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- PMLiVE

UniQure

- PMLiVE

Pfizer enters late-stage haemophilia B gene therapy trial

Race is on to free patients of regular injections

AstraZeneca AZ

AZ makes gene therapy move with 4DMT alliance

The new partnership will focus on creating an AAV for lung disease

- PMLiVE

Axovant licenses another gene therapy as new strategy beds in

Group pursues gene therapy strategy

- PMLiVE

UniQure’s starts pivotal haemophilia B gene therapy trial

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

- PMLiVE

UK firm Nightstar bags FDA fast-track status for gene therapy

Its choroideremia disease candidate gets RMAT designation

- PMLiVE

AMICULUM boosts global healthcare communications offering with a new specialist agency, Seques®

AMICULUM is entering its 18th year of growth as an independent healthcare communications and consulting business. Today we are delighted to announce the launch of the first communications agency specializing...

AMICULUM

Biomarin

BioMarin ups the ante for pivotal haemophilia gene therapy trial

Adds another 90 patients to the GENEr8-1 registration study

- PMLiVE

Morning brief: Herzuma launched in Europe, rare disease status for Orchard candidate and more

A rapid round-up of pharma, biotech and healthcare news

- PMLiVE

Novartis buys gene therapy firm AveXis for $8.7bn

Lead candidate AVXS-101 could potentially replace regular drug treatment for SMA with a one-off intervention

- PMLiVE

Novartis negotiates ex-US rights to Spark gene therapy

Strikes $170m deal for Luxturna

- PMLiVE

EMA hands UniQure orphan status first in Huntington’s

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

- PMLiVE

Pfizer buys into Sangamo’s motor neuron disease gene therapy

The pharma giant will pay $12m upfront and $150m in potential milestones

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