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).
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.
Alnylam Pharmaceuticals heralded positive preclinical data for its RNAi drug that targets a specific protein to treat a range of life-threatening diseases.
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.
Alnylam Pharmaceuticals has reported upbeat preclinical findings in a proof-of-concept study for its RNAi therapeutic program to treat porphyria, including acute intermittent porphyria, a rare genetic metabolic disorder caused by an enzyme deficiency.
Alnylam began Phase I clinical trials for its RNAi-based treatment of TTR-mediated amyloidosis, or ATTR, a particularly menacing protein disorder that affects the nerves and the heart.