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- PMLiVE

Restructuring

- PMLiVE

New data shows benefits for presymptomatic SMA patients treated with Zolgensma

Zolgensma was initially approved in 2019 for the treatment of SMA, launching with a list price of $2.125m

- PMLiVE

bluebird bio presents new data for beti-cel in beta thalassemia at EHA2021

Gene therapy shows sustained efficacy across paediatric and adult patient populations

- PMLiVE

FDA lifts clinical hold on bluebird bio’s LentiGlobin studies

Company previously put trials on hold after suspected unexpected serious adverse reactions

- PMLiVE

bluebird bio’s CALD gene therapy Skysona gains positive opinion from CHMP

CHMP's marketing authorisation recommendation is a step towards gaining EU approval

- PMLiVE

Sarepta Therapeutics’ DMD gene therapy shows early success

Most patients with Duchenne muscular dystrophy require full-time use of a wheelchair by their early teens

- PMLiVE

Biogen and Capsigen to collaborate on gene therapies

Biogen will gain exclusive right for undisclosed number of CNS and neuromuscular disease targets

- PMLiVE

bluebird bio aiming to separate business units by year-end

Biotech company has revealed that its new, separate oncology company will be named 2seventy bio

- PMLiVE

Pfizer pauses enrolment for trial evaluating anti-BCMA bispecific antibody

Trial pause follows reports of three cases of peripheral neuropathy in phase 1 study

- 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

bluebird bio anticipates lift of clinical holds on LentiGlobin in mid-2021

Previously reported case of MDS in phase 1/2 study has been re-classified as transfusion-dependent anaemia

- PMLiVE

BMS and bluebird bio gain FDA approval for multiple myeloma therapy

Abecma is a CAR T cell therapy approved for pre-treated myeloma patients

- PMLiVE

bluebird bio reveals long-term data for Lenti-D gene therapy in CALD

At 24 months of follow-up, 90% of patients were free of major functional disabilities

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