StemoniX® Uses microBrain® 3D, a Functional In Vitro Human Neural Model, to Identify Potential Treatment Modalities for Rett Syndrome
MAPLE GROVE, Minn., Feb. 14, 2019 /PRNewswire/ -- StemoniX® Inc., a biotech company revolutionizing how new medicines are discovered, announced today that Cassiano Carromeu, PhD, lead neuroscientist at StemoniX, presented research during the SLAS2019 annual meeting demonstrating the ability of microBrain® 3D to produce a functional in vitro disease model for Rett Syndrome (RTT) via human induced Pluripotent Stem Cells (hiPSCs) derived from RTT patients. Importantly, the findings specifically demonstrated how the microBrain 3D platform and multiparametric analysis rapidly identified novel therapeutic compounds to potentially treat Rett Syndrome, which has no cure, and could be applied more broadly as a paradigm to accelerate drug discovery efforts for neurodevelopment disorders in general.
During the podium presentation, titled, "3-Dimensional Human Cortical Neural Platforms for Drug Discovery in Neurodevelopmental Disorders," Dr. Carromeu described several studies involving microBrain 3D, a highly homogenous human cortical spheroid screening platform. Data from these studies collectively demonstrated the platform's ability to produce consistent functional neuronal signaling across wells, plates, and manufacturing batches, as well as its applicability for interrogating large libraries of compounds based on their ability to modulate human neurological activity. These breakthroughs enabled StemoniX's scientists to use hiPSCs derived from RTT patients to develop a disease-specific RTT microBrain 3D model that displayed a compromised neural network with a clear abnormal phenotype based on calcium signaling. A focused screen using a targeted compound library then rapidly identified compounds that rescued the abnormal phenotype, representing the discovery of potential therapeutic approaches at an unprecedented rate and cost-savings.
"This research signals an important advancement in finding new therapeutics for diseases of the central nervous system," stated Ping Yeh, co-founder and CEO of StemoniX. "Scientists have long sought to develop effective in vitro models of the human central nervous system, however the unique structural organization that enables the CNS to perform its complex functions is not easily reproducible. StemoniX developed microBrain 3D to address this significant opportunity, and we believe the data presented at SLAS2019, particularly our ability to generate a functional in vitro Rett Syndrome model, exemplify the technology's potential to dramatically change the current drug discovery paradigm for neurodevelopmental disorders and other neural diseases."
StemoniX is empowering the discovery of new medicines to treat challenging diseases via the world's first ready-to-use assay plates containing living human microOrgans®, including electrophysiologically active neural (microBrain®) and cardiac (microHeart®) cells. Predictive, accurate, and consistent, StemoniX's products enable pharmaceutical scientists to quickly and economically conduct high-throughput screening in a simplified workflow. Through their Discovery as a Service branch, the company also partners with organizations to screen compounds as well as create customized microOrgan models and assays tailored to individual discovery and toxicity needs. Visit www.stemonix.com to learn how StemoniX is helping global institutions humanize drug discovery and development to bring the most promising medicines to patients.
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