Therapeutics

OPKO-CURNA

OPKO-CURNA is developing a unique therapeutic modality focusing on the targeted up-regulation of protein levels via the targeted up-regulation of gene expression.

Up-Regulation Through Antisense

  • In contrast to RNA interference or gene therapy, our CURNA platform utilizes antagoNATs: chemically-modified, single-stranded oligonucleotides that interfere with the function of natural antisense transcripts (NATs).
  • NATs normally decrease the expression of the corresponding sense gene, so antagoNATs oppose this by suppressing the activity of a gene-specific inhibitor.
  • This then enables the up-regulation of specific endogenous proteins, both in vitro and in vivo.
  • OPKO-CURNA is currently screening over 400 gene targets.

Broad AntagoNAT Applicability

AntagoNAT Therapeutics

  • OPKO is currently conducting preclinical studies for proteins associated with orphan diseases such as Dravet syndrome, Rett syndrome, and MPS-1.
  • Animal studies confirm increases of target mRNA and proteins expressed by the target gene.
  • 8 AntagoNATs have been successfully tested in animals.
  • OPKO believes that antagoNAT therapeutics have the potential to treat a wide variety of illnesses, including cancer, heart disease, metabolic disorders, neurological disorders, and a range of genetic disorders.