Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease characterised by motor neuron degeneration. The development of robust models of ALS remains a significant challenge for drug development and preclinical validation studies. BrainEver is a preclinical stage biotech targeting ALS with an innovative homeoprotein therapy approach. In vivo studies using a mouse model with ALS-like phenotypes has demonstrated a strong therapeutic potential for the homeoprotein Engrailed 1 in ALS. The use of patient-derived iPSCs and subsequent differentiation into motor neurons represents a unique opportunity to recapitulate disease phenotypes and test disease-modifying drugs. Modelling TDP43 pathology is of notable interest due to its observed presence in both familial and sporadic forms of ALS. BrainEver has been investigating if 糖心原创 ioMotor Neurons harbouring a TDP43 mutation demonstrate disease-associated mutations such as TDP43 mis-localisation, TDP43 aggregation and the expression of cryptic exons which can serve downstream for drug development assays.