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Friday, September 28, 2018

Another Second-Generation GalNAc-RNAi Rocked by Off-Target Concerns

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With the approval of ONPATTRO and the current overall clinical picture, in particular the highly encouraging safety profile seen with PCS...
Monday, September 10, 2018

Second-Tier RNAi Therapeutics Companies Starting to Deliver Clinically

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Alnylam has had a 3 year head-start on the competition with GalNAc RNAi Therapeutics, but now additional companies are populating the fie...
Monday, August 13, 2018

RNAi Therapeutics Now a Commercial Reality

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Last Friday, Alnylam received notification from the US Food and Drug Administration (FDA) that ONPATTRO (aka Patisiran) has been approve...
Thursday, August 9, 2018

TEGSEDI worse than tetramer stabilizer according to newly released EMA document

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When Ionis presented data from the phase III NEURO-TTR study last year in Paris, they clung to numbers close to ‘zero’ to make the point...
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Friday, August 3, 2018

Taking Stock of RNAi Therapeutics

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Though currently vacationing, this summer is starting to get quite busy for the RNAi Therapeutics space, so I thought a quick update summ...
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Monday, July 9, 2018

Regulus Therapeutics Declares MicroRNA Therapeutics Bankrupt

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Once considered a new star on the biotech firmament, the most prominent microRNA Therapeutics company is going from bad to worse fast.   ...
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Monday, July 2, 2018

Arrowhead Achieves Robust Alpha-1 Antitrypsin Lowering As Grounds for Trial Termination Still Shrouded in Mystery

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Last Friday, Arrowhead Pharmaceuticals presented first data from its phase I study of ARO-AAT being developed for addressing liver diseas...
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