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- PMLiVE

Decibel Therapeutics

- 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

Detailed results for Regeneron’s antibody cocktail continue to show benefit in high-risk COVID-19 outpatients

Treatment shortened symptom duration and reduced viral load in non-hospitalised patients with COVID-19

- PMLiVE

Developers of COVID-19 vaccines and therapeutics highlight continued need for treatments

Continued need for vaccines/treatments 'likely' as world moves into the next phase of the pandemic

- 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

Roche, Regeneron’s COVID-19 antibody cocktail cuts infection risk

REGEN-COV reduced the risk of symptomatic infections by 81% in a phase 3 prevention trial

- PMLiVE

Roche, Regeneron’s COVID-19 antibody cocktail cuts hospitalisation or death by 70%

Phase 3 trial is evaluating the therapy in high-risk non-hospitalised patients with COVID-19

- PMLiVE

Sanofi, Regeneron’s Libtayo shows marked reduction in risk of death in cervical cancer patients

Phase 3 trial showed drug reduced the risk of death by 31% compared to chemotherapy

- 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

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