Companies to focus on small molecule discovery for Rett syndrome
Maple Grove, MN and San Francisco, CA /PRNewswire/ - StemoniX, Inc. and Atomwise Inc. today announced a joint venture that combines StemoniX's human microOrgan® platform with Atomwise's AI technology to enable the rapid discovery and development of novel small molecule therapies. The joint venture will initially target Rett syndrome, a rare neurodevelopmental disease.
Rett syndrome is a severe neurological disorder due to mutations in the MECP2 gene. The disorder often manifests in infant girls and results in impairment of intellectual and motor development, seizures, and loss of speech. There is no known cure for Rett syndrome.
StemoniX is a leader in developing models for specific diseases via its microOrgan platform, the world's first ready-to-use assay plates containing living microtissues engineered from human induced pluripotent stem cells (iPSC). Atomwise is the leader in using artificial intelligence (AI) for small molecule drug discovery. In the joint venture, Atomwise will use its AI technology to analyze billions of compounds to identify potent and selective binders for proteins that are important for Rett syndrome. StemoniX will test the compounds on its human microBrain® 3D disease model of Rett syndrome to determine biological efficacy.
Traditional drug discovery and early drug development require thousands of research hours and millions of dollars to identify a potential therapeutic for clinical development. Despite the time and expense, the vast majority of potential drugs – 90% – ultimately fail in the clinic. The high costs and high failure rate often make it infeasible to pursue drug discovery and development for rare diseases. It is especially challenging for rare neurological diseases due to the complexity of human brain function, which is not well replicated in animal models.
Next generation technologies, such as AI and human microOrgans, have the potential to transform the speed of drug discovery. The combination of AI with human microOrgans can reduce both time and cost while increasing the chance of clinical success by focusing on only those compounds with novel chemical structures that are predicted to be most effective in a relevant human system. Streamlined efforts with faster development times and lower failure rates are critical for the discovery of new therapies for patients with rare and challenging neurological diseases such as Rett syndrome.
"The joint venture brings together the complementary technologies of StemoniX and Atomwise, creating an opportunity to go from model to molecule to validated drug in a fraction of the time and cost required with traditional methods," said Ping Yeh, StemoniX Co-founder and CEO. "Atomwise is a great partner for a notoriously challenging disease area. Their expertise will enable drug discovery on historically undruggable targets and provide new opportunities for treating rare neurological diseases."
"New technologies and approaches are vital to address the needs of patients," said Abraham Heifets, Ph.D., Co-founder and CEO of Atomwise. "StemoniX has made remarkable progress with their microBrain 3D platform and its application to Rett syndrome. It is a potentially powerful tool to rapidly evaluate compound efficacy and assess their suitability for clinical trials, and we are excited to partner with them."
StemoniX is accelerating 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 combined with its proprietary data management and analytical tools (AnalytiXTM) are revolutionizing traditional drug discovery and development by radically improving the speed, accuracy, and costs required to identify new drugs and conduct initial human toxicity and efficacy testing. Through its Discovery as a Service offering, the company partners with organizations to screen compounds as well as to create customized microOrgan models and assays tailored to specific 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.
Atomwise Inc. invented the first deep learning AI technology for structure-based small molecule drug discovery. Created in 2012, today Atomwise performs hundreds of projects per year in partnership with some of the world's largest pharmaceutical and agrochemical companies, as well as more than 200 universities and hospitals in 40 countries. Atomwise has raised over $50 million from leading venture capital firms to support the development and application of its AI technology. Learn more at atomwise.com or follow @AtomwiseInc.