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 >
Finding the right information shouldn’t slow you down. Whether you’re troubleshooting your mass spec, learning something new, or optimizing performance, access to the right resources at the right moment makes all the difference.
As an analytical strategy, middle-down mass spectrometry (MS) workflows characterize biotherapeutic proteins by analyzing large, digested protein fragments or defined subunits, rather than fully intact proteins (top-down) or digested peptides (bottom-up). A middle-down strategy combines the strengths of top-down and bottom-up approaches by delivering high sequence coverage and structural specificity while maintaining relatively simple sample preparation. In practice, middle-down analysis enables accurate mass measurement, rapid sequence confirmation, and localization of key post-translational modifications (PTMs) on protein subunits that are directly relevant to product quality.
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.
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