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ENABLING GENE THERAPY

GuideTx is solving the challenge of DNA and RNA delivery

Gene therapy is a reality

Drugs composed of DNA or RNA can specifically turn any gene on or off, which allows them to treat disease at the genetic level. However, safely and specifically delivering gene therapies to diseased cells is a significant challenge.

One type of delivery system, named a lipid nanoparticle (LNP), has delivered RNA to hepatocytes in an FDA approved drug. But targeted delivery to other cells is required for gene therapies to reach patients afflicted with other diseases.

Guide Therapeutics pioneers new gene therapies by developing field-leading drug delivery vehicles. Using a proprietary screening platform that performs thousands of drug delivery experiments directly in vivo, we identify nanoparticles that deliver DNA or RNA to cell types others cannot reach. We then focus on manufacturing, product development, and target selection, in order to develop drugs internally or partner.

We deliver gene therapies

Guide has developed methods to perform thousands of in vivo drug delivery experiments at once. Each method is tailored to the payload: mRNA delivery is studied using mRNA, and siRNA delivery is studied using siRNA. Our approach, which has led to awards including Top 10 Emerging Technologies in the World by the World Economic Forum, allows us to pursue gene therapies in cells that cannot be targeted otherwise.

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SCIENTIFIC AMERICAN | 2019

Could the Next Big Information Technology Be ... DNA? How DNA is used to store—and generate-information at extreme scales

In this piece, Guide co-founder James Dahlman describes how DNA can be used to store information. The DNA barcoding process Guide Therapeutics uses to efficiently create safer gene therapies is highlighted.

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JACS | 2018

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

Using DNA barcoding, nanoparticles that deliver siRNA to bone marrow endothelial cells are identified. This paper includes two important scientific advances. First, bone marrow endothelial cells had not been efficiently targeted by nanoparticles before. Second, the paper utilizes iterative nanoparticle barcoding; the chemical structures that promoted nanoparticle delivery in the first experiment informed the design of nanoparticles in the second experiment.

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