Gene Therapy Formulation Genetic Stability Testing
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Genetic stability is a key factor in the development and production of gene therapy preparations to ensure product safety and efficacy. Genetic stability testing is an important means of assessing whether the genome of a gene therapy preparation remains stable during production, storage and use. By monitoring genomic variation, it ensures that the product meets stringent regulatory requirements and that the quality of the gene therapy formulation is maintained.
CD Formulation is an industry leader in the development of gene therapy formulations. Based on our advanced technology platform and experienced team of experts, we provide reliable testing services to support genetic stability testing of gene therapy formulations.
The Importance of Gene Therapy Formulation Genetic Stability Testing
- Ensure product quality. Ensure that the safety and efficacy of gene therapy formulations are maintained throughout the entire process from production to end use by ensuring the stability of the genome.
- Comply with regulatory requirements. Regulatory agencies require biological products to be genetically stable throughout their lifecycle, and regular genetic stability testing ensures that gene therapy formulations meet the requirements of the relevant agencies.
- Risk management. Monitor genomic changes in gene therapy products to avoid potential mutations or structural changes affecting their product effects. Risk factors that may affect product quality, such as genetic mutations or structural changes, can also be identified and controlled promptly.
Explore Our Gene Therapy Formulation Genetic Stability Testing
We offer comprehensive genetic stability testing services using state-of-the-art next-generation sequencing (NGS) technology to ensure highly accurate genomic monitoring. Our services cover the following key components.
Whole genome sequencing
We perform full sequencing of the genome in cells or gene therapy preparations using NGS technology to identify any mutations or structural changes in the genome.
Multi-temporal analysis
We analyze samples collected at different times against each other to detect changes in the genome at different points in time to ensure its stability.
Plasmid and viral vector analysis
We monitor the insertion location, insertion size and number of plasmids or viral vectors to ensure their correct expression in the genome.
Mutation detection and evaluation
Identify emerging mutations and their potential impact to ensure the efficacy and safety of our gene therapy formulations.
Other Services of Our Gene Therapy Formulation Genetic Stability Testing
Complete method validation services
We develop genetic stability assays on the NGS technology platform for our clients' samples and provide optimal method validation reports.
Sample testing
We provide genetic stability testing services for samples used in the development of gene therapy formulations and issue related test reports.
Our Technologies for Gene Therapy Formulation Genetic Stability Testing
| Platforms & Technologies |
Content Description |
| Next generation sequencing (NGS) |
NGS technology plays an important role in comprehensively analyzing genome sequences, enabling the detection of small genetic variations by deep sequencing the genome of a sample. NGS technology exhibits high throughput in its application and can detect a wide range of mutation types. |
| Flow cytometry |
Flow cytometry can be used to detect gene expression and gene transfection efficiency in cells and to monitor genomic stability. The application of this technology is important for assessing gene expression levels and gene stability in cell populations. |
| Sanger sequencing |
Sanger sequencing can verify and confirm gene sequences in specific regions and is particularly suitable for post-NGS validation. It can accurately detect variants in individual nucleotides with high accuracy. |
| Southern blotting |
Used to detect insertion sites and copy number in gene therapy products, mainly used to verify insertion events in viral vectors or plasmids. This technique is highly specific and can effectively assess whether the inserted exogenous gene in the genome is stable or not. |
Highlights of Our Gene Therapy Formulation Genetic Stability Testing
- Flexibility and rapid response. When facing problems in gene therapy formulation development, we can quickly adjust test conditions or designs based on our extensive experience, and have the ability to adapt to any challenges or changes encountered during formulation development.
- Comprehensive service capabilities. We provide full-process support for gene therapy formulation development. In addition to genetic stability testing, we offer comprehensive services from formulation development to manufacturing process optimization.
- Attentive development services. We offer long-term test monitoring and data analysis in addition to assisting researchers in the development of gene therapy formulations so that clients may avoid any hazards and understand the formulation's status in real-time.
- Innovative technology frontiers. We always stay on the cutting edge of the latest technology, such as employing modern technology platforms and testing systems to improve the efficiency and accuracy of testing.
Published Data
Technology: Gene therapy formulation stability testing
Journal: Eur J Pharm Biopharm
IF: 4.9
Published: 2023
Gene therapies are emerging as promising treatments for various disorders with limited therapeutic options. However, delivering polynucleic acids effectively to target cells remains a key challenge due to their chemical and physical properties. Adeno-associated viruses (AAVs) have gained prominence for delivering therapeutic single-stranded DNA (ssDNA), with over a hundred products tested. Efforts focus on optimizing recombinant AAV (rAAV) vectors to ensure safety, low immunogenicity, and consistent quality for both local and systemic use. Unlike protein therapeutics, most rAAV products are supplied as frozen liquids, posing distribution challenges. This paper discusses the difficulties in rAAV product development, especially in formulation, and highlights recent advances aimed at creating stable liquid or lyophilized products for broader use.
CD Formulation provides reliable, efficient and regulatory-compliant genetic stability assays for gene therapy formulations to ensure the safety and efficacy of your gene therapy formulation development. If you are interested in us, please feel free to contact us.
References
- Grossen P, et al. The ice age - A review on formulation of Adeno-associated virus therapeutics. Eur J Pharm Biopharm. 2023, 190:1-23.
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