Standardizing cell models is essential for drug discovery and investigating mechanisms influencing disease onset. Using iPSC-derived neurons and glia marks a revolutionary shift in drug discovery, enabling direct study of novel therapeutics on human neurons without relying on primary human tissue. By creating specific neuron and glia subtypes, researchers can delve into disease pathways, comparing edited to control cell lines to aid in developing therapeutics to treat those diseases.
To accelerate the drug discovery workflow focused on curing neurodegenerative and neuro-related diseases, Synthego and BrainXell’s partnership is streamlining this workflow by leveraging Synthego’s CRISPR expertise and BrainXell’s mastery of working with iPS cells and neurons to make curing those diseases within reach.
Key learning objectives will include:
- Synthego’s CRISPR-based ECLIPSE™ platform capabilities and its design for high-throughput generation of knock-in iPS cell models.
- BrainXell’s extensive abilities take iPS cell models to differentiated neurons to efficiently run complex assays with ease.
- Two Examples:
- Synthego played a pivotal role in the iPSC Neurodegenerative Disease Initiative (iNDI) spearheaded by the National Institutes of Health (NIH) by generating over 264 clones, encompassing 22 gene targets with 12 variants each, all within a remarkable timeframe of under six months.
- BrainXell’s power to easily create disease models like APOE E4/E4 and TREM2 edited lines in cortical glutamatergic neurons, cortical astrocytes, and microglia which could be used to perform complex functional assays.