Charles River Announces Rare Disease Gene Therapy Collaboration with Axovia Therapeutics
2024年4月9日 - 9:00PM
ビジネスワイヤ(英語)
HQ plasmid DNA production to support Phase I
trials for gene therapy targeting Bardet-Biedl Syndrome
Charles River Laboratories International, Inc. (NYSE: CRL) today
announced a plasmid DNA contract development and manufacturing
organization (CDMO) collaboration with Axovia Therapeutics Ltd.
Charles River will manufacture High Quality (HQ) gene of interest
plasmid to support the development of Axovia’s gene therapies for
ciliopathies, including Bardet-Biedl Syndrome (BBS), a condition
with limited treatment options and no cure.
Ciliopathies are diseases caused by cilia dysfunction, which is
a group of complex disorders caused by genetic mutations that
result in defective or dysfunctional cilia in many organs of the
human body. Dysfunctional cilia can cause blindness, deafness,
chronic respiratory infections, kidney disease, heart disease,
infertility, obesity and diabetes. Over 20 ciliopathies have been
identified, collectively affecting an estimated 1 in 1,000
people.
Axovia’s lead program, AXV101, is an adeno-associated virus
(AAV9)-based gene therapy targeting retinal dystrophy associated
with BBS. In preclinical studies for BBS, the novel gene therapy
modified the underlying disease, rescuing vision loss by halting
retinal degeneration, stopping BBS-induced weight gain and the
development of obesity. Axovia has been granted FDA Orphan Drug and
Rare Pediatric Disease Designations.
Plasmid DNA Manufacturing Services
Axovia will leverage Charles River’s established plasmid
platform, eXpDNA™, and premier expertise in plasmid DNA production,
including HQ plasmid which combines key features of good
manufacturing practice (GMP) manufacture with a rapid turnaround to
accelerate time to clinic.
In recent years, Charles River has significantly broadened its
cell and gene therapy portfolio to simplify complex supply chains
and meet the 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.
Available on-demand is a 30-minute case study webinar led by Dr.
Andrew Frazer, Associate Director, Scientific Solutions, Gene
Therapy CDMO Services, that explores the role of plasmid DNA as a
critical starting material, the latest advancements in associated
regulatory guidance to safeguard your therapeutic programs, and
case studies demonstrating expedited development and manufacture of
ATMPs: https://bit.ly/3vbI9yS
Approved Quotes
- “We are committed to supporting Axovia in the pursuit of
creating treatment options for patients living with ciliopathies.
The Charles River team brings decades of expertise in HQ plasmid
development, and we look forward to using these capabilities to
make a difference for patients.” - Kerstin Dolph, Corporate Senior
Vice President, Global Manufacturing, Charles River
- “This collaboration brings us closer to accomplishing our goal
to develop potentially transformative therapies for ciliopathies.
We are excited to leverage Charles River’s expertise in plasmid DNA
production to help drive our work forward.” - Victor Hernandez,
Co-Founder and Chief Scientific Officer, Axovia
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 Axovia Therapeutics Ltd.
Axovia Therapeutics, an ALSA Ventures portfolio company, is
developing the first novel gene therapies for ciliopathies and has
a pipeline of products for these devastating diseases, including
Bardet-Biedl Syndrome (BBS). For more information, visit
www.axoviatherapeutics.com.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240409253110/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
Charles River Laboratories (NYSE:CRL)
過去 株価チャート
から 5 2024 まで 6 2024
Charles River Laboratories (NYSE:CRL)
過去 株価チャート
から 6 2023 まで 6 2024