Alnylam hooks up with MIT for RNAi therapeutics delivery

By Pete Mansell

- Last updated on GMT

US company Alnylam Pharmaceuticals has consolidated its
relationship with researchers from the Massachusetts Institute of
Technology (MIT) by signing an agreement to sponsor a five-year
program at the Institute's Center for Cancer Research, focused on
the delivery of therapeutics based on RNAi interference.

Alnylam, which has a number of RNAi therapeutics in development, including a lead programme, ALN-RSV01, in Phase I clinical trials for the treatment of respiratory syncytial virus infection, will provide research funding for around ten post-doctoral researchers annually over the five-year term, in a programme led by MIT's Robert Langer and Daniel Anderson.

No financial details were disclosed.

In return, Alnylam will have an exclusive option to license for worldwide commercialisation any RNAi technology that results from the collaboration.

This builds on a pre-existing licensing agreement with MIT, under which Alnylam has exclusive development and commercialisation rights to a lipidoid-based delivery technology for RNAi therapeutics originating from the Langer laboratory.

The US company said the new agreement was "very broad" and not tied to one particular form of delivery.

"The collaboration will investigate and evaluate all types of delivery - both systemic and direct, lipidoid and other," it commented.

Clinical trials with RNAi therapeutics to date have tended to involve routes of administration where it is relatively straightforward to bring the drug into contact with its target tissue.

Phase I trials with Alnylam's lead compound ALN-RSV01, for example, have involved both intranasal administration and delivery of a nebulised formulation directly to the lungs through inhalation.

Data from the more recent inhalation study are expected in the second half of 2007.

In January, Alnylam secured a worldwide exclusive license to liposomal delivery formulation technology from Canada's Inex Pharmaceuticals Corporation for the discovery, development and commercialization of RNAi therapeutics.

The companies also expanded an existing research and manufacturing alliance for lipid-based delivery technology.

This relationship now continues with Tekmira Pharmaceuticals Corporation, which completed its spin-off from Inex on May 1.

According to Alnylam, the new agreement with MIT is "another example of our strategy to be open and explore opportunities for advancing and improving delivery of RNAi therapeutics" .

To this end, the company is building a "delivery toolkit" of solutions that include cholesterol conjugation, peptides, antibodies and other approaches.

"We do not believe that there will be a 'one size fits all' approach but rather, depending on the disease area, tissue type and cell type, different approaches will be needed for optimal delivery," Alnylam commented.

Delivery has been "the most important challenge in the development of RNAi therapeutics" , the company pointed out.

"Although there are a large number of therapeutic areas that can be addressed with direct delivery of an RNAi therapeutic, it is believed that systemic delivery of these agents opens up the broadest potential applications for RNAi therapeutics in the treatment of human disease."

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