During the 1990's there was great hope for the great promise of bio drugs for the 21st century, with new scientific discoveries and understanding, new genome projects and new tools.
New Wine in Old Skin
The classical way of thinking in drug development from chemical to bio drug is still dominated by the paradigm of trying to find a highly selective drug at a single target and show that changing that target changes the outcome.
Looking for the Master Regulators in the Womb
Throughout the embryonic development process, there are crucial master regulators that oversee cell proliferation/apoptosis, differentiation, repair, inflammation/anti-inflammation, and overall developmental balance. Embryonic development is incredibly intricate and yet is accurately repeated with remarkable consistency.
Genervon set out to find and found the specific master regulators. One of the master regulators discoveries is motoneuronotrophic Factor (MNTF) that participates in regulating embryonic nervous system development. We hypothesis and confirmed that it is able to correct inadvertent errors in fetal development and thus be life sustaining and potentially therapeutic
Finding the Active Sites of Master Regulators
For drug development purposes, a smaller drug target is more desirable. Within the original MNTF 33mer sequence, Genervon scientists developed a proprietary In Silico Analysis platform and further identified nine active sites and multiple drug targets.
In vitro and in vivo experiments have confirmed that all identified targets have statistically similar biological effects as the MNTF. GM6 the chosen drug candidate penetrates BBB and has shown neuroregenerative and neuroprotective properties in pre-clinical studies. GM6 is safe and well-tolerated in human subjects at very high and repeated doses.
CNS Disorders & Diseases
Genervon hypothesis and recent genomic, proteomic, and systems biology studies confirmed that Central Nervous System (CNS) disorders and diseases involve the interplay of a highly complex, multi-factorial process of many non-dominant effectors in an interwoven dynamic network.
That reduced the probability that single-action drugs will demonstrate efficacy in CNS diseases because, at best, they could interrupt only a part of the pathological process.
From Function to Pathways
DNA micro-array analysis indicated that GM6 up- or down-regulates, by at least two fold, more than 1,600 genes. GM6 likely functions as a master regulator that signals through multiple pathways to induce the expression of anti-inflammatory, anti-apoptotic and anti-oxidant proteins.
Many of the modulated genes are related to nervous system disorders and diseases, and neurodegenerative diseases. GM6 modulated significantly genes through pathways such as PI3K-AKT/p53. These analyses showed that GM6 activates genes involved in 22 specific biological functions including neurogenesis, neural development, neuronal signaling, etc.