Many companies are realizing that the development of new delivery vehicles for therapeutics based on RNA interference (RNAi) will require collaborative efforts. “We view the delivery of small RNAs as one of the most important biomedical endeavours in modern biological science,” says John Maraganore, chief executive of Alnylam Pharmaceuticals in Cambridge, Massachusetts. “And when you have such a broad-scale challenge and opportunity, you can't do everything internally — you have to work with the best groups outside as well.”

This spring, Alnylam initiated a collaboration with Daniel Anderson and Robert Langer at the Massachusetts Institute of Technology (MIT) in Cambridge. Under the agreement, Alnylam will provide funding for ten postdocs at MIT to work on issues directly related to small-RNA delivery while having the exclusive option to any intellectual property that comes out of the effort.

Anderson says that at its core this is an academic programme, so the goal is to get postdocs to work on the delivery issue while being trained as future leaders in the field.

“One nice feature is that we have collaborations with a company that is a leader in this field, so it allows us to accelerate our efforts tremendously,” Anderson says. For example, when the MIT researchers develop any new delivery technologies, scientists at Alnylam can provide animal models and other methods to test and evaluate these new vehicles, providing quick feedback on whether or not the research is on the right track.

An electron microscope image of nanoparticles that can be used as delivery vehicles for siRNA. Credit: NANO LETT. 7, 874–879 (2007).

Langer's lab has a long history of translational research. It has developed principles that have led to some 40 products that are now in clinical trials or have been approved by the US Food and Drug Administration. “The hope is that we can do that here,” says Langer. “We can do the kind of basic research to help solve the RNAi delivery problem and then work closely with Alnylam to take the basic research into the clinic where it can be used to treat different diseases.”

In addition to the MIT collaboration, Alnylam also funds R&D and manufacturing activities in Vancouver, Canada, to further the development of cationic lipids for delivery, and has some 25 feasibility agreements in place in which Alnylam is testing and evaluating technology that has been introduced from either companies or academic groups.

N.B.