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UniQure

- PMLiVE

Novartis builds case for SMA gene therapy as FDA verdict looms

One shot therapy provided rapid improvement in motor function

- PMLiVE

Sangamo surges on data for haemophilia A gene therapy

After recent setbacks, news lifts shares

- PMLiVE

UniQure edges closer to haemophilia B gene therapy OK

The candidate could free patients from regulator injections of FIX products

- PMLiVE

J&J bolsters gene therapy unit with $100m MeiraGTx deal

Licenses two inherited retinal disorder candidates

- 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

Orchard plans IPO as gene therapy pipeline advances

UK biotech seeks funds for expanded rare disease portfolio

- PMLiVE

EMA to review Bluebird’s blood disorder gene therapy

LentiGlobin could free patients of regular blood transfusions

- PMLiVE

Sangamo bulks up in cell therapy with TxCell acquisition

Hopes to start cell-based immunotherapy trials next year

- PMLiVE

Pfizer enters late-stage haemophilia B gene therapy trial

Race is on to free patients of regular injections

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