Skip to main content
Premium Trial:

Request an Annual Quote

Isis TTR Amyloidosis Drug Fast Tracked by FDA

Premium

Isis Pharmaceuticals this week announced that the US Food and Drug Administration has fast tracked its antisense-based transthyretin amyloidosis drug ISIS-TTRRx for use in patients with familial amyloid polyneuropathy, the rarer form of the disease.

The drug is being developed by Isis in collaboration with GlaxoSmithKline. A phase II/III study is expected to start this month, triggering a $7.5 million milestone payment from GlaxoSmithKline, Isis said.

Alnylam Pharmaceuticals is developing an RNAi-based treatment for ATTR, ALN-TTR02, which is in phase II testing and was recently partnered with Sanofi subsidiary Genzyme in Asia (GSN 10/25/2012).

The Scan

Interfering With Invasive Mussels

The Chicago Tribune reports that researchers are studying whether RNA interference- or CRISPR-based approaches can combat invasive freshwater mussels.

Participation Analysis

A new study finds that women tend to participate less at scientific meetings but that some changes can lead to increased involvement, the Guardian reports.

Right Whales' Decline

A research study plans to use genetic analysis to gain insight into population decline among North American right whales, according to CBC.

Science Papers Tie Rare Mutations to Short Stature, Immunodeficiency; Present Single-Cell Transcriptomics Map

In Science this week: pair of mutations in one gene uncovered in brothers with short stature and immunodeficiency, and more.