Enabling Precision Medicine
Unlocking Oligonucleotide Therapeutics
At DTx, we are creating novel RNA-based therapeutics to treat the genetic drivers of disease. Our proprietary delivery technology platform utilizes fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. In preclinical studies, we have demonstrated cellular uptake and broad activity of oligonucleotides in the retina, muscle, heart, neurons, T cells and many other specialized cell types.
DTx believes its approach to delivering RNA medicines solves some of the issues that limited previous-generation platforms, including poor pharmacokinetics and insufficient cellular uptake.
DTx motifs drive cargo to muscle, heart and lung and, away from the kidney and liver.
DTx technology markedly enhances the residence time of RNA medicines in the circulation, markedly enhancing exposure to tissues beyond the liver.
DTx motifs enhance cellular uptake via receptor-mediated mechanism at doses equivalent to GalNac efficacy for hepatocytes.
By penetrating tissue layers that virus-based vectors cannot, DTx motifs can be enabling or complementary to other modalities.
DTx is currently exploring a library of motifs that selectively target specific cell types.
DTx is advancing several internal candidates in addition to partnering on several external programs.
Retinitis pigmentosa (RP) is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, the light sensitive tissue that lines the back of the eye. Common symptoms include difficulty seeing at night and a loss of side (peripheral) vision. DTx is developing an asset that can address specific genetic subtype of RP called P23H and a separate asset designed to address RP irrespective of the genetic mutation driving disease pathophysiology.
Duchenne muscular dystrophy
DMD is a degenerative disease affecting one in 2,000 boys, whereby a genetic mutation prevents the body from producing dystrophin, a protein that muscles need to work properly. Without dystrophin, muscle cells become damaged and weaken. Duchenne is an irreversible, progressive disease with no cure.
It is known that even if a small amount of functional protein can be made, the disease is significantly ameliorated. Current scientific efforts are focused on creating drugs that allow for production of a truncated, but functional dystrophin protein. The FDA has approved one such drug but its efficacy remains uncertain. Among several reasons, only low levels of the drug reach affected muscle. DTx technology appears well suited to solve this problem in Duchenne, as well as other conditions where exon skipping can be used to produce a functional protein.
Neurodegenerative Diseases of the Central Nervous System (CNS)
Undruggable targets in the CNS cause many of the most serious, debilitating, and life-changing human maladies, including Alzheimer’s, frontotemporal dementia and Huntington’s disease. DTx Pharma has been awarded multiple grants from the NIH (NCATS, NIA), and foundations including the TPEP program of the Alzheimer’s Association and Rainwater Charitable Trust and the CMTRF to advance its efforts to treat CNS diseases.
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DTx is rapidly growing and is currently recruiting scientists across chemistry, biology and in-vivo disciplines. Please see link for a list of current open positions.