AAN 2019 – Roche confirms its place in spinal muscular atrophy
A slew of data on spinal muscular atrophy projects over the weekend shows that this tiny market is set for some big competition, with Novartis, Roche and Biogen jostling…
Alnylam swaps Regeneron for Sanofi
The RNAi specialist gets an $800m cash injection under a central nervous system tie-up with Regeneron.
Readouts approach for biggest Huntington’s hopes
Upcoming data from Roche/Ionis and Wave Life Sciences will be an important test of potentially disease-modifying candidates.
Alnylam touts givosiran win despite toxicity fears
Alnylam looks likely to get approval for its second RNAi candidate, givosiran. But toxicity issues have tainted the Envision trial win.
Upcoming events – Fibrogen and Alnylam look to broaden their horizons
Duchenne muscular dystrophy attracts a new entrant in pamrevlumab, while Alnylam awaits more pivotal data with givosiran.
Ionis's third shot at the Nash target goes astray
After two other liver disease projects fade into the background Ionis delays the antisense asset AKCEA-ANGPTL3-LRx by a year.
Another knock back for urea cycle disorders
Curevac’s decision to exit work on an mRNA approach to OTC deficiency follows a clinical hold on Translate Bio’s similar project, and raises broader concerns.
Higher dose points Sangamo no closer to zinc finger success
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
JP Morgan 2019 – Alnylam turns Onpattro frown upside-down
Alnylam appears to have turned its Onpattro launch around, but is adamant that investors should have been more patient all along.
Kickstarting ideas
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