gene therapy

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Charles River Collaborates with Patient Advocacy Group, FOXG1 Research Foundation to Advance Rare Disease Gene Therapy Development

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WILMINGTON, Mass.--(BUSINESS WIRE)--Jul. 30, 2024-- Charles River Laboratories International, Inc. (NYSE: CRL) announced today a collaboration with the FOXG1 Research Foundation (FRF) highlighting the patient advocacy group’s model to independently drive drug development through the clinical phase. The parent-led global organization driving the research to cure FOXG1 syndrome and related neurological disorders will collaborate with Charles River in a comprehensive gene therapy contract development and manufacturing organization (CDMO) agreement.

“Charles River is proud to work with the FOXG1 Research Foundation to advance its gene-therapy through clinical trials,” said Kerstin Dolph, Corporate Senior Vice President, 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.

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New publication shows groundbreaking results in FOXG1 AAV9 gene therapy studies; rescuing structural brain abnormalities

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Groundbreaking Study Shows Promise in AAV9 Gene Therapy for FOXG1 Syndrome; Rescue of Brain Structure Abnormalities and Deficits.

[Buffalo, New York June 10, 2024] – A landmark study led by Dr. Soo-Kyung Lee, Chief Scientific Officer at the FOXG1 Research Foundation and Empire Innovation Professor and Om P. Bahl Endowed Professor in the Department of Biological Sciences at University at Buffalo, and Dr. Jae Lee, Professor in the Department of Biological Sciences at University at Buffalo, in collaboration with Dr. Kathrin Meyer (responsible for the SMA gene therapy), has been published in Molecular Therapy Methods & Clinical Development.

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University at Buffalo launches center to find treatments for FOXG1 syndrome

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University at Buffalo launches center to find treatments for FOXG1 syndrome

University at Buffalo announces the launch of the FOXG1 Research Center to study FOXG1 syndrome’s impact on brain development and translate research to treatments for FOXG1 syndrome. The FOXG1 Research Center will be led by leading experts Soo-Kyung and Jae Lee, whose own daughter has FOXG1 syndrome.

“This center will make UB the home of the world’s premier research center devoted to the studies of FOXG1 syndrome, as well as provide our campus with a new neurodevelopmental biology training program and numerous research funding opportunities,” says Soo-Kyung Lee, PhD, Empire Innovation Professor and Om P. Bahl Endowed Professor in the UB Department of Biological Sciences, who will serve as the FRC’s inaugural director as well as the Chief Scientific Officer of the FOXG1 Research Foundation.

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 known in the industry as innovators as we  focus 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. Here are the FOXG1 Research Foundation highlights from 2023 and goals for 2024.


FRF Appoints Chief Drug Development Officer, Dr. Gai Ayalon

The FOXG1 Research Foundation announces the appointment of Dr. Gai Ayalon as the Chief Drug Development Officer..

Dr. Ayalon is a distinguished neuroscientist and drug developer, who over many years led teams and spearheaded drug development programs for neurological diseases including rare neurodevelopmental disorders, spanning the discovery, translation and clinical phases. He joins our foundation at a critical and pivotal juncture as we enter Phase Four on our Path to a Cure, taking programs through safety studies and clinical trials. 

Dr. Soo-Kyung Lee Named FOXG1 Research Foundation Chief Scientific Officer

The FOXG1 Research Foundation (FRF), announced today the appointment of Dr. Soo-Kyung Lee as the new Chief Scientific Officer of the FOXG1 Research Foundation. Dr. Lee is an Empire Innovation Professor and Om P. Bahl Endowed Professor at University at Buffalo Department of Biological Sciences. Since joining the FRF in 2017, Dr. Lee has elevated the FOXG1 research center at the University at Buffalo to a global leader in the field. Her exceptional work has garnered around $3M annually from federal agencies, FRF, UB, and the Simon Foundation Autism Research Initiative (SFARI), notably receiving SFARI's 2022 Genomics of ASD: Pathways to Genetic Therapies award.

Dr. Lee’s dedication to curing FOXG1 syndrome is deeply personal as a mother to Yuna, diagnosed with FOXG1 syndrome in 2009. Her passion and commitment have inspired everyone fortunate enough to work with her. It's a rare and special circumstance to have FOXG1 parents leading the scientific journey towards effective therapeutics for all FOXG1 children globally.

2022 Impact Report

2022 Impact Report

2022 Research Highlights (see details below)

  • Breakthrough gene therapy results showing rescue of FOXG1 brain structure, behavior, memory and cognition symptoms in animal models

  • Positive results on initial compound drug screens to identify molecules to increase FOXG1 levels; now moving forward to larger screens

  • Never-before understanding of FOXG1 biology uncovered from six FOXG1 patient human cell lines and mouse models 

  • Successful testing of guide RNAs to increase FOXG1 expression with a CRISPRa Cas-9 system

  • Discovered ASO sequences (antisense gene therapy) to modulate FOXG1 expression; testing initiated with Creyon Bio  

  • FOXG1 data package presented to several biotech companies with high interest


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The Johnson Family - Changing the World Right Here in Port Washington

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The Johnson Family - Changing the World Right Here in Port Washington

FOXG1 Research Foundation co-founder and Executive Director shared her story with her hometown local magazine called Port Washington Living. This feature article celebrates the Johnson family and Nicole’s work to find a cure for FOXG1 syndrome, while helping FOXG1 families around the world, including helping the FOXG1 family in the Ukraine to safety.