gene therapy

News

bluebird claims first approval for gene therapy Zynteglo in ...

bluebird bio has its first ever product approval, getting a green light from the EMA for Zynteglo, its ex vivo gene therapy for the rare blood disorder beta thalassaemia.

Market Access

pharmaphorum podcast bluebird bio Nicola Redfern

Gene therapy and market access: the pharmaphorum podcast

For episode 10 of the pharmaphorum podcast I spoke to Nicola Redfern, who’s general manager at bluebird bio in the UK.

News

engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

Amicus expands gene therapy collaboration

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania have announced a major expansion to their R&D collaboration for gene therapies.

News

BioMarin seeks haemophilia gene therapy approval after promi...

BioMarin is set to file for approval for its haemophilia gene therapy valoctocogene roxaparvovec, after it achieved pre-specified clinical criteria for regulatory review.

Events

Partner Content

SMi's 9th Annual Orphan Drugs & Rare Diseases Conference

Orphan Drugs & Rare Diseases

News

Former Alexion CEO Hallal makes comeback at cell therapy hol...

Massachusetts holding company ElevateBio has launched with $150m in initial funding from a range of investors on a mission to build a portfolio of cell and gene therapy companies.<

bluebird claims first approval for gene therapy Zynteglo in ...

Gene therapy and market access: the pharmaphorum podcast

Amicus expands gene therapy collaboration

BioMarin seeks haemophilia gene therapy approval after promi...

SMi's 9th Annual Orphan Drugs & Rare Diseases Conference

Former Alexion CEO Hallal makes comeback at cell therapy hol...

CDC panel votes to scrap hepatitis B birth dose advice

How to meet your physicians where they are online

gene therapy

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