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Evaluate

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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

Latest Reports

July 27, 2023

H1 Round Up: The haves and the have-nots

July 21, 2023

ADA/EASL eBook

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Editor's Picks

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July 03, 2023

Astrazeneca and Daiichi’s big reveal disappoints

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July 13, 2023

Pharma finds a way to gain weight

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July 03, 2023

Protagonist investors flake as psoriasis pill disappoints

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June 29, 2023

Approaching datasets for big pharma

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June 27, 2023

Nkarta goes back to basics

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