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September 06, 2021

Another gene therapy safety scare hits

The clinical hold for Biomarin’s phenylketonuria project comes on the heels of an FDA adcom to discuss gene therapy toxicity.

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July 13, 2021

Novo bets against Crispr for amyloidosis

Novo moves into amyloidosis not long after Intellia made a splash here.

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March 23, 2021

The Huntington’s pipeline takes a blow

The failure of Roche and Ionis’s late-stage candidate tominersen casts doubt on Wave’s approach, too.

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April 04, 2018

Therapy focus – Pfizer blows hereditary amyloidosis fight open

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August 03, 2016

Therapy focus – One step forward but two steps back in Huntington’s

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June 23, 2016

Therapy focus – Amyloidosis pipeline still looks to Alnylam for answers

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