Centogene and Evotec have expanded their collaboration agreement to focus on the development of new treatments for Gaucher disease and other rare disorders caused by mutations in the GBA gene, the companies announced.
This expanded partnership builds upon a previous collaboration agreement, launched in 2018, to develop a high-throughput platform to assess the therapeutic potential of several small molecules in rare hereditary metabolic diseases.
“Our collaboration with Evotec further underlines CENTOGENE’s commitment to driving biomarker discoveries and accelerating orphan drug development by leveraging our global expertise in rare hereditary diseases,” Arndt Rolfs, CEO of Centogene, said in a press release.
“In particular, we are able to draw on over 20 years of knowledge and research of Gaucher disease,” Rolfs said. “By continuing our work alongside Evotec, we will accelerate transformational medical solutions in the rare disease field.”
Gaucher is caused by a mutation in the GBA gene, which compromises the production of an enzyme called beta-glucocerebrosidase. In the absence of this enzyme, a fatty substance called glucocerebroside starts to accumulate inside immune cells, which then infiltrate into different tissues and organs, causing problems with swelling, blood flow, or breathing.
In addition to Gaucher, mutations in GBA have been associated with a higher risk for Parkinson’s disease and dementia with Lewy bodies.
“The potential to further the understanding of rare diseases connected to mutations in the GBA gene is extremely important to patients and their families around the world, and we hope that today’s collaboration agreement will help lead to transformational personalized treatments for these patients,” said Philip Lambert, chief scientific officer at Centogene.
To achieve this goal, the partnership will combine Evotec’s platform of induced pluripotent stem cells (iPSCs), as well as the company’s treatment discovery and development capabilities, with Centogene’s platform on rare disorders. Notably, iPSCs are fully matured cells that were reprogrammed back to a stem-cell state to generate other types of cells in the body.
“The partnership with Centogene supports and complements our patient-centric drug discovery approach in Gaucher disease,” said Cord Dohrmann, chief scientific officer of Evotec.
“We highly value Centogene’s biomarker expertise and real-world data-based global proprietary rare disease platform, which fits perfectly to our iPSC-based drug discovery platform,” Dohrmann said. “The combination should help identify disease-modifying treatments for this underserved patient population.”