Alnylam Pharmaceuticals, a leader in the research and development of RNAi drug delivery, arrived at a successful proof-of-concept result for its gene-silencing treatment to temper a rare and deadly autosomal disease.
Small interfering RNA (siRNA), a type of RNAi molecule, has the potential to be a powerful therapeutic agent. And while delivering the bits of genetic material to cells has proven challenging, a new nanotechnology might be on the right track.
Researchers at Barcelona's Vall d'Hebron Institute of Oncology in Spain and U.S. biotech Alnylam Pharmaceuticals have shown that, with a novel delivery technique, they can actively "silence" cancer genes with molecules using the selective interference technique, RNAi.
Israeli company Quiet Therapeutics has captured funding of $5.5 million to develop its nanoparticle-based delivery system for RNAi and small-molecule drugs for cancer and inflammation.
Alnylam Pharmaceuticals is preparing to move ALN-TTRsc, its lead product using the GalNAc-siRNA delivery system, into a Phase I study in the U.K.
ALN-AT3, Alnylam Pharmaceuticals' RNAi therapeutic in development for the treatment of hemophilia, has shown positive results in a preclinical study in nonhuman primates by increasing thrombin generation as much as fourfold.
The legal squabble between Tekmira Pharmaceuticals and Alnylam ($ALNY) over rights to the RNAi delivery technology Alnylam uses has been called off. Alnylam is handing over $65 million in cash along with a potential $10 million near-term payout to resolve the showdown over the IP related to lipid nanoparticle technology.
Alnylam revealed that the rare-disease specialists at Genzyme--the biologics arm of pharma giant Sanofi--had stepped in to license Asian rights to its program for ATTR, putting down $22.5 million as an upfront along with an unspecified lineup of milestones.
Tel Aviv University in Israel says it has worked with Alnylam Pharmaceuticals ($ALNY) and DNA Technologies to concoct a new class of RNAi drugs with a shot at combating mantle cell lymphoma, an incurable blood cancer.
RXi Pharmaceuticals will use a new government grant to pursue preclinical work on an RNAi-related treatment for a nasty form of eye cancer. The news underscores the fact that research in the field is advancing, even as progress toward development of new RNAi treatments continues to be frustrating and sluggish.