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- PMLiVE

UniQure

- PMLiVE

Biogen and Capsigen to collaborate on gene therapies

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

- 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 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

FDA panel votes to keep accelerated approval for Roche’s Tecentriq in breast cancer

Oncologic Drugs Advisory Committee voted seven to two in favour of drug

- 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

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

- 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

Gene Editing and Precision Medicine: The Future of Healthcare?

Dr. Oscar Segurado, Chief Medical Officer at ASC Therapeutics, dives into advanced gene therapies, gene editing, and personalized medicine. We also explore the many different hats he wears as CMO, consultant,...

Impetus Digital

- 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

bluebird bio temporarily suspends trials of sickle cell gene therapy LentiGlobin

Company will also suspend marketing of beta-thalassemia gene therapy Zynteglo

- PMLiVE

NICE turns down bluebird bio’s gene therapy Zynteglo

Draft guidance does not recommend routine NHS funding for beta thalassaemia treatment

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