After earlier this month announcing promising new data supporting its genetic treatment for hepatitis B, Arrowhead Research is moving forward with the candidate, submitting its application to begin a Phase IIa trial of ARC-520, which uses its polymer-based method to effectively deliver RNAi to the nucleus.
Using RNA interference technology, scientists have discovered a network of genes that could serve as new therapeutic targets for Parkinson's disease as well as other diseases characterized by mitochondrial damage.
San Diego RNAi specialist Arcturus Therapeutics pulled in $5 million this week to support its RNAi tech, which it acquired earlier this year from Marina Biotech, and its own nanoparticle delivery platform. The company expects to unveil its novel gene-silencing drugs with a candidate early next year.
This week, Arrowhead Research announced the completion of its enrollment for a Phase I RNAi-based hepatitis B trial as well as a planned $60 million private offering.
Alnylam is pushing its latest RNAi drug through early clinical trials, highlighting the company's delivery platform that enables an effective gene-silencing treatment. This time, the RNA specialist published results from a successful Phase I study of its cholesterol drug ALN-PCS.
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.
San Diego police say that biotech entrepreneur Hans Petersen shot and wounded his brother-in-law Ron Fletcher and the University of California, San Diego scientist Steven Dowdy at separate times and locations Thursday, The Associated Press reported.
Alnylam won two orphan drug designations this week and last, both for the same drug--one designation is for hemophilia A and the other for hemophilia B. Alnylam's RNA interference drugs use small pieces of the genetic code to silence, or turn off, genes that cause diseases.
Alnylam chugged along this week in its efforts to advance its line of RNAi drugs to the market, notching positive results for two of its candidates in Phase II and preclinical trials.