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
- The antagoNAT approach, based on research developed by the University of Miami’s Claes Wahlestedt, may be applicable to many different genes, and recent data published in Nature Biotechnology indicates that natural antisense is a common phenomenon in the human genome.
- OPKO-CURNA has currently developed antagoNAT molecules able to up-regulate over 80 essential proteins.
- 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.