Charles River to Perform Plasmid Manufacturing for AAVantgarde
Charles River Laboratories and AAVantgarde have announced a CDMO agreement for the production of GMP plasmid DNA. AAVantgarde, a clinical-stage biotech firm, will use Charles River’s manufacturing expertise for their AAV-based gene delivery platforms, targeting inherited retinal diseases like Stargardt’s disease. This disease, affecting 1 in 6,500 people, causes progressive vision loss due to ABCA4 gene mutations. The collaboration will utilize Charles River’s GMP plasmid DNA center in Keele, UK, enhancing Charles River's cell and gene therapy portfolio and supporting AAVantgarde’s clinical trials.
- Charles River expands its cell and gene therapy portfolio, potentially increasing revenue.
- The collaboration targets Stargardt’s disease, addressing a high unmet medical need.
- No immediate financial gains or revenue projections from the collaboration were mentioned.
Insights
The partnership between Charles River and AAVantgarde signifies a notable advancement in addressing a significant unmet need within the ophthalmology sector. Stargardt's disease, an inherited retinal disorder with no current treatment options, affects approximately 1 in 6,500 individuals and leads to severe vision loss.
The collaboration will utilize AAVantgarde's AAV-intein platform, which has shown promising results in delivering therapeutically meaningful protein levels through efficient recombination. This innovation could lead to a breakthrough in treating genetic disorders by delivering large genes effectively to targeted tissues.
Charles River's GMP plasmid DNA manufacturing capabilities will be important in advancing AAVantgarde's Stargardt's disease program to clinical trials. This collaboration highlights the increasing importance of CDMOs in the biotechnology sector, where specialized skills and facilities accelerate the development of advanced therapies.
For retail investors, this news underscores the growth potential in companies specializing in gene therapy and genetic disorder treatments. The partnership could enhance both companies' reputations and lead to significant advancements in addressing genetic diseases.
This partnership should be closely examined for its potential financial implications. Charles River's expansion into plasmid DNA manufacturing aligns with its strategy to broaden its cell and gene therapy portfolio. This move could lead to increased revenue streams from the burgeoning gene therapy market.
For AAVantgarde, securing a reliable GMP plasmid DNA manufacturer is important for progressing their therapeutic candidates into clinical trials. This partnership not only de-risks their development pipeline but also positions them favorably for future funding rounds or partnerships.
From a retail investor's perspective, this collaboration may signal a strategic growth opportunity for Charles River and an accelerated path to potential market approval for AAVantgarde's therapies. Monitoring financial performance metrics and regulatory milestones will be key in assessing the long-term value of this partnership.
Critical starting material manufacture for therapeutic targeting high unmet need ophthalmology indication
AAVantgarde has two proprietary AAV-based large gene delivery platforms, both of which aim to enable efficient delivery of large genes to tissue and cells in vivo. Within this collaboration, Charles River will develop the plasmid DNA for AAVantgarde’s Stargardt’s disease program (AAVB-039), using their AAV-intein platform, which has demonstrated a very efficient recombination to deliver therapeutically meaningful protein levels.
Stargardt’s is the most prevalent inherited macular dystrophy and is an autosomal recessive genetic disorder due to mutations in the ABCA4 gene characterized by progressive loss of central vision starting from childhood or adolescence, leading to profound vision loss. It is the most common form of inherited juvenile macular degeneration representing a very high unmet need as there are currently no therapies available for a disease that affects approximately 1:6,500 people.
Plasmid DNA Manufacturing Services
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 Charles River’s legacy testing capabilities, developers can leverage a comprehensive “concept to cure” advanced therapies solution.
Through this collaboration, AAVantgarde will have access to Charles River’s GMP plasmid DNA CDMO center of excellence based in Keele,
To learn more about Charles River’s CDMO product and service portfolio, and gain real-world insights from a development, manufacturing, and testing perspective, available on demand is an expert roundtable hosted by Human Gene Therapy, Streamlining Cell and Gene Therapy Scalability: Progress Towards a Gold Standard, featuring panelists James Cody, PhD Associate Director, Technical Evaluations, Charles River and Lisa Kirkwood, Associate Director, Analytical, AAVantgarde: https://bit.ly/4b7aN3A
Approved Quotes
- “Charles River is thrilled to produce GMP plasmid DNA to help advance AAVantgarde’s platform for the treatment of Stargardt’s disease – a condition in which there is high unmet need. Our team brings more than two decades of CDMO expertise, and we look forward to leveraging these capabilities to make a difference for patients.” – Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River
- “By collaborating with Charles River, we are one step closer to beginning clinical trials to ensure our therapeutic product for Stargardt’s Disease is safe and efficacious for patients. We trust the team’s decades of success developing, producing, and reliably delivering plasmid DNA and look forward to expanding treatment options for this patient population.” – Nina Kotsopoulou, PhD, Chief Technical Officer, AAVantgarde
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 AAVantgarde
AAVantgarde is a clinical-stage, Italian headquartered, international biotechnology company that has developed two proprietary adeno-associated viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues. Co-founded by Professor Alberto Auricchio at TIGEM (Telethon Institute of Genetics and Medicine) in
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781.222.6455
todd.spencer@crl.com
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Source: Charles River Laboratories International, Inc.
FAQ
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