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The mission of the CCDD is to discover novel small-molecule therapeutics for the treatment of cancer and progress them to hypothesis testing phase 1 clinicaltrials. We support the drug discovery projects of the CCDD with assay development and screening, biophysics, structural biology and recombinant proteinproduction.
My last stop at Arrakis Therapeutics is with a company targeting RNA with smallmolecules. I was enticed by Arrakis’ founder Jen Petter to serve in one more operational role, leading the research group at Arrakis as the company launched as one of the first biotechs to target RNA with drug-like smallmolecules.
Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated proteinproduction from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.
Unfortunately, in a Phase 3 clinicaltrial, treatment with remdesivir did not make a difference in mortality for Ebola virus disease patients. Without the stop sign, viral transcription no longer follows the correct order, which has a negative impact on viral proteinproduction,” says Fang.
Imprinting disorders can also result from differences between parental genders in gene expression – that is, proteinproduction from the encoded information. We were able to show that (S)-PHA533533 had better uptake and that the same smallmolecule could be translated in human-derived neural cells, which is a huge finding.
While small-molecule drugs continue to play a critical role in modern medicine, we are witnessing a shift toward the increasing development of amino acid-based therapeutics. Toxic peptides and proteins, particularly from the venoms of animals, are predicted to be a major source of next-generation peptide and protein-based drugs.
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