Ricoh Develops a New Pathological Model for Rett Syndrome in iPSC-derived Neurons

Baltimore, MD, USA (August 30, 2024) －Elixirgen Scientific, Inc., a biotechnology leader focused on advancing drug discovery through hiPSC technology, announced today the launch of its new in vitro assay service for Rett syndrome that mimics the disease pathology. This cutting-edge assay was developed by Ricoh Company, Ltd, the parent company of Elixirgen Scientific, to address a critical gap in Rett syndrome research.

 

“This proprietary assay service we are announcing today offers researchers biologically and clinically relevant cell models to propel therapeutic discovery for Rett syndrome.” 

Keiki Sugimoto, Ph.D.
Chief Executive Officer
Elixirgen Scientific, Inc.

Rett syndrome, a devastating neurodevelopmental disorder primarily affecting young girls, has long posed challenges to the scientific community due to its complex and elusive disease mechanisms. The vast majority of typical Rett syndrome cases are triggered by sporadic mutations in the methyl CpG-binding protein 2 (MeCP2) gene.

Although hiPSC-derived cells with MeCP2 mutations are a useful in vitro model for disease mechanism analysis and drug development, the rarity of the disease, with only about 64,000 patients worldwide, makes obtaining patient-derived cells extremely challenging. An alternative is gene editing of hiPSCs derived from healthy donors; however, this approach requires significant cost and time.

In response to these challenges, Ricoh has developed a pioneering pathological model that accurately mimics Rett syndrome in hiPSC-derived neurons using shRNA knockdown of MeCP2.

This innovative in vitro model has shown a marked reduction in neuronal activity and neurite atrophy, two key indicators of Rett syndrome pathology, as observed in cells from patients with Rett syndrome. Furthermore, these phenotypes were rescued by brain-derived neurotrophic factor (BDNF) treatment. These results suggest that quantitative drug evaluation using neurite length and neuronal activity are useful indicators.

The new in vitro assay designed for Rett syndrome is anticipated to advance our understanding of the disease mechanisms and pave the way for fundamental treatments.

About Ricoh

Ricoh is a leading provider of integrated digital services and print and imaging solutions designed to support digital transformation of workplaces and optimize business performance. Headquartered in Tokyo, Ricoh's global operation reaches customers in approximately 200 countries and regions, supported by cultivated knowledge, technologies, and organizational capabilities nurtured over its 85-year history. In the financial year ended March 2024, Ricoh Group had worldwide sales of 2,348 billion yen (approx. 15.5 billion USD). It is Ricoh's mission and vision to empower individuals to find Fulfillment through Work by understanding and transforming how people work, so we can unleash their potential and creativity to realize a sustainable future. For further information, please visit www.ricoh.com.

About Elixirgen Scientific

Elixirgen Scientific, headquartered in the Science + Technology Park at Johns Hopkins in Baltimore, Maryland, is at the forefront of leveraging stem cell-based technologies to accelerate scientific discovery and the development of cures for a broad spectrum of diseases. Their products and services, including the revolutionary Quick-Tissue™ and Quick-Glia™ series, have positioned them as a premier partner in the global scientific community, committed to advancing human health through scientific excellence.

For Further Information, Contact:

Elixirgen Scientific, Inc.
[email protected]
(443) 869-5420