Keyword: Ionis Pharmaceuticals
ProQR is licensing an RNA medicine from Ionis Pharmaceuticals for the treatment of retinitis pigmentosa, a form of rare, inherited blindness.
Stoke has completed a meaty $90 million second funding round as the startup eyes getting its leading candidate into the clinic by 2020.
An antisense technology for silencing genes that contribute to hampered insulin production may help treat Type 2 diabetes.
Intellia Therapeutics is developing a CRISPR-based treatment to edit the mutation that causes transthyretin amyloidosis.
Ionis signed on with Roche to develop its RNA drug for advanced dry AMD, in a deal worth up to $760 million, after being snubbed by GSK last year.
Akcea has its first FDA approval for hATTR therapy Tegsedi but will be burdened with a black box warning as it chases rival Alnylam.
The interim readout showed that two-fifths of infants who received PTC and Roche’s oral SMA drug were sitting eight months after starting treatment.
Akcea reported topline data showing its hyperlipoproteinemia drug lowered lipoprotein(a) levels in a phase 2 study in patients with cardiovascular disease.
The cuts come 10 days after the FDA rejected a filing for approval of ultrarare lipid disorder drug volanesorsen.
Swedish researchers have found that the rogue SOD1 protein can “transmit” protein misfolding in ALS.