug development programs 

• Exciting new resources for families to help assist managing their medical journeys, which includes new parent support webinars, virtual support meetings, and more

• Publication on FOXG1 Natural History Study data and FOXG1 Epidemiology papers in peer-reviewed medical journals  

• FOXG1 Research Center of Excellence at the University at Buffalo official ribbon cutting

• Growing FOXG1 clinicians network towards Standard of Care Guide for FOXG1 syndrome

• Establishment of expanded biobank with Coriell

• Continuing to expand our toolkit of assets to evaluate current and future therapeutic approaches
  

2023 FOXG1 Research Foundation Media 

*CARETALK Podcast: Why Rare Disease Research is SO Important with FOXG1 Research Foundation CEO, Nasha Fitter: Rare Disease Research and Its Potential To Unlock Medical Mysteries

*Ranked #9 Top Podcast Episode on Apple Health!

San Francisco Business Times: How two mothers are finding hope in searching for their children’s cures

Video Recap: The 2022 FOXG1 Science Symposium and Parents Conference

SFARI News: Dr. Soo-Kyung Lee, FOXG1 Research Foundation Scientist, Earns Grant from Simon Foundation Autism Research Initiative (SFARI) for Genomics of ASD: Pathways to Genetic Therapies 

Cafe Mom: 'Power of Moms': This Mom Quit Her Job To Find a Cure for Her Daughter’s Rare Disease

Press Release: New Children's Book "Joyfully Josie" Aims to Spark Conversations Around Disabilities, Rare Diseases, and Inclusion

Enable Magazine: Disability Pride Month: Educating the next generation to create an inclusive future

FOXG1 Research Foundation July Newsletter

As we've reached the midway point of 2023, we want to take a moment to provide you with an update on our progress, share some exciting news, and express our heartfelt gratitude for your support in our mission to cure FOXG1 syndrome and related neurological disorders.

First and foremost, we are immensely proud to report that we are well on track to achieve our goals for the year. This progress would not have been possible without the continued dedication and encouragement from supporters like you.

On the research front, we have made significant strides in our gene therapy and antisense programs. Encouragingly, both of these initiatives have yielded promising results in our preclinical models. Additionally, we have identified key pathways that can be targeted by FDA-approved drugs, offering potential avenues to alleviate major symptoms. The screening of several hundred FDA-approved drugs on our zebrafish models has opened up new possibilities for future treatments.

A major breakthrough in our efforts is a new publication from our FOXG1 Patient Registry, which is the most comprehensive data on FOXG1 syndrome in patients to date. This invaluable information is instrumental in ensuring our preparedness for clinical trials, bringing us closer to the day when effective treatments can be made accessible to those in need.

As we continue our relentless work with utmost efficiency and diligence, we cannot ignore the heartache we have experienced in losing too many FOXG1 children this year and over the years. Their memory drives us forward, reinforcing the critical nature of our mission to make a difference in the lives of those affected by this devastating condition.

It is with the deepest conviction that we firmly believe, together, we can provide every FOXG1 child in the world with a life free from suffering. Your support has been an integral part of this collective effort, and for that, we extend our heartfelt gratitude.

Thank you for standing with us on this challenging, but immensely rewarding journey. We are confident that brighter days lie ahead for all those affected by FOXG1 syndrome.

Congratulations and thank you to our Director of Clinical Research, Elli Brimble and our esteemed team of Key Opinion Leaders. This publication marks a major milestone in the work to deeply understand the phenotypes of patients with FOXG1 syndrome so that we can target the most effective therapies.

A special thank you to every FOXG1 parent and caregiver who registered their child and who lives this challenging life every day. 

Read FOXG1 KEY PAPERS HERE     |     Register Your FOXG1 Child HERE

After learning about the success of the new anti-epilepsy drug, Fintepla in other rare disease epilepsy clinical trials, we began working with NYU and Zogenix (Now UCB) to launch a clinical trial for FOXG1 patients. It was important for us to make this an open trial with a remote option so that FOXG1 patients do not have to travel to NYC to participate. 
This will be the first of many clinical trials designed to help all of our children live easier, healthier lives.  
Learn More HERE. 

Our lead scientists and parents to Yuna, Dr. Soo-Kyung Lee and Dr. Jae Lee, who run the The FOXG1 Center of Excellence at UB have been awarded $1.5 million from The Simons Foundation Autism Research Initiative for our FOXG1 gene therapy work!

Did you know that FOXG1 is an autism-related gene and the work we're doing has a great potential to help solve autism?

We are extremely lucky to have the Lee's working nonstop to find a cure for FOXG1 syndrome. The combination of being brilliant scientists as well as FOXG1 parents make their determination and devotion to finding a cure unparalleled in the field.

This grant is a great help towards our $20M fundraising goal for our promising FOXG1 gene therapy program.  

Continue reading the July newsletter HERE