Pasadena, Calif., Multiple targets, highly effective, breakthrough therapy for ALS sufferers may now be within reach. Winston Ko, the CEO of Genervon Biopharmaceuticals, will be presenting on Friday at the Neurotech Conference in San Francisco that its endogenous regulatory drug GM604 modulates over 80 ALS related gene targets interactively and dynamically through 12 pathways and up to 22 biological processes, including neurogenesis, neural development, signaling and transport.
GM604 is not a single target drug or a cocktail of single target drugs for ALS. It is an endogenous, embryonic/fetal stage, multiple target, and multifactorial human master regulator peptide. It controls more than 4000 genes regulating the development and protection of the human nervous system. For example, in the axonal transport stimulation pathway #8, GM604 improves axonal transport and delays ALS onset by down regulating SOD1, restoring damaged mitochondria, and up regulating kinesin and dynactin genes, involving 25 gene targets in this pathway alone.
Scientists now have enough evidence to confirm that ALS is a complex, multi-system, neurodegenerative disorder. That is one of the main reasons why clinical trials of single target drugs have uniformly failed. Genervon hypothesized in the early 1990s that neurological diseases involve the interplay of multiple target processes of many non-dominant factors in an interactive dynamic network. This insight has guided the drug development strategy of Genervon from the beginning many years ago.
GM604 was discovered by Genervon scientists using proprietary technologies. With peaked expression in human placenta at week 9 of gestation and also expressed in many human fetal tissues, GM604 plays a significant role in nervous system development, protection and correction. This discovery is changing the drug development paradigm from hitting single gene/pathway to a comprehensive, dynamic, multiple target approach that efficaciously treats complex neurodegenerative diseases.
The impressive effects of GM604 are consistent with the role of a master regulator of the nervous system in embryonic development. ALS has many forms with different pathogeneses. Previous single target drug trials restricted the enrollment to a small, selective segment of ALS patients. Since GM604 controls multiple ALS gene targets through multiple pathways, Genervon is proposing a broadly inclusive ALS Phase 2 clinical trial.
Genervon is an innovative clinical stage biopharmaceutical company providing breakthrough biological drugs for some of the world's critical unmet medical needs.
Genervon Biopharmaceuticals LLC