PIPELINE | CV-17
Antisense oligonucleotides (ASOs) are short, single-stranded oligodeoxynucleotide that interact with complementary messenger RNA (mRNA) to prevent translation of a targeted gene into protein.
Thus, synthesizing an antisense oligonucleotide with the complementary sequence can be a drug which represent to work by binding to RNA. ASOs can reduce, restore, or modify protein expression through several distinct mechanisms by designing any sequences of RNA, so it can apply to treat a vast array of diseases.
Intracellular delivery of ASOs is recognized as the major barrier to effective ASO activity within the target cell.
Therefore, to improve poor cellular uptake process, Cellivery used its technology Therapeuticmolecule Systemic Delivery Technology (TSDT) to improve cell-/tissue-permeability of ASOs. CP-17 can represent a new and valid approach to regulate the expression of disease-related genes and will greatly helpful for patients who are suffering from varying diseases.