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Isis and Alnylam launch microRNA spin-out

By Mike Nagle , 10-Sep-2007

Two specialists in the field of RNA-based drug discovery have decided to launch a joint spin-out company to focus solely on microRNA (miRNA) research.

The new entity is the brain child of Isis Pharmaceuticals and Alnylam Pharmaceuticals, which have been collaborating together for over 3 years. This alliance has now culminated in the formation of Regulus Therapeutics, with $10m (€7.25m) of investment coming from Alnylam to balance venture ownership.


Once established, the two companies will then share the funding costs of research into miRNAs, an area they both already work on. These molecules regulate whole networks of genes that can be involved in disease and so could provide a new therapeutic approach to treating a plethora of diseases, including cancer, viral infection, and metabolic disorders.


Recently, the pharma industry has been paying more and more interest to RNA interference (RNAi) technology, where strands of nucleotides are used to therapeutically 'silence' genes that can either cause disease or enable it to prosper (it is also a useful research tool). microRNA molecules are small RNAs (around 20 nucleotides long) that can regulate a large number of disease. When improperly encoded, blocking their action could correct an entire disease pathway.


Regulus will be helped on its way by a new scientific advisory board, chaired by Dr David Baltimore, who shared the 1975 Nobel Prize in Physiology or Medicine for discoveries "concerning the interaction between tumour viruses and the genetic material of the cell."


This is the same award that Andrew Fire and Craig Mello won last year for their research into RNAi.


"The emerging biology of microRNAs points to a completely new understanding of cellular mechanisms for regulation of gene expression," said Baltimore.


"I believe that microRNAs represent previously unexplored disease targets where pharmacological approaches could lead to the emergence of novel therapies for many human disorders."


The two companies have granted Regulus exclusive rights to their intellectual property for miRNA therapeutics applications as well as other relevant patents.


"The opportunity to antagonise microRNAs could create a new frontier for pharmaceutical research where an entire disease pathway is targeted for intervention, not just a single disease target," said Dr John Maraganore, CEO of Alnylam.

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