Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Touts Advances with Lipid, Conjugate Delivery Technologies


Alnylam Pharmaceuticals said this week that it and collaborators have developed a novel class of lipid nanoparticles capable of greater target inhibition than the company's existing lipid delivery agents.

According to the company, the so-called reLNPs demonstrate “potent target gene silencing in vivo, with an ED50 at doses of less than 0.05 mg/kg while demonstrating tolerability at doses exceeding 10 mg/kg.”

This represents a roughly 10-fold improvement over the company's second-generation MC3 lipids, and an approximately 100-fold increase in efficacy over first-generation technologies.

The MC3 lipid is currently at issue in a lawsuit between Alnylam and partner Tekmira Pharmaceuticals (GSN 8/18/2011).

Alnylam also said that it has made improvements to its early-stage GalNAc conjugation technology for siRNA delivery

“Improvements in chemistry lead to markedly increased potency for in vivo target gene silencing with GalNAc-conjugated siRNAs administered by subcutaneous injection at low, clinically relevant doses,” Alnylam said.

Meanwhile, administration of GalNAc-conjugated siRNAs against transthyretin, the target of the firm's phase I transthyretin-mediated amyloidosis therapy ALN-TTR01, resulted in robust gene silencing in preclinical animal models with an ED50 of approximately 5 mg/kg after a single subcutaneous injection.

Earlier this year, Alnylam CEO John Maraganore called conjugate-based delivery approaches “the future for delivery of small interfering RNAs” (GSN 6/2/2011).

The Scan

Study Reveals New Details About Genetics of Major Cause of Female Infertility

Researchers in Nature Medicine conducted a whole-exome sequencing study of mote than a thousand patients with premature ovarian insufficiency.

Circulating Tumor DNA Shows Potential as Biomarker in Rare Childhood Cancer

A study in the Journal of Clinical Oncology has found that circulating tumor DNA levels in rhabdomyosarcoma may serve as a biomarker for prognosis.

Study Recommends Cancer Screening for Dogs Beginning Age Seven, Depending on Breed

PetDx researchers report in PLOS One that annual cancer screening for dogs should begin by age seven.

White-Tailed Deer Harbor SARS-CoV-2 Variants No Longer Infecting Humans, Study Finds

A new study in PNAS has found that white-tailed deer could act as a reservoir of SARS-CoV-2 variants no longer found among humans.