GM6 A Disruptive medical discovery of A Regulator for Central and Peripheral Nervous System Disorders
September 22, 2015 Pasadena Calif. -- Genervon Biopharmaceuticals LLC ("Genervon") today reported that it has produced data, which has been reviewed and confirmed by an external and independent bioinformatics CRO, demonstrating that its clinical stage signal peptide GM6 being developed for Amyotrophic Lateral Sclerosis (ALS) as well as other neurodegenerative disorders, modulates many genes associated with the nervous system, many of which are known to have significant impact on defense against stress and maintenance of cellular homeostasis. The independent CRO has analyzed the complete expression profile of cells treated with GM6 versus controls (7 folders and 83 files, figures and tables) which can be organized into 24 known pathways for neurological development, maintenance, and repair. The number of specific genes modulated by GM6 in one cell line (SH-SY5Y neuroblastoma cells) was as follows:
15597 genes measured on array, 13452 expressed genes
- Strong increase (FDR < 0.10, FC > 2.00) → 581 genes
- Mild increase (FDR < 0.10, FC > 1.50) → 1418 genes
- Weak increase (FDR < 0.10, FC > 1.00) → 1600 genes
- Strong decrease (FDR < 0.10, FC < 0.50) → 678 genes
- Mild decrease (FDR < 0.10, FC < 0.67) → 789 genes
- Weak decrease (FDR < 0.10, FC < 1.00) → 998 genes
As shown above, > 1,200 genes showed strong expression shifts in response to GM6 treatment, based upon the most stringent statistical thresholds. However, given less conservative statistical criteria, > 3,000 genes showed altered expression. This affirms that GM6 has major regulatory effects in human neuronal cells. GM6 impacting so many different genes, both upregulating and downregulating expression to achieve homeostasis and the larger peptide MNTF from which it is derived, can be classified as "master regulators" of the human nervous system.
Genervon's drug candidate GM6 is a peptide derived from the active site of human growth factor motoneuronotrophic factor ("MNTF"), a multifactorial regulatory peptide that governs the differentiation, development, protection, and correction of the human nervous system during fetal development. MNTF is highly specific to the human nervous system and is expressed rapidly during the first trimester, peaking at week nine. It was initially discovered by Genervon using a novel methodology called "protein band-fishing by cells". Genervon subsequently determined the 33mer polynucleotide sequence of MNTF as well identifying the 9 active sites, one of which is the source of the small 6 amino acid sequence peptide that can replicate many of the activities of the larger peptide from which it is derived.
Genervon is developing GM6 for a range of neurological disorders, with a primary focus on neurodegenerative diseases including ALS, Parkinson's disease (PD), Muscular Dystrophy (MS), Huntington Disease (HD) and Alzheimer's disease (AD). A recently completed Phase IIA trial in ALS demonstrated effects consistent with a slowing progression of the disease.
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Company Name: Genervon Biopharmaceuticals LLC
Contact Person: Emily Wang
Country: United States