GEN-MKT-18-7897-A
Jun 8, 2020 | Biopharma, Blogs, Pharma | 0 comments
There are over 7,000 genetic diseases that could potentially be cured using gene therapy. Rare metabolic diseases, autoimmune disorders, cardiovascular disease and cancers are some of the top disease classes that can be addressed with gene therapies. With over 1,000 clinical trials involving gene therapies or oligonucleotides currently in various stages, and 11 gene therapy drugs already on the market, it is clear the potential benefit to human health is profound.
This new generation of therapeutic modalities presents unprecedented technological challenges in bringing therapies to patients. These challenges have affected both the speed and ultimate cost of bringing new therapies to market, with many gene therapeutics now categorized as some of the most expensive medications currently in existence. Ultimately, there is a lack of sufficient tools as we seek faster and more accurate methods for characterizing these new classes of drugs.
Although faced with many unique challenges and still at its very early stages, the global gene therapy market is growing rapidly. As of 2019, the size of this market is estimated to be more than $1 billion, and it is expected to expand at a compound annual growth rate (CAGR) of 32%. This growth is primarily driven by an increase in the number of clinical trials, the amount of government and private funding and the number of partnerships between small biotech and large pharmaceutical companies.
This type of growth brings many opportunities, and to take advantage of them, it is crucial that you are prepared with solutions to the challenges ahead. To help you succeed, we invite you to download a copy of the gene therapy and oligonucleotide compendium. This compendium was created with you in mind, and it aims to provide you with precision analytics that will help bring your therapies to market faster.
The compendium includes:
Download 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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