Empowered Patient Podcast Repressing Expression of Disease-Causing Genes with Dr. Arthur Suckow DTx Pharma
Dr. Arthur Suckow is the Co-Founder and CEO of DTx Pharma, in search of an approach to administering sRNA therapeutics using a solution of fatty acids, which every cell in the body has a mechanism to take up. Leveraging those mechanisms, receptors, and transporters, the DTx concept is to trick the cells into bringing the sRNA molecules in so they can repress the expression of disease-causing genes.
Arthur explains, "Well, the one that everyone is familiar with now because it's on the front page of the news almost every day are mRNA modalities where you put a gene essentially back into cells. The poster child for that is putting the spike protein in to generate a vaccine response. But that's not what we do. We actually inhibit the expression of disease-causing genes. When you have an extra copy or a mutant protein that mucks up cellular function, we try to inhibit the expression of those genes. And those are called sRNA or antisense therapeutics. We specifically focus on the flavor called siRNA as our kind of modality of choice."
"The bigger challenge and the challenge that DTx is trying to take on is to enable this class of therapeutics beyond the liver to other tissues. We focus on the peripheral nervous system, muscle, brain, etc. Where they're most effective from a disease perspective is if you have expression of a gene or the expression of a mutation. We usually think dominant gain of function is kind of our bread and butter. So those kinds of lesions are where these are particularly effective."
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