Charles River Collaborates with Patient Advocacy Group, FOXG1 Research Foundation to Advance Rare Disease Gene Therapy Development
Charles River Laboratories International, Inc. (NYSE: CRL) has announced a collaboration with the FOXG1 Research Foundation (FRF) to advance gene therapy development for FOXG1 syndrome, a severe rare neurological genetic disorder. The partnership highlights FRF's innovative model for independently driving drug development through clinical phases.
Charles River will provide FRF with access to its cell and gene therapy expertise, generating materials for FRF's Phase I-II AAV vector-based gene therapy clinical trials. This includes supplying High Quality (HQ) plasmid starting materials and GMP AAV9 viral vectors from its centers of excellence.
The collaboration aims to streamline FRF's path to clinical trials, leveraging Charles River's integrated manufacturing and biologics testing portfolio. This partnership exemplifies a new approach to rare disease drug development, with patient advocacy groups taking control and driving the process when no treatment options exist.
Charles River Laboratories International, Inc. (NYSE: CRL) ha annunciato una collaborazione con la FOXG1 Research Foundation (FRF) per promuovere lo sviluppo della terapia genica per la sindrome FOXG1, un grave disturbo neurologico genetico raro. Questa partnership mette in evidenza il modello innovativo di FRF per guidare in modo indipendente lo sviluppo di farmaci attraverso le fasi cliniche.
Charles River fornirà a FRF accesso alla propria esperienza in terapia cellulare e genica, generando materiali per i trial clinici di terapia genica AAV basata su vettori Fase I-II. Questo include la fornitura di materiali a partire da plasmidi di Alta Qualità (HQ) e vettori virali GMP AAV9 dai suoi centri di eccellenza.
La collaborazione mira a semplificare il percorso di FRF verso le sperimentazioni cliniche, sfruttando il portafoglio integrato di produzione e test biologici di Charles River. Questa partnership esemplifica un nuovo approccio allo sviluppo di farmaci per malattie rare, con i gruppi di advocacy dei pazienti che prendono il controllo e guidano il processo quando non esistono opzioni terapeutiche.
Charles River Laboratories International, Inc. (NYSE: CRL) ha anunciado una colaboración con la FOXG1 Research Foundation (FRF) para avanzar en el desarrollo de terapias génicas para el sindrome FOXG1, un grave trastorno neurológico genético raro. La asociación destaca el innovador modelo de FRF para impulsar de manera independiente el desarrollo de medicamentos a través de las fases clínicas.
Charles River proporcionará a FRF acceso a su experiencia en terapias celulares y génicas, generando materiales para los ensayos clínicos de terapia génica basada en vectores AAV Fase I-II. Esto incluye el suministro de materiales iniciales de plásmidos de Alta Calidad (HQ) y vectores virales GMP AAV9 de sus centros de excelencia.
La colaboración tiene como objetivo agilizar el camino de FRF hacia los ensayos clínicos, aprovechando la cartera integrada de fabricación y pruebas biológicas de Charles River. Esta asociación ejemplifica un nuevo enfoque en el desarrollo de medicamentos para enfermedades raras, con grupos de defensa de pacientes tomando el control y guiando el proceso cuando no existen opciones de tratamiento.
찰스 리버 연구소 국제 주식회사(NYSE: CRL)가 심각한 희귀 신경 유전 질환인 FOXG1 증후군을 위한 유전자 치료 개발을 추진하기 위해 FOXG1 연구 재단(FRF)과 협력한다고 발표했습니다. 이 파트너십은 FRF가 임상 단계를 통해 독립적으로 약물 개발을 이끄는 혁신적인 모델을 강조합니다.
찰스 리버는 FRF에 대한 세포 및 유전자 치료 전문 지식에 접근을 제공하며, FRF의 1-2상 AAV 벡터 기반 유전자 치료 임상 시험을 위한 재료를 생성할 것입니다. 여기에는 고품질(HQ) 플라스미드 시작 물질과 그들의 우수 센터에서 생산된 GMP AAV9 바이러스 벡터의 공급이 포함됩니다.
이 협업의 목표는 FRF의 임상 시험 경로를 간소화하고 찰스 리버의 통합 제조 및 생물학적 테스트 포트폴리오를 활용하는 것입니다. 이 파트너십은 치료 옵션이 존재하지 않을 때 환자 옹호 그룹이 통제를 받고 과정을 이끄는 희귀 질병 약물 개발에 대한 새로운 접근 방식을 예시합니다.
Charles River Laboratories International, Inc. (NYSE: CRL) a annoncé une collaboration avec la FOXG1 Research Foundation (FRF) pour faire progresser le développement de thérapies géniques pour le syndrome FOXG1, un trouble génétique neurologique rare et sévère. Ce partenariat met en évidence le modèle innovant de la FRF pour conduire de façon indépendante le développement de médicaments à travers les phases cliniques.
Charles River fournira à la FRF un accès à son expertise en thérapie cellulaire et génique, générant des matériaux pour les essais cliniques de thérapie génique basés sur des vecteurs AAV de Phase I-II. Cela inclut la fourniture de matériaux de départ de plasmides de Haute Qualité (HQ) et de vecteurs viraux GMP AAV9 de ses centres d'excellence.
La collaboration vise à rationaliser le parcours de la FRF vers les essais cliniques, en tirant parti du portefeuille intégré de fabrication et de tests biologiques de Charles River. Ce partenariat illustre une nouvelle approche dans le développement de médicaments pour les maladies rares, avec des groupes de défense des patients prenant le contrôle et dirigeant le processus lorsque aucune option de traitement n'existe.
Charles River Laboratories International, Inc. (NYSE: CRL) hat eine Zusammenarbeit mit der FOXG1 Research Foundation (FRF) angekündigt, um die Entwicklung von Gentherapie für das FOXG1-Syndrom, einer schweren, seltenen neurologischen Erbkrankheit, voranzutreiben. Die Partnerschaft hebt das innovative Modell von FRF hervor, um die Arzneimittelentwicklung unabhängig durch klinische Phasen zu steuern.
Charles River wird FRF Zugang zu seiner Expertise in der Zell- und Gentherapie bieten, um Materialien für die Phase I-II AAV-Vektor-basierte Gentherapie klinischen Studien zu erstellen. Dies umfasst die Bereitstellung von hochwertigen (HQ) Plasmidstartmaterialien und GMP AAV9-Virusvektoren aus seinen Exzellenzzentren.
Die Zusammenarbeit zielt darauf ab, den Weg von FRF zu klinischen Studien zu erleichtern und das integrierte Herstellungs- und Biologietestportfolio von Charles River zu nutzen. Diese Partnerschaft ist ein Beispiel für einen neuen Ansatz in der Arzneimittelentwicklung für seltene Krankheiten, bei dem Patientenvertretungsgruppen die Kontrolle übernehmen und den Prozess leiten, wenn keine Behandlungsoptionen vorhanden sind.
- Collaboration with FOXG1 Research Foundation expands CRL's presence in rare disease gene therapy development
- Utilization of CRL's extensive cell and gene therapy expertise for clinical trial material production
- Potential for increased revenue through comprehensive CDMO agreement
- Strengthening of CRL's position in the growing advanced therapies market
- None.
Insights
This collaboration between Charles River Laboratories and the FOXG1 Research Foundation (FRF) represents a significant milestone in rare disease gene therapy development. The partnership leverages Charles River's expertise in cell and gene therapy contract development and manufacturing (CDMO) to support FRF's innovative patient advocacy-driven drug development model.
Key points to consider:
- FRF's approach as a "virtual biotech company" is pioneering a new model for rare disease research, potentially influencing future drug development strategies for other rare conditions.
- The collaboration provides FRF access to Charles River's advanced capabilities in plasmid DNA and viral vector manufacturing, critical components for AAV9 gene therapy development.
- This partnership could accelerate the timeline for bringing a potential treatment to clinical trials for FOXG1 syndrome, a severe neurological disorder with no current approved treatments.
- The success of this model could attract more investment and attention to rare disease research, potentially catalyzing similar initiatives in the biotech industry.
From an investor's perspective, this collaboration showcases Charles River's strategic positioning in the growing cell and gene therapy market. It demonstrates the company's ability to support both traditional pharma/biotech clients and innovative patient advocacy groups, potentially expanding its customer base and revenue streams in the advanced therapies sector.
The collaboration between Charles River Laboratories and the FOXG1 Research Foundation (FRF) marks a significant advancement in rare disease research and gene therapy development. Here's why this partnership is noteworthy from a medical research perspective:
- FOXG1 syndrome, affecting approximately 1,000 diagnosed patients worldwide, represents a critical unmet medical need. The lack of approved treatments for this severe neurological disorder underscores the importance of this research initiative.
- The use of AAV9 vector-based gene therapy for FOXG1 syndrome is a promising approach. AAV9 has shown the ability to cross the blood-brain barrier, making it a suitable candidate for treating neurological disorders.
- FRF's model of independently driving drug development through clinical phases is innovative and could serve as a template for other rare disease advocacy groups. This approach may accelerate the typically slow process of rare disease drug development.
- The collaboration leverages Charles River's expertise in plasmid DNA and viral vector manufacturing, which are important components for developing gene therapies. This integrated approach, from High Quality (HQ) plasmid production to GMP AAV9 viral vector manufacturing, could streamline the path to clinical trials.
While the potential impact on FOXG1 patients is significant, it's important to note that gene therapy development is complex and success is not guaranteed. However, this collaboration represents a promising step forward in addressing a severe rare disease with no current treatment options.
Clinical trials for AAV9 gene therapy bolstered with plasmid and viral vector CDMO expertise
“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.”
Through the collaboration, Charles River will provide FRF with access to extensive cell and gene therapy expertise and generate materials for FRF’s Phase I-II adeno-associated viral (AAV) vector-based gene therapy clinical trials at its plasmid DNA and viral vector CDMO centers of excellence (CoE). The established CDMO will supply phase-appropriate High Quality (HQ) plasmid starting materials manufactured at its Alderley Park CoE in addition to good manufacturing practice (GMP) AAV9 viral vector manufactured at its Rockville CoE, leveraging an integrated manufacturing and biologics testing portfolio to streamline their path to the clinic.
“Given the limited investment for rare disease groups like ours, our foundation has created a model that allows us and other patient advocacy groups to operate like a virtual biotech company and independently and efficiently drive drug development,” said Nasha Fitter, FOXG1 Parent, Co-founder and Chief Executive Officer, FOXG1 Research Foundation.
A New Approach to Drive Rare Disease Drug Development
Founded in 2017 and propelled by innovation and an urgency to accelerate the road to therapeutics for FOXG1 syndrome, FRF has created a replicable model for rare disease patient advocacy groups to take control and drive the development of treatments when no options exist. This includes pioneering novel AI platforms for patient data and streamlining preclinical work.
“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,” continued Fitter.
FOXG1 syndrome is a severe rare neurological genetic disorder that greatly impacts early brain development and typically causes epilepsy and a host of medical complexities and disabilities. There are approximately 1,000 patients diagnosed with FOXG1 syndrome worldwide, with the diagnosis rate climbing steadily year-over-year and no approved treatments. Most children with FOXG1 syndrome cannot walk or talk or take care of their basic needs.
Cell and Gene Therapy CDMO Solutions
In recent years, Charles River has significantly broadened its cell and gene therapy portfolio with several acquisition integrations and expansions to simplify complex supply chains and meet growing demand for plasmid DNA, viral vector, and cell therapy services. Combined with the Company’s legacy testing capabilities, Charles River offers an industry-leading “concept to cure” advanced therapies solution.
Presented live at the Charles River Cell and Gene Therapy Summit, March 19, 2024, in
Also, listen to "A Mom’s Mission", a Vital Science Podcast, broadcast July 16, 2024, where Nasha discusses the origins of FOXG1 Research Foundation, how daughter Amara’s life has been shaped by her diagnosis, and how collaboration has helped advance their mission: https://bit.ly/3LuCpog
About Charles River
Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit www.criver.com.
About FOXG1 Research Foundation
The FOXG1 Research Foundation (FRF) is the global, parent-led rare disease patient organization dedicated to advancing treatments for FOXG1 syndrome and related disorders, while advocating for and supporting patients and families worldwide. FOXG1 syndrome is a rare neurological developmental disorder linked to Autism Spectrum Disorder, epilepsy, and developmental disabilities. Founded in 2017, FRF has rapidly emerged as a leader and innovator in the rare disease patient advocacy space, developing novel platforms and an efficient blueprint to accelerate drug development for rare diseases. As a Chan Zuckerberg Initiative partner and recognized through its CEO’s presentation at the inaugural White House Rare Disease Forum, FRF is committed to radically improving the landscape for the 300 million patients affected by rare diseases worldwide. www.foxg1research.org
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Charles River Investor Contact:
Todd Spencer
Corporate Vice President,
Investor Relations
781.222.6455
todd.spencer@crl.com
Charles River Media Contact:
Amy Cianciaruso
Corporate Vice President,
Chief Communications Officer
781.222.6168
amy.cianciaruso@crl.com
FOXG1 Research Foundation Media Contact:
Nicole Johnson
Co-Founder and Executive Director
nicole@foxg1research.org
Source: Charles River Laboratories International, Inc.
FAQ
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