An analyst wrote that while the data are positive, the bigger question is how the drug will be priced, based on another drug already on the market for the same disease, HoFH, which affects about 1,300 people in the U.S.
The oversubscribed round comes just four months after acquisition of GSK's gene therapy portfolio.
This eBook provides actionable strategies, real-world best practices to help payers stay ahead in a value-based care environment.
Promising Phase 3 study results for its RNAi treatment, called Patisiran, for a rare, genetic nerve disorder have increased the chances that Alnylam could be the first to commercialize a RNAi drug.
No mutation left behind – that’s the concept of Perlstein Lab, a Bay Area biotech and public benefit corporation that explores precision orphan drug delivery. Its thesis is that though 1 in 12 people have some variety of orphan disease, the vast majority – some 95 percent – have no treatment. Because each orphan condition is caused by a “spectrum […]