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- PMLiVE

Spark Therapeutics

- PMLiVE

bluebird bio’s beta-thalassaemia gene therapy receives FDA approval

Patients with the most severe form of the blood disorder develop life-threatening anaemia and have to receive regular blood transfusions

- PMLiVE

Pfizer set to acquire Global Blood Therapeutics in deal worth $5.4bn

The acquisition will help accelerate research into sickle cell disease

- PMLiVE

bluebird bio reveals plans to launch two gene therapies

Both therapies were endorsed by the US FDA Cell Tissue & Gene Therapy Advisory Committee

- PMLiVE

Sarepta to submit application for Duchenne muscular dystrophy gene therapy

The condition occurs in approximately one in every 3,500-5,000 newborn males worldwide

- PMLiVE

MHRA grants marketing authorisation to Global Blood Therapeutics for sickle cell disease drug

Oxybryta is the first treatment in Britain to directly block sickle haemoglobin polymerisation

- PMLiVE

Novartis and Precision BioSciences announce in vivo gene editing collaboration

The companies will work together to develop potentially curative treatments for disorders including sickle cell disease and beta thalassaemia

- PMLiVE

bluebird bio maintains its focus on gene therapy opportunities during structural changes

The company plans to reduce its workforce amid efforts to reduce its operating costs

- PMLiVE

Novartis and Voyager Therapeutics agree on gene therapy deal worth 1.7bn

The licence option agreement is for next-generation gene therapy vectors for neurological diseases

- PMLiVE

bluebird bio shares therapy updates for 2022

The company provided information for its first two gene therapies and its lovo-cel BLA submission for sickle cell disease

Takeda agrees on $2bn deal with Code Bio to expand its gene therapy programmes

The pharma company will gain opt-in rights for four rare diseases candidates

Biomarin

FDA keeps BioMarin’s gene therapy for phenylketonuria on hold

The decision follows data suggesting BioMarin’s experimental gene therapies may have the potential to progress cancers

- PMLiVE

bluebird bio’s beti-cel accepted for priority review by FDA

The tough journey to approval for beta-thalassemia gene therapy (beti-cel) in the US may be coming to an end, with approval possible by 2022

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