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Opko Offers Glimpse at Oligo Medicines Efforts


Opko Health last month provided an update on its oligonucleotide medicines efforts, stating that it has programs in orphan neurologic and genetic disorders.

In early 2011, Opko acquired Curna, an early-stage developer of non-coding RNA-based drugs (GSN 2/3/2011). Since that time, however, the company has been quiet on the progress of Curna's programs.

In a filing with the US Securities and Exchange Commission last month, Opko said that Curna has developed a therapeutic approach called AntagoNat, which uses short, single-stranded oligos to interfere with natural antisense to up-regulate target protein expression.

Curna has obtained positive results in in vitro and animal testing and “has identified and developed compounds which increase the production of over 80 key proteins involved in a large number of individual diseases,” according to Opko.

“We have ongoing pre-clinical studies for several of these compounds, with an initial focus on orphan diseases” including the epileptic disorder Dravet syndrome, the neurodevelopmental disorder Rett syndrome, and the enzyme-deficiency condition MPS-1, the firm added.

Notably, Opko made no mention of its RNAi activities.

Opko was one of the first firms to get an RNAi-based drug into the clinic, advancing its wet age-related macular degeneration drug bevasiranib into phase III testing. However, in 2009, the company halted the drug's development on poor clinical data (GSN 3/12/2009).

It has not named any other RNAi drug-development programs since.