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- PMLiVE

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- PMLiVE

Novartis and Voyager enter gene therapy partnership worth over $1.2bn

The companies will advance candidates for Huntington’s disease and spinal muscular atrophy

- PMLiVE

Novartis to temporarily suspend Huntington’s disease phase 2b study after side effects reported

A data review found that branaplam may cause peripheral neuropathy, which is the result of nerve injury outside the brain and spinal cord

- PMLiVE

FDA removes clinical hold on uniQure’s haemophilia gene therapy

Clinical hold placed following a case of hepatocellular carcinoma in a patient in the trial

- PMLiVE

New results back Sangamo, UniQure haemophilia gene therapies

Encouraging results for firms in four-way race

- PMLiVE

UniQure gets speedy US review for Huntington’s gene therapy

Pioneer gaining momentum again

- PMLiVE

UniQure edges closer to haemophilia B gene therapy OK

The candidate could free patients from regulator injections of FIX products

- 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

UniQure’s starts pivotal haemophilia B gene therapy trial

The treatment could relieve haemophilia B patients from factor IX replacement drugs

- PMLiVE

EMA hands UniQure orphan status first in Huntington’s

The Dutch biotech expects to file the treatment for approval later this year

- PMLiVE

FDA grants orphan drug status to UniQure’s Huntington’s Disease treatment

Becomes first investigational gene therapy to receive ODD for the disease

- PMLiVE

UniQure pulls first EU gene therapy

Decision made due to lack of demand

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