Alnylam Pharmaceuticals this week announced that it has received an orphan drug designation from the US Food and Drug Administration for its preclinical siRNA-based drug ALN-AT3 as a treatment for hemophilia A.

Last week, the company said that the drug received an orphan drug designation from the FDA for hemophilia B.

The designation provides a drugmaker with, among other things, extended market exclusivity for its product, tax credits, and marketing incentives.

ALN-AT3 is expected to enter phase I testing next year.

Get the full story with
GenomeWeb Premium

Only $95 for the
first 90 days*

A trial upgrade to GenomeWeb Premium gives you full site access, interest-based email alerts, access to archives, and more. Never miss another important industry story.

Try GenomeWeb Premium now.

Already a GenomeWeb Premium member? Login Now.
Or, See if your institution qualifies for premium access.

*Before your trial expires, we’ll put together a custom quote with your long-term premium options.

Not ready for premium?

Register for Free Content
You can still register for access to our free content.

The Jackson Laboratory has filed a complaint accusing Nanjing University of breeding and re-selling its mouse models, the Hartford Courant reports.

Oxford researchers are turning to virtual reality to visualize genes and regulatory elements, Phys.org says.

In Science this week: neutrophils rely on microRNA to protect against lung inflammation, and more.

China is moving forward with plans to sequence a million citizens, the Wall Street Journal reports.