Saying it is on track to exceed its goal of having 5 key products in clinical development through 2015, Alnylam recently announced that it intends to focus on three strategic therapeutic areas, or what it dubs "STArs," as it races to commercialize its first candidate delivered using the RNAi pathway and reassure sometimes antsy investors.
RNAi specialist Alnylam is focusing on rare diseases at the moment (with the exception of hepatitis B), but Chief Business Officer Laurence Reid told investors at the Leerink Rare Disease Roundtable conference that the company is also looking to enter bigger markets via partnerships.
Alnylam announced July 22 that its intellectual property estate has been strengthened due to the U.S. Patent Office's issuance of a Notice of Allowance for claims in one of its patent applications that covers "chemically modified RNA therapeutics conjugated with an N-acetylgalactosamine (GalNAc) ligand independent of length, sequence, or disease target," according to a statement.
Here at FierceDrugDelivery, we write often about innovations in nanotechnology as it becomes more and more important in the delivery field. These advances are mostly in the very early stages, but they're still promising as the delivery field moves even further into the nanoscale.
Sanofi is doubling down on its RNAi development deal with Alnylam. The pharma giant says it will pay a hefty premium to buy up a 12% stake in the biotech, investing $700 million and greatly expanding its rights to Alnylam's lead drug along with a portfolio of current and prospective therapies.
Gene-silencing expert Alnylam is $7 million richer thanks to some positive mid-stage results for its Genzyme-licensed RNA-interference candidate, and the Cambridge, MA, company is looking to be the first to market with the difficult-to-deliver class of treatments.
Alnylam, a specialist in RNAi drug delivery, welcomed positive results in an early-stage trial of its treatment designed to silence the gene that causes a rare, deadly form of the disease amyloidosis. The data establish human translation for Alnylam's conjugate delivery platform, the company says.
Cambridge, MA-based Alnylam Pharmaceuticals says it garnered positive interim data from a Phase I study of ALN-TTRsc, an RNAi therapy targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.
Regulus Therapeutics, a biotech using microRNA as a target for drug delivery, raised nearly $49 million through a public offering completed Tuesday, up from an anticipated $43 million.
The top-line results from a Phase I study show that its therapy ALN-TTRsc reduced levels of Transthyretin (TTR)-mediated amyloidosis protein by more than 80%, setting the stage for a mid-stage study planned for late this year in patients with a genetic heart disease called familial amyloidotic cardiomyopathy (FAC).