Keyword: Ionis Pharmaceuticals
The week’s biotech news included a mutation-targeting treatment for ALS and a malaria vaccine targeting a transmission protein.
Orion Pharma is pressing ahead with a pivotal trial for its amyotrophic lateral sclerosis drug ODM-109, despite mixed results in a phase 2 trial.
The French biotech picked up the antisense oligonucleotide centronuclear myopathy prospect from its cofounder, Ionis Pharmaceuticals.
Eidos' $106 million IPO comes on the heels of a $64 million series B and will support phase 2 and phase 3 trials of its TTR amyloidosis drug.
Scientists have developed a small molecule that can selectively target RNA and could pave the way for a pill that can treat genetic diseases.
After a turbulent year, Prothena is reorganizing to rally its resources around what remains in its neuroscience pipeline.
An FDA panel voted 12-8 to recommend the approval of Akcea's volanesorsen, an RNAi drug for a rare lipid disorder.
Alnylam is gearing up to move RNAi therapies for central nervous system disorders into the clinic.
Akcea suffered the setback after the FDA asked for more time to review its response to requests for information on the rare disease antisense drug.
This comes after two other biotechs had to pull their attempts last week.