Charles River Collaborates with Patient Advocacy Group, FOXG1 Research Foundation to Advance Rare Disease Gene Therapy Development
2024年7月30日 - 9:00PM
ビジネスワイヤ(英語)
Clinical trials for AAV9 gene therapy bolstered
with plasmid and viral vector CDMO expertise
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.”
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 San Francisco, watch Nasha Fitter’s
FOXG1 Research Foundation Patient Story session on-demand, and
explore the Summit resource hub: https://bit.ly/4bb2uDu
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
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240730710706/en/
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
Charles River Laboratories (NYSE:CRL)
過去 株価チャート
から 8 2024 まで 9 2024
Charles River Laboratories (NYSE:CRL)
過去 株価チャート
から 9 2023 まで 9 2024