Alnylam signs second research agreement

CAMBRIDGE, Mass.—At the same time it announced the signing of a research collaboration with Vancouver-based Inex Pharmaceuticals (see E050617), RNAi specialist Alnylam announced a similar liposome research agreement with Dr. Robert Langer and colleagues at the Massachusetts Institute of Technology.

o a large extent, the deal stems from a number of scientific connections that already existed between the two groups, such as Langer's position on Alnylam's Scientific Advisory Board and one of Langer's graduates who is a leader of drug delivery research at Alnylam.

According to MIT research associate Dr. Dan Anderson, MIT has granted Alnylam an option to take licenses to novel liposomal drug delivery systems that Langer's group has developed. "Preclinical testing indicates that these novel lipid delivery systems offer considerable promise for systemic delivery of RNAi therapeutics," Anderson says. "We are continuing to work with Alnylam towards the eventual goal of pushing these into the clinic."

For Alnylam, the licensing effort is an important next step in its efforts to develop effective methods for the delivery of RNAi-based therapeutics. "The Langer lab at MIT is a world-leading biomedical research group with expertise in the engineering of delivery technologies and a track record of many approved products," says Barry Greene, Alnylam COO. "This technology enables the development of additional systemic delivery approaches based on liposomes."

The deal also follows closely on positive liposome-based delivery results reported with collaborators at Protiva Biotherapeutics. "The data published in Nature represent a major advancement in the development of systemic RNAi therapeutics," Greene says.

"Working with primates—an animal model that is very close to humans—we were able to demonstrate rapid, dramatic and sustained therapeutic effects following systemic administration of an siRNA. Systemic delivery of an siRNA through the bloodstream clearly opens up the possibility of targeting disease-causing genes in a wide variety of tissues, significantly expanding the already broad potential of RNAi therapeutics to treat human disease."

Aside from direct licensing, the MIT group will continue to work with Alnylam to improve the liposomal delivery vehicles, which Anderson suggests "work very well and are better than other [delivery] systems we have tried."

Meanwhile, Alnylam is keeping its options open. "There are a number of therapeutic areas that we believe would benefit from a liposomal systemic RNAi therapeutic approach, such as cancer, metabolic and cardiovascular disease, and viral infections," Greene says. "However, it is premature to determine the disease area that we will pursue initially with a systemic, and potentially liposomal, approach."