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An update on oligonucleotide drug development

Vice President of Development, Nitto Denko Avecia Inc.
8560 Reading Rd, Cincinnati, OH 45215 USA

ABSTRACT: Oligonucleotide therapeutics are a new class of drugs with only three commercialized drugs to date. In spite of this limited commercial success, the oligonucleotide field continues to grow rapidly.  Many drugs have advanced to late clinical phases. A variety of drug delivery strategies have proven effective, and improvements continue to be made in the manufacturing technologies used to produce oligonucleotides. Growth in the oligonucleotide field is fueled by the renewed interest of large pharmaceutical companies and the emergence of startup companies with new intellectual property. The oligonucleotide field appears to be poised for success.
KEYWORDS: Oligonucleotide therapeutics; solid phase oligonucleotide synthesis; drug development; drug delivery.


Oligonucleotide therapeutics have tantalized drug developers with the promise of rational drug design, lower drug development costs, and the ability to reach targets that conventional small molecule drugs cannot. Three oligonucleotide drugs have been approved by regulators, but oligonucleotides have not yet fully delivered upon their elusive promise. Large pharmaceutical companies have entered, exited, and then re-entered the oligonucleotide field in dramatic fashion. In 2012, Dr. Art Krieg, co-founder of Coley Pharmaceutical Group and now CEO of Checkmate Pharmaceuticals, summarized the evolving perspective of pharmaceutical companies towards oligonucleotides. Dr. Krieg stated that the first phase is an irrational exuberance that is followed by excessive skepticism when problems arise. Excessive skepticism is then followed by resurging enthusiasm and reinvestment as products are developed (1).  Unfortunately, the period of excessive skepticism towards oligonucleotides also coincided with the economic turmoil of the Great Recession resulting in several very bleak years for the field. Venture capital was difficult to obtain, and frequently small biotech companies were forced to scale back or delay clinical trials due to lack of funding. The last several years have been marked by a resurging enthusiasm in oligonucleotide therapeutics.  Numerous clinical candidates have advanced, and several drugs appear poised for regulatory approvals.  Big pharmaceutical companies seem to once again be investing in oligonucleotides through licensing deals, partnerships, and acquisitions. An example of this shift in enthusiasm is the Roche acquisition of Danish company Santaris Pharma for $250M upfront and potential payout of $200M based on achievement of certain predetermined milestones (2). This announcement came in August 2014, nearly four years after Roche announced it was restructuring and discontinuing its RNAi research center in Kulmbach Germany. Raw material suppliers, equipment manufacturers, and contract manufacturing organizations have all responded to the renewed interest in oligonucleotides, and they continue to innovate in order to meet the changing needs of the market. 


The number of oligonucleotides currently in clinical trials is still small relative to other therapeutic classes.  According to Nitto Denko Avecia internal research, just 116 oligonucleotides were in active clinical trials as of August...
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