samedan logo
 
 
 
spacer
home > ebr > autumn 2007 > oligonucleotide drugs – within sight of the finish line?
PUBLICATIONS
European Biopharmaceutical Review

Oligonucleotide Drugs – Within Sight of the Finish Line?

Henrik Ørum at Santaris Pharma reviews new developments in the field of antisense therapies and considers how close we are to a breakthrough

The past decade has witnessed a tremendous increase in our knowledge of the human genome and the link between genes and disease. Soon, sequence and functional information will be available on all the human genes, of which thousands are expected to be attractive points for pharmaceutical intervention. Harvesting the medical potential of this vast number of novel targets holds great promise for better and safer treatments of more diseases. At the same time, however, it also presents a formidable challenge to the drug industry.

Part of the challenge is financial and relates to the excessive costs associated with the discovery and development of drugs against novel targets by contemporary approaches, such as small molecules and antibodies. Part of the challenge, however, is also technical and relates to the fact that many of the new gene targets encode intracellular proteins that act through protein-protein interactions – a class of proteins that have proven difficult, and often impossible – to target. Add to that challenge, the emergence of a novel, abundant class of noncoding, regulatory RNAs (microRNAs) that seems poised to become therapeutically-relevant targets and which will almost certainly be non-drugable by traditional approaches.

The concept of using short synthetic oligonucleotides as drugs (antisense therapy) provides the means to meet these challenges. Notably, antisense therapy is applicable to all targets and offers a number of advantages that can both shorten drug development time and reduce the risk of costly failures. These include the ability to rationally design the drugs for maximum target affinity and specificity; ease of synthesis and rapid selection/profiling of lead compounds in vitro and in vivo; predictability of ADME and toxicity profiles; and use of well established, uniform and cost-competitive processes for large-scale manufacture.


Read full article from PDF >>

Rate this article You must be a member of the site to make a vote.  
Average rating:
0
     

There are no comments in regards to this article.

spacer
Henrik Ørum is co-founder of Cureon A/S, now merged with Pantheco A/S to create Santaris Pharma A/S, where he acts as CSO and VP of Business Development. He was previously Director R&D and CSO at PNA Diagnostics A/S (formerly a subsidiary of Boehringer Mannheim and Hoffmann-La Roche). Henrik is also co-founder and a previous board member of Exiqon A/S – a diagnostics company that was floated on the Copenhagen Stock exchange in May 2007. He holds a MSc in Molecular Biology from the Technical University of Copenhagen, a PhD in Molecular Biology from the Medical University of Copenhagen and undertook post-doctoral studies at the Copenhagen Royal School of Pharmacy. He is the author and co-author of more than 40 scientific publications and books and holds several patents. He is often invited to speak at scientific and biotech conferences and symposiums.
spacer
Henrik Ørum
spacer
spacer
Print this page
Send to a friend
Privacy statement
News and Press Releases

Syntegon and Vetter win PDA Drug Delivery Innovation Award for Versynta microBatch

Waiblingen/Germany, October 6, 2021. Together with Vetter, an international pharmaceutical service provider for injectables, Syntegon (formerly Bosch Packaging Technology) has won the PDA Drug Delivery Innovation Award. In the "Partnership Innovation" category, the Parental Drug Association (PDA) honors development projects by pharmaceutical and machine manufacturers that advance the production of (bio)pharmaceuticals. The collaboration between Vetter and Syntegon resulted in Versynta microBatch, a highly flexible and fully automated production cell with a gloveless isolator, the smallest possible dimensions and a complete batch-to-batch changeover of less than two hours.
More info >>

White Papers

Challenges of Analytical Method Transfer in the Pharmaceutical Industry

RSSL

The development and validation of suitable analytical methods is a critical part of the overall drug-development life-cycle. For the majority of products, particularly those that are clinically successful, the transfer of the analytical method between laboratories will be required. This process is designed to verify that a given laboratory is capable of performing a test method for its intended purpose. This can be performed either internally (at the same company), or, with the on-going increasing trend in outsourcing, to an external Contract Research or Development organisation (CRO or CDO).
More info >>

 

 

 

©2000-2011 Samedan Ltd.
Add to favourites

Print this page

Send to a friend
Privacy statement