Thermo Fisher, Genentech Collaborate on RNAi Tech Development
Thermo Fisher Scientific said this week that it will collaborate with Genentech to develop novel siRNA technologies for use in RNAi therapeutics.
Thermo Fisher Scientific said it will provide Genentech with siRNA design, stabilization, delivery, and related RNAi technologies from its RNAi Discovery and Therapeutic Services laboratory in Lafayette, Colo. Genentech will then use the technologies to develop siRNA drugs for undisclosed indications.
Additional terms of the arrangement were not provided.
The move sets up Genentech to become a player in the RNAi therapeutics field, joining a number of other biopharmaceutical firms including Roche, which earlier this year made an offer to acquire the roughly 44 percent stake in Genentech it does not already own.
Alnylam Adds Orphan Disease TTR Amyloidosis to Development Pipeline
Alnylam Pharmaceuticals this week announced that it has moved a new preclinical program into its formal development pipeline.
The program, called ALN-TTR, is focused on TTR amyloidosis, a hereditary, systemic disease caused by a mutation in the transthyretin gene. The company said TTR is a carrier for thyroid hormone and retinol binding protein and is produced almost exclusively in the liver.
The disorder is characterized by the buildup of TTR-containing amyloid fibrils in extrahepatic tissues including peripheral nerves and the heart, Alnylam said. It is an orphan disease, affecting an estimated 10,000 people worldwide.
Alnylam, University of Bonn Team Publish On Immunostimulatory siRNAs
Alnylam Pharmaceuticals this week announced the publication of data on so-called 3p-siRNAs, a class of siRNA designed to silence a target gene while triggering a desired immunostimulatory response.
According to the company, the p3-siRNAs are chemically modified to activate retinoic acid-induced gene I, or RIG-I, which is a cytoplasmic immunoreceptor which strongly induces type I interferon and natural killer cell responses.
In a paper appearing this week in the online version of Nature Medicine, Alnylam researchers and collaborators from the lab of Gunther Hartmann at the University of Bonn reported that a 3p-siRNA specific for the anti-apoptotic factor bcl-2 may be a highly effective anti-cancer agent in several animal models, the company said.
Specifically, 3p-siRNAs were designed through a combination of “sequence-based complementarity to a target gene and chemical modification of the 5' ends of the sense and antisense strands of the same siRNA with a triphosphate moiety,” Alnylam said. “With a bcl-2-specific sequence, 3p-siRNAs led to a robust in vivo apoptotic effect, provoking massive programmed cell death of tumor cells” and resulting in a greater than 80 percent reduction in total lung metastases in mouse models of melanoma and adenocarcinoma.
The benefits of the dual-target approach were also confirmed by the observation of more modest anti-tumor effects with either bcl-2 targeting with standard siRNAs or RIG-I activation with non-specific 3p-siRNAs.
"In this new research, 3p-siRNAs were designed to intentionally promote a potent immune stimulatory mechanism in concert with gene silencing,” Rachel Meyers, senior director of RNAi lead development at Alnylam and a study co-author, said in a statement. “We believe this approach could represent a potentially new and effective strategy for cancer therapy, as it has been shown that many cancers are better treated through a combination therapy approach."
Devgen to Shutter Pharma Unit, Focus on Agro Business
Devgen said this week that it plans to close its pharma business, which is developing small-molecule drugs for inflammatory diseases, after efforts to sell the unit failed.
According to the company, it will cut 19 employees in connection with the move, and now focus on its agro business, which includes using RNAi for crop protection, trait development, and molecular breeding.
“In the current market, the chance to valorize Devgen pharma assets without further major investments of the company is remote," Devgen CEO Thierry Bogaert said in a statement. "Devgen will focus its resources and will further develop in the agro-market segment where the company has the strongest technology and business opportunities: the trait and seed opportunities in rice, an important growth market in which the company is well positioned to develop its technology and business faster than the market and to build a leadership position."