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- PMLiVE

Hemophilia

- PMLiVE

UniQure gets speedy US review for Huntington’s gene therapy

Pioneer gaining momentum again

- PMLiVE

Sanofi exits R&D pact with Alnylam on rare disease

Exit also frees up $700m equity investment

- PMLiVE

BioMarin prepares for haemophilia A gene therapy data readout, filing

Key long-term data expected in July

- PMLiVE

UniQure edges closer to haemophilia B gene therapy OK

The candidate could free patients from regulator injections of FIX products

- PMLiVE

Trial dents confidence in Sangamo’s gene-editing tech

Company proposing switch to next-gen version

- PMLiVE

UniQure cleared to start Huntington gene therapy trial

AMT-130 becomes the first AAV-based therapy approved for trials in Huntington’s disease

- PMLiVE

UniQure skyrockets on haemophilia B gene therapy data

Competition with Pfizer and Spark rival heats up

- PMLiVE

Roche gets wider approval for haemophilia drug Hemlibra

Approved to treat bleeding episodes in patients without inhibitors

- PMLiVE

Sangamo’s first gene-editing trial doesn’t convince investors

Market spooked by inconclusive early results

- PMLiVE

Roche highlights Hemlibra data as key FDA date approaches

Expanded use would see revenues take off

- PMLiVE

Sangamo bulks up in cell therapy with TxCell acquisition

Hopes to start cell-based immunotherapy trials next year

- PMLiVE

NHS England says yes to Roche’s Hemlibra

But stalls on funding for Actelion's Uptravi

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