Alnylam ($ALNY) began Phase I clinical trials for its RNAi-based treatment of TTR-mediated amyloidosis, or ATTR, a particularly menacing protein disorder that affects the nerves and heart.
The first-of-its-kind treatment, ALN-TTRsc, is designed to deliver the gene-inhibiting RNAi to the liver via an injection under the skin, shutting down the part of the patient's DNA that causes amyloidosis-driven cardiomyopathy, a weakening of the heart. The delivery platform makes use of a specific ligand, GalNAc, that enables the targeted gene silencing.
Alnylam, which partnered with Sanofi's ($SNY) Genzyme last year to develop these and other RNAi-based treatments, dosed the first patients in the U.K. with plans to enroll up to 40 healthy patients in the study. In prior animal studies, the treatment showed an 80% reduction in the disorder with no significant adverse reactions, according to Alnylam.
"RNAi therapeutics hold great promise for the treatment of ATTR since they have demonstrated rapid, potent and durable knockdown of TTR, the disease-causing protein," Alnylam VP and chief medical officer Akshay Vaishnaw said in a statement.
Alnylam is at the forefront of RNAi technology, having completed another Phase I trial for a liver cancer treatment this year. Despite some promising results at Alnylam, though, other companies have encountered difficulties delivering the gene silencer, including Novartis ($NVS), Merck ($MRK) and Roche ($RHHBY), all of which have cut back on their RNAi lines in the last few years.
- here's the release