Alnylam's makes progress with RNAi program

RNAi trailblazer Alnylam has published a paper in the journal Human Molecular Genetics that details RNAi's ability to silence a gene implicated in early onset torsion dystonia. Dystonia is a neurological movement disorder in which sustained muscle contractions cause twisting and repetitive movements or abnormal posture. According to Dinah Sah, Ph.D., Senior Director of Research at Alnylam, the data demonstrates RNAi therapeutics can discriminate between a disease target of interest and a closely related gene, which could help treat genetic disorders.

"We are encouraged by these results, as well as results we have seen to date with our other neurological RNAi therapeutic programs, such as Huntington's disease, Parkinson's disease, and others," added Sah.

- read the release

Suggested Articles

At ASH, Dana-Farber researchers presented promising preclinical data on AMG 701, Amgen's long-lasting BiTE for multiple myeloma, but rivals loom.

The takeover, the first of Paul Hudson’s time as CEO, will give Sanofi an IL-2 drug it thinks can become a foundation of future cancer combinations.

A phase 1/2b test showed Roche's antibody mosunetuzumab can trigger durable responses in a significant minority of hard-to-treat NHL patients.