Alnylam records RNAi delivery success to treat rare disease

Alnylam Pharmaceuticals ($ALNY), a leader in the research and development of RNAi drug delivery methods, arrived at a successful proof-of-concept result for its gene-silencing treatment to temper a rare and deadly autosomal disease.

Alnylam's new drug targets aminolevulinate synthase-1 (ALAS-1) to treat the genetic metabolic disorder porphyria, including acute intermittent porphyria (AIP). Porphyria is an extremely rare disease caused by a mutation that leads to an enzyme deficiency. Collaborating with researchers from the Icahn School of Medicine at Mount Sinai in New York City, the Cambridge, MA-based company showed that the treatment can block the abnormal production of toxic intermediates that cause the painful and potentially fatal symptoms of AIP.

The researchers gave a single subcutaneous dose of ALAS-1-targeted RNAi to mice with the AIP mutation, and the drug proved effective at protecting the animals from symptomatic attacks for up to two weeks at a time. It's good news for the approximately 5,000 sufferers of AIP in the U.S. and Europe who experience recurrent debilitating attacks, according to the release.

The delivery of RNAi drugs, which use a portion of genetic code to "silence" disease-causing sequences in DNA, has been a difficult obstacle to overcome for many companies, and the success of the ALAS-1 treatment demonstrates Alnylam's leading the way in the arena. The drugs have the potential to be a powerful strategy to combat many genetic diseases, including cancer, but harnessing the power of their targeting mechanisms has been troublesome.

And the innovation continues at Alnylam: "We are now extending these preclinical results to a GaINAc-siRNA development candidate for our ALN-AS1 program in late 2013, leading to an investigational new drug filing in 2014," said Vice President of Clinical Research Jared Gollob in a statement. "Our preclinical data clearly show that RNAi therapeutics targeting ALAS-1 can achieve potent, rapid and durable suppression of the toxic heme biosynthesis intermediates that cause the symptoms and disease pathology of AIP."

- here's the release

Special Report: Harnessing RNAi as a gene silencer - The Future of Nanotech Drug Delivery