Charles River and Genetic Cures for Kids Announce Gene Therapy Manufacturing Collaboration
28 Novembro 2023 - 10:00AM
Business Wire
Company will perform plasmid DNA production in
support of early phase trials for SPG56 Hereditary Spastic
Paraplegia
Charles River Laboratories International, Inc. (NYSE: CRL) and
Genetic Cures for Kids Inc (GC4K), an Australian non-profit
foundation focused on research programs and clinical trials to find
cures for rare diseases, today announced a plasmid DNA contract
development and manufacturing organization (CDMO)
collaboration.
Founded in 2021 by Golden and Chris Whitrod, parents of now 4
year old Tallulah Moon, GC4K’s first mission, known as Our Moon’s
Mission, is to develop a gene therapy for the treatment of
Hereditary Spastic Paraplegia Type 56 (SPG56). SPG56 is a
debilitating neurodegenerative disease that robs children of the
ability to walk, talk, and move independently.
What is SPG56?
SPG56 manifests as a progressive neurological disease
characterised by varying degrees of spasticity and muscle weakness.
This degenerative disease typically begins with motor and cognitive
regression in childhood and continuously worsens through life.
Additional clinical manifestations include intellectual disability,
dystonia, cerebellar ataxia, subclinical peripheral neuropathy,
seizures, and visual impairment.
Tragically, there is currently no treatment for this ultra-rare
genetic disease. In addition, limited access to genetic testing
means diagnoses are hard to achieve and as such, fewer than one in
1 million people have ever been diagnosed with SPG56 globally. GC4K
is leading a gene therapy program that aim not to only cure SPG56,
but to create a replicable framework that paves the way to develop
treatments for some of the 7,000 other genetic diseases in the
world.
Plasmid DNA Manufacturing Services
GC4K will leverage Charles River’s established plasmid platform,
eXpDNA™, and premier expertise in plasmid DNA production, including
High Quality (HQ) plasmid which combines key features of good
manufacturing practice (GMP) manufacture with a rapid turnaround to
accelerate time to clinic. As a critical starting material in the
development of cell and gene therapies, in 2022 Charles River
opened a state-of-the-art HQ Plasmid Manufacturing Center of
Excellence dedicated to addressing global supply shortages and
support the growing needs of customers.
In recent years, Charles River has significantly broadened its
cell and gene therapy portfolio 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 a comprehensive
“concept-to-cure” advanced therapies solution.
To learn more about plasmid DNA production, watch Advancements
in Manufacturing for Rapid Delivery of pDNA Starting Materials,
presented by Andrew Frazer, PhD, Associate Director, Scientific
Solutions, Gene Therapy CDMO Services, on-demand:
https://bit.ly/3ZGgPnd
Approved Quotes
- “The opportunity to work with GC4K is exactly why we do what we
do, playing a role in delivering potentially curative treatments to
patients suffering with an ultra-rare disease. We are excited to
support the team as they work through the next stage of their
program development.” - Kerstin Dolph, Corporate Senior Vice
President, Biologics Solutions, Charles River
- “What was once a tragic diagnosis, has the potential to become
a triumph, as we draw nearer to finding a cure for SPG56. With the
advancement of this groundbreaking treatment, our partnership with
Charles River is bringing us closer to aiding children like
Tallulah, who, until now, have faced the daily hardships of SPG56
without any glimmer of hope. We're truly excited to have the
backing of this skilled team as we gear up to produce this historic
treatment. By making this announcement on Giving Tuesday, we are
giving thanks to Charles River. This symbolic occasion not only
commends the generosity of CRL in supporting GC4K’s quest to cure
SPG56, but also to recognizes the broader goal that our partnership
is striving for - a brighter future for all children living with
rare genetic diseases." - Golden Whitrod, Tallulah Moon’s mother
and Co-Founder and President, Genetic Cures for Kids, Inc.
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.
Genetic Cures for Kids, Inc., and Our Moon’s Mission
GC4K is a family-driven research foundation at the forefront of
genetic research, with a primary objective to not only develop a
cure for SPG56 but also to establish a reproducible framework that
can catalyze the development of treatments for some of the 7,000
known genetic diseases worldwide.
The foundation's campaign to cure SPG56 is "Our Moon's Mission",
named in honor of the founders’ daughter Tallulah Moon, who lives
with SPG56. By leveraging crowdfunding to assemble an expert
international research team, which has since gained recognition and
funding from the Australian Government and is guided by an
Independent Scientific Advisory Board, GC4K aims to expedite the
development of effective SPG56 therapies for clinical use.
Additionally, the foundation is dedicated to advocating for 'rare
families' and increasing awareness of rare diseases. To learn more
about GC4K's commitment to advancing genetic research and to
support the foundation's mission to cure SPG56, please visit
www.ourmoonsmission.org
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version on businesswire.com: https://www.businesswire.com/news/home/20231128522610/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
Genetic Cures for Kids Inc Media Contact: Golden Whitrod
Co-Founder, Board President, Genetic Cures for Kids (Our Moon’s
Mission) E: golden@ourmoonsmission.org M: +61449154411
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