Oligonucleotides: Promising Therapeutics With Significant Challenges

In recent years, interest in oligonucleotide therapeutics has resurged, as new approaches, clinical applications, advances in delivery, and a deeper biological understanding of these compounds emerge. However, expectations have been largely unfulfilled, as only three oligonucleotide drugs have been approved by the FDA for use in the United States as of August 2017.

Today, close to 200 clinical trials are registered at ClinicalTrials. gov in 2017, and as of 2016, more than 10,000 patients will have been treated with oligonucleotide drugs.1 Further, more than 120 companies globally are active in therapeutic oligo development,2 with the global market for nucleic acid aptamers expected to reach about $5.4 billion by 2019.

As a result, Contract Development & Manufacturing Organizations (CDMOs) with expertise in this field, such as CordenPharma, are experiencing increased demand for these services. However, while the outlook is positive, significant challenges remain.