Stemonix and Atomwise Join Forces to Develop Small Molecules Targeting Rett Syndrome

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by Alice Melao |

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StemoniX and Atomwise will work together to discover and develop small molecules that could be potential new therapies for people with Rett syndrome.

This collaboration will take advantage of StemoniX’s human microOrgan platform and combines it with Atomwise’s artificial intelligence technology to expedite the development process and increase the chances of clinical success of new therapeutic molecules.

“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,” Ping Yeh, co-founder and CEO of StemoniX, said in a press release.

“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,” he added.

Researchers at StemoniX use human induced pluripotent stem cells (iPSCs) to build ready-to-use functional tissues that mimic the biological responses of human organs. iPSCs are developed by reprogramming cells to revert to an embryonic-like state which are then able to differentiate into all cell types.

With the company’s microOrgan platform, researchers have used iPSCs collected from patients with Rett syndrome to build 3D microbrains made of nerve cells and astrocytes (cells that support neurons, respond to injury, and regulate blood flow and inflammation in the brain). Such microbrains recapitulate features of the disease.

This platform could provide greater accuracy and consistency to clinical trials, according to the company.

In turn, Atomwise’s artificial intelligence technology uses statistical approaches that were designed to overcome limitations of current preclinical treatment discovery and development methods.

It combines information from millions of measurements and thousands of protein structures to predict the binding of small molecules to protein targets. This approach makes it possible to pursue a comprehensive therapy discovery with unparalleled precision and accuracy, according to Atomwise.

“New technologies and approaches are vital to address the needs of patients,” said Abraham Heifets, PhD, 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.”