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- PMLiVE

beta-thalassemia

- 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

BMS, Acceleron’s Reblozyl shows new improvements for beta thalassemia patients

The data, presented at the EHA 2021 virtual congress, shows a 77% haemoglobin increase in patients treated with Reblozyl

- PMLiVE

FDA lifts clinical hold on bluebird bio’s LentiGlobin studies

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

- PMLiVE

FDA approves expanded use for Chiesi’s sickle cell drug Ferriprox

Iron-chelating agent approved for the treatment of transfusional iron overload due to SCD or other anaemias

- PMLiVE

Vertex to lead development of CRISPR Therapeutics-partnered gene therapy

Amended agreement sees Vertex take control of gene therapy programme

- 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

EMA recommends new manufacturing sites for COVID-19 vaccines

EMA also recommends new storage temperature for Pfizer/BioNTech vaccine

- PMLiVE

bluebird bio says AML case “very unlikely” to be related to gene therapy LentiGlobin

bluebird bio's gene therapy LentiGlobin is used to treat sickle cell disease

- PMLiVE

Novartis partners with Bill & Melinda Gates Foundation on ‘accessible’ sickle cell gene therapy

Project will aim to discover and develop a gene therapy for sickle cell disease

- PMLiVE

EMA fast-tracks review of bluebird bio’s LentiGlobin for sickle cell disease

Gene therapy has been given a PRIME designation

- PMLiVE

Bluebird bio’s sickle cell gene therapy shows early-study benefit

Closely follows approval of beta thalassaemia therapy Zynteglo

- PMLiVE

FDA backs GBT’s sickle cell drug Oxbryta

Second new treatment for blood disorder to be approved this year

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