Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

haemophilia B

- PMLiVE

Shire’s gene therapy candidate receives orphan drug status in the US

Investigational factor VIII treatment SHP654 is being developed for haemophilia

Will gene therapies finally be commercially viable to pharma?

Our latest Free Thinking white paper explores the science behind the idea of gene therapy and takes a look at some of the core challenges medical researchers have faced when...

Research Partnership

- PMLiVE

Spark closes on first US gene therapy approval

FDA starts review of Luxturna for patients with inherited retinal disease

- PMLiVE

Novo Nordisk’s long-acting haemophilia B drug cleared by FDA

US approval sets up marketing battle with Bioverativ and CSL Behring

- PMLiVE

Opening up innovation

Paul-Peter Tak on GlaxoSmithKline’s R&D strategy and the pivotal role of immunology

- PMLiVE

Pfizer buys gene therapy specialist Bamboo

Expands pipeline with Duchenne muscular dystrophy candidate in deal worth $700m

Harnessing complexity

Companion diagnostics in oncology

- PMLiVE

Mixed news for gene therapies at ASH conference

GSK unveils successful trial, but there was disappointing news for Bluebird

- PMLiVE

Haemophilia Society launches UK awareness drive

Charity tackles poor understanding of bleeding disorder

- PMLiVE

FDA approves drugs for rare genetic liver condition and to reverse anticoagulation

Raptor’s Procysbi and CSL Behring’s Kcentra recommended in US

- PMLiVE

Boehringer backs gene therapy firm Eyevensys

Invests in French eye disease specialist

- PMLiVE

Basilea’s Hans Christian Rohde takes up commercial role at uniQure

Will serve as gene therapy specialist's chief commercial officer

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links