3D brain model will be used in pursuit of therapies
Stem Pharm, Verge Genomics developing a 'state-of-the-art Parkinson’s disease model'
The collaboration will leverage Stem Pharm’s human-derived “mini-brains” that model inflammation of the brain — neuroimmune organoids — and Verge’s expertise in human genomics and machine learning technology to identify and substantiate potential therapies.
“Verge’s all-in-human approach to drug discovery aligns perfectly with our ideology, and by combining our strengths and resources, we aim to establish a state-of-the-art Parkinson’s disease model,” Steven Visuri, PhD, Stem Pharm’s CEO, said in a press release.
“This collaboration represents a significant step forward in our collective mission to discover transformative therapies for patients suffering from this debilitating disease,” Visuri added.
Parkinson’s disease’s complexity and variability have limited the development of effective therapies and is one of the diseases with the lowest drug approval rate.
Knowing the urgent need for innovative approaches, the two companies partnered to create a Parkinson’s disease model based on neural organoids of human tissue derived from stem cells.
Creating the 3D brain model
Organoid models were developed as a tool for studying the mechanisms of human diseases and testing responses to therapies. They develop as a 3D self-organizing structure that mimics features of the original tissue.
Stem Pharm’s human neural organoids are produced from human induced pluripotent stem cells (iPSCs), and are composed of neurons, astrocytes — brain cells that support neuronal function and health — vascular cells, and microglia, which are the brain’s resident immune cells.
iPSCs can be used to generate any kind of organoid. They are fully matured cells reprogrammed back to a stem cell state and capable of growing into almost any type of cell.
The model provides a platform to study diseases that are associated with neuroinflammation — inflammation of the nervous system, including neurodegenerative diseases such as Parkinson’s disease. It can be used to analyze underlying disease pathways and identify and validate new therapeutic candidates.
The ConVERGE discovery platform
Verge has created the ConVERGE discovery platform, by combining human genomics with advanced computational tools to discover new drugs with a high probability of success. It uses human brain tissue obtained directly from patients to predict new targets based on gene analysis combined with a machine-learning system.
“When combined with genetics, this reveals targets that are [three times] more likely to succeed in the clinic,” Verge wrote on its website.
The targets will be validated using neural organoids to mimic Parkinson’s in a laboratory setting, contributing to early drug discovery.