GEN-MKT-18-7897-A
May 7, 2020 | Biopharma, Blogs, Pharma | 0 comments
For many of you working to develop gene therapy drugs, you know that the time to market the drug is critical. Because gene therapeutics cure diseases by targeting specific genes, it is a constant race to see who develops the drug first. Unlike other classes of drugs where multiple medications can be used to treat a disease, whoever is first to develop a gene therapy drug wins.
When it comes to adeno-associated virus-based gene therapies, there is a lack of reliable and reproducible methods to consistently produce them. One of the key challenges you face when analyzing AAVs is determining whether the therapeutic transgene payload has been successfully incorporated into the AAV vector product.
During the manufacturing of AAV vectors, capsids containing the full payload of transgenes are produced. There is also a high percentage of capsids that might not incorporate any of the transgenes (empty), or contain fragments of the transgene (partial), that are produced as well. The presence of these impurities could increase immunogenicity or inhibit transduction of full capsids by competing for vector binding sites on cells. That is why successful incorporation of the transgene is critical for the efficacy and safety of gene therapies.
SCIEX has developed a breakthrough analytical method that is able to detect with great precision whether the AAV capsids are full, partially full or empty.
You will discover:
This is instrumental in improving and streamlining the development and production process for your AAV-based therapeutics. By giving you the right analytics, you will be able to develop better quality and safer products, all while reducing the cost to manufacture.
With the prospect of shorter analysis time and better analytics, request a copy of our technical note dedicated to teaching you all about our novel method. Find out how you can improve your drug development process with this method now.
In biopharmaceutical development, sequence variants (SV) are considered an inherent risk of producing complex proteins in living systems. Sequence variants are unintended changes to the amino acid sequence of a biotherapeutic and can be caused by errors in transcription or translation in the host cell, or cell culture and process conditions. Detailed analysis of SVs is important in process and product development to ensure the drug’s safety and efficacy. Even low‑level sequence variants can have significant implications for product quality, safety, and efficacy, making their accurate detection and characterization a critical requirement across development, process optimization, and regulatory submission.
CE‑SDS remains a cornerstone assay for characterizing fragmentation, aggregation, and product‑related impurities in therapeutic proteins. UV detection has been the long‑standing standard. However, it frequently struggles with baseline noise, limited sensitivity for minor fragments, and subjective integration.
At SCIEX, innovation doesn’t stop at instruments; it extends to how you interact with your LC-MS/MS or CE systems every day. That’s why we’re excited to introduce the SCIEX Now spring 2026 improvements: a set of meaningful enhancements shaped directly by your feedback.
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