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- PMLiVE

Sarepta Therapeutics

- PMLiVE

FDA acting commissioner Janet Woodcock requests independent review of Aduhelm approval

Approval of Biogen's Alzheimer's disease treatment has raised concerns over FDA staff interactions with the company

- PMLiVE

Pfizer, BioNTech eye US approval for COVID-19 booster dose

Companies plan to request an emergency use authorisation from the FDA for the booster dose in August

- PMLiVE

bluebird bio lodges counterclaim against Spark Therapeutics

Spark previously accused bluebird of misusing trademarks relating to the word ‘spark’ in marketing/educational materials

- 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

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

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

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

FDA advisory committee to review oncology accelerated approvals

Review comes after voluntary indication withdrawals for a number of cancer therapies

- 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

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