Genervon scientists have confirmed that the target of GM604 regulates over 4,000 genes. More significantly, it modifies ALS disease progression by modulating at least 30 significant ALS related genes. It does this through at least 8 pathways, potentially more, and up to 22 biological processes. These impressive and broad effects are totally consistent with the role of a master regulator of motoneurons and nervous system in embryonic development. They also offer tremendous potential for broader benefits in fighting ALS and tackling its many disease alterations.
GM604 also proved to be quite effective in the disease modification in ALS animal model with mutant SOD1. It led to improved clinical scores in SOD1 mice (p<0.001 for all groups compared to control). Moreover, GM604 delayed the onset of ALS symptoms by 27%, extended life by 30% and delayed the median clinical score deterioration time in ALS mice by 41%.
GM604 also provided neuroprotection against soluble inflammatory factors from ALS human patients' Cerebral Spinal Fluid (CSF) in vitro by 175%.
GM604 dramatically increased the survival life span by 500% (6 fold from 7-14 weeks to 55-65 weeks) and increased preservation of motoneurons by 160% (2.6 fold) in a Wobbler Mice Model for motoneuron diseases such as ALS.
Genervon has discovered a novel endogenous embryonic stage family of 9 human Master Regulators of the Nervous System. It has obtained 33 Composition of Matter and Use patents. The company has developed one of the Master Regulator named GM600 for disease modification in neurodegenerative diseases and for neuroprotection. A GM600 serial number is assigned to each indication. Ischemic stroke and Parkinson's disease are in Phase 2 Clinical Trials.
The discovery of endogenous embryonic stage human master regulators is changing the drug development paradigm from hitting single gene/pathway to a comprehensive and dynamic multi-factorial approach to treat the complex neuro-degenerative diseases. Genervon is at the forefront of a drug development paradigm shift that is beginning to be appreciated.