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C. Anthony Altar, PhD, Senior Scientific Fellow and Advisor of Verge Genomics, delivers a thorough overview of new mechanisms and treatments for brain illnesses.

Tony is helping to guide Verge Genomics forward with his 35+ years of experience helming CNS drug discovery and development teams, from initial concept to commercial productivity and success. Altar is the former Global Head of Neuroscience at Otsuka. During Altar’s tenure he led his team in the discovery and FDA approval of aripiprazole (Abilify™) for multiple uses in bipolar disease, depression, as well as schizophrenia. Altar was also the Chief Scientific Officer at Assurex Health, CSO of Psychiatric Genomics, and he co-founded the Regeneron vision program, and led teams at Genentech.

Altar discusses his background and how he became interested in the brain and the potential problems that can develop. He talks about his time as a high school student when there was a lot of use of psychedelic drugs in the community. As a curious high school scientist in the making, Altar’s scientific mind wondered… if a single molecule could create temporary psychosis and states of euphoria, etc. then could the mechanism of psychiatric illnesses perhaps share the same simple feature? Completing his high school studies in advance and before attending UCLA, Altar began a volunteer position at the Neurobiochemistry Lab at the Brentwood Veteran’s Administration, which accelerated his interest in disease and brain issues.

As Altar explains there are approximately 20,000 genes in the human body and the brain expresses almost half of those. Fortunately, it is now possible to study all 20,000 of them to understand the coding of proteins and measure mRNA in diseased tissue. The mRNA is ‘messenger RNA,’ a subtype of RNA. The mRNA are molecules that carry a portion of the DNA code to other parts of a cell for the creation of proteins.

Altar discusses cell types including neurons and how various brain diseases impact them. He explains how some neurons are more vulnerable and thereby more susceptible to succumb to disease. He talks about the various methods and companies that are working toward drug and gene therapy solutions to treat schizophrenia, Alzheimer’s disease, ALS,  and other CNS related health issues. Altar explains the mechanisms of Parkinson’s disease and how disease-causing changes can be very subtle. He explains the loss of dopamine neurons and the impact that has, and he details some of the protective mechanisms and explains why cells die.

Altar talks extensively about gene therapy, including trans-splicing and brain injection techniques that are utilized for better results due to the fact that the brain is not easy to access versus intravenous injections that are much less likely to enter the brain.

Verge was founded by some of the industry’s best machine learning experts and neuroscience drug developers. Altar and his fellow Verge contemporaries are excited about the amazing advances in computational genomics and new insights into neuroscience that are creating great progress in drug discoveries to combat neurodegenerative diseases.


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