A rare (orphan) disease is medically defined as a condition that only affects a small percentage of the population. While the latest tally of people suffering from rare diseases is estimated to be 300 million (globally), it is also important to realize that many cases often go unnoticed/undiagnosed due to the scarcity in research, funding, as well as the lack of awareness from the public. To put these numbers into perspective, 1 in 20 people in the world will, at some point in their life, live with a rare condition. As such, Rare Disease Day is a good opportunity to help shed light on this noteworthy subject, with the hopes of accelerating cures and/or treatment options.

In this regard, advances in next generation sequencing technologies have paved the way to uncover the genetic causes of orphan diseases, while novel gene editing tools have allowed researchers to put forward innovative approaches in tackling these challenges at their own source. In turn, the advent of genetic engineering has spawned the exciting and burgeoning field of cell & gene therapy, which holds tremendous promise for the treatment of many incurable and debilitating diseases.

How does Cergentis fit the big picture? Driven by an unwavering commitment to advance science, our mission is to help improve the quality of genetic research through our unique proprietary TLA technology. We are proud to have been able to position our TLA technology as a rapid, robust, and cost-effective method for the complete transgene integration site characterization for producer cell banks and immunotherapy products. Thanks to the unmatched insights generated by our TLA data, our technology distinctly outperforms traditional assays in the analytical QC of therapeutic products.

Click here to learn about the capabilities of our TLA analysis in several stages during the manufacturing of cell and gene therapy products.