RNAi therapeutics market hots up

By Gareth Macdonald

- Last updated on GMT

Related tags Rna interference Rna

Novartis’ extension of its collaboration with Alnylam Therapeutics and Roche’s acquisition of gene silencing specialist Mirus Bio Corp for $125m (€78.5m) suggest that big pharma is beginning to recognize the commercial potential of RNAi-based therapeutics.

RNA interference offers a way of blocking protein production without the problems of strand invasion and triple helix formation common to gene silencing. Although there are technical hurdles to be overcome, primarily related to effective delivery, the technology can be applied to the large number of diseases caused by aberrant protein expression.

Although the majority of RNAi drugs have been developed for gene therapy, last month Calando completed the first trial of its siRNA nanoparticle CALAA-01 in a cancer patient. The increasing demand for effective medications coupled with the broad applicability of RNAi therapeutics indicates that the potential market for such drugs is huge.

As a result, effective RNAi platforms like Alnylam’s have attracted interest from licensing partners. In 2007 for example, Switzerland's Roche paid $1bn for rights to the company’s technology while, earlier this year, Alnylam’s Regulus Therapeutics joint-venture began working with GlaxoSmithKline (GSK) to develop drugs for inflammatory disorders.

Novartis Alnylam deal extension

Under the terms of the deal extension, Novartis will continue to use Alnylam’s technology in its drug development programmes in return for license fees, milestone payments and royalties if any of the medications covered by the accord are successfully commercialized.

The non-exclusive nature of the Novartis deal allows Alnylam to develop its own pipeline of RNAi therapeutics, the most advanced of which is a candidate respiratory syncytial virus (RSV) treatment.

While discussing the deal extension, Jeffrey Lockwood from the Novartis Institutes for BioMedical Research media relations department told in-PharmaTechnologist.com that the original 2005 development agreement had been remarkably productive.

He explained that: “Novartis elected to exercise its right to extend by one year last week. If the collaboration is successful and multiple products are developed and commercialized, collective payments to Alnylam could exceed $700 million, not including royalties​.”

Lockwood commented that “The collaboration has provided both partners with critical information on the potential of therapeutic RNAi. We are currently focused on formulation and delivery, which obviously requires differing solutions depending upon target and disease location. We have several projects running in the collaboration and are pleased with their progress.”

As with any new technology there are several issues that need to be resolved. In our collaboration we are currently focused on formulation and delivery, which obviously requires differing solutions depending upon target and disease location​,” he added.

Lockwood concluded that: “RNAi holds great promise as a new therapeutic modality for treating many diseases. In particular, this new area of biology has potential to treat diseases that have not been able to be addressed by traditional approaches. This collaboration complements Novartis’ existing therapeutic programs that utilize small molecule, therapeutic proteins and antibody technologies.”

John Maraganore, Alnylam’s CEO, said that described the Swiss major role in and enthusiasm for the development of RNAi therapeutics as being nothing short of pioneering.

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