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GM604 Is a Multi-Target Regulator That Provides a Novel Treatment Strategy for ALSGenervon Biopharmaceuticals LLC ("Genervon") is presenting its recent findings at the 28th national symposium on ALS/MND in Boston on Saturday, December 9, 2017. Genervon has discovered and developed GM604 (GM6), a multi-target regulator that provides a novel therapeutic strategy for treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. GM6 is a 6-amino acid peptide drug modeled upon an endogenous embryonic stage neurotrophic factor isolated from the developing nervous system. Using a systems biology approach, we discovered that GM6 has multi-target effects consisted with a neurotrophic factor, leading to improved survival of neurons in neurodegenerative patients. Amyotrophic lateral sclerosis (ALS) is a fatal heterogeneous neurodegenerative disease that lacks effective treatment option. Genervon hypothesized that the traditional single-target approach to treat neurodegenerative disease may not be effective due to the complexity of these conditions and their multifactorial pathogenesis. We have shown that GM6 promotes neuroprotection against toxic factors contained in SF from patients with neurodegenerative diseases and importantly can cross the blood brain barrier. In the SOD1 mouse model of ALS, we demonstrated that GM6 delays disease onset and reduces mortality, with concomitant improvements in physiological function. Finally, the data in our Phase 2A clinical trial of only 2 weeks (6 doses) suggested a positive signal of GM6 in ALS patients with favorable shifts in disease biomarkers and functional measurements. The data supports further study. A Phase 3 study based on a longer treatment period and larger sample size is now being planned. RNA-seq was used to determine whether ALS-associated genes exhibit unique responses to GM6 treatment in SH-SY5Y cells, which would suggest that GM6 is impacting signaling pathways linked to processes that underlie ALS onset and/or progression. Bioinformatic analyses identified GM6-regulated genes with potentially important roles in ALS development and progression. These genes were categorized with respect to 6 hypothesized mechanisms of action (MOA). This MOA posits that GM6 protects neurons by combating oxidative damage while bolstering neurogenesis, microtubule stability, immune- mediated neuroprotection, synaptic transmission and glutamate clearance. Genervon is happy to report the successful filing of patent application for the pathways and genes regulated by GM6 in association with ALS and neurodegenerative diseases. Link to Genervon's presentation https://www.genervon.com/s/2017-12-06-GM604-ALS-poster.pdf.
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