Antisense oligonucleotides (ASOs) in drug development

A developing field of medicine is based on therapeutical oligonucleotides. In recent years different types of oligonucleotide-based drug products have been approved by health authorities, among other the EMA and FDA. One of these types are antisense oligonucleotides (ASOs). ASOs are synthetically modified single stranded RNA or DNA molecules, that bind to a complementary target RNA strain. They can modulate protein expression by either RNA cleavage or RNA blockage.

An interesting application of ASOs is that custom development for a specific genetic variant is possible. Therefore, they can be used for treatment of extremely rare diseases (e.g. for cases where only 1 or 2 patients with a certain mutation have been identified). Often such genetic disorders are life-threatening and rapidly progressing. In such cases early contact with the agencies to discuss the clinical and filing strategy is of importance and orphan designation can be assigned.

Besides the application of ASOs for genetic disorders, also drug products against viral diseases are being developed. ASOs can either directly target the viral RNA to prevent it from being translated into viral proteins or target host cell genes that are required for viral replication.

At STARoDub B.V. we are very interested in new fields of medicine development. For more information about this topic and the regulatory challenges that come with it, you are welcome to reach out to our company.