Charles River Announces Rare Disease Gene Therapy Collaboration with Axovia Therapeutics
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Insights
The collaboration between Charles River Laboratories and Axovia Therapeutics marks a significant step in the development of gene therapies targeting ciliopathies, particularly Bardet-Biedl Syndrome (BBS). The manufacturing of High Quality (HQ) plasmid DNA is a critical component in the gene therapy production process. Plasmids serve as vectors to deliver the corrected gene to patient cells and the quality of these plasmids directly impacts the efficacy and safety of the therapy.
Considering the rarity of BBS and the Orphan Drug and Rare Pediatric Disease Designations granted by the FDA, this partnership could lead to advancements in a market with high unmet medical needs. These designations also offer certain benefits, such as market exclusivity upon approval, which can be financially beneficial. However, the complexity and cost of gene therapy development are considerable and the path to market approval is fraught with both scientific and regulatory challenges.
Charles River Laboratories' expansion into plasmid DNA production for gene therapies is indicative of the company's strategic focus on growing its presence in the burgeoning field of advanced therapies. By offering a 'concept-to-cure' solution, Charles River is positioning itself as a key player in the cell and gene therapy supply chain. This could potentially lead to a diversification of revenue streams and bolster its market position.
For investors, the implications of this collaboration could be significant in the long term, as successful development and commercialization of gene therapies for rare diseases often command premium pricing due to their transformative impact on patients' lives. However, it is important to monitor the progress of clinical trials and regulatory milestones, as these will be key indicators of the potential return on investment.
The gene therapy sector is rapidly evolving, with technological advancements reducing production time and potentially improving the safety profile of these therapies. Charles River's eXpDNA™ platform exemplifies this progress, aiming to expedite the time to clinic for gene therapies. The demand for plasmid DNA and viral vectors is expected to rise as more gene therapies enter clinical development.
The partnership with Axovia could enhance Charles River's reputation in the gene therapy CDMO space, potentially attracting more collaborations. However, market success is contingent upon the clinical success of Axovia's AXV101 and other gene therapies. The industry trend is towards personalized medicine and companies that can deliver effective and efficient solutions in this space may see increased demand for their services.
HQ plasmid DNA production to support Phase I trials for gene therapy targeting Bardet-Biedl Syndrome
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.
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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
Source: Charles River Laboratories International, Inc.
FAQ
What is the focus of the collaboration between Charles River Laboratories (CRL) and Axovia Therapeutics ?
What is Axovia's lead program in the collaboration with CRL?
What are ciliopathies?
How many ciliopathies have been identified?
What regulatory designations has Axovia received for its lead program?
