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Benitec Biopharma

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NHS earmarks £340m for new medicines fund to fast-track ‘innovative’ treatments

The proposed Innovative Medicines Fund is an extension of the existing Cancer Drugs Fund

Realizing the Potential of Genomics-Driven Precision Medicine

Jennifer Helfer, Senior Director, Patient Advocacy & Engagement at Encoded Therapeutics Inc., discusses genomics-driven precision medicine, gene therapy misconceptions and fears, involving patients and caregivers in designing gene therapy trials,...

Impetus Digital

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Researchers publish promising results for ultra-rare disease gene therapy method

Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency

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bluebird bio lodges counterclaim against Spark Therapeutics

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

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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

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First NHS patient treated with Novartis’ SMA gene therapy Zolgensma

Novartis and NHS England reached a landmark deal for the gene therapy earlier this year

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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

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Biogen and Capsigen to collaborate on gene therapies

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

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Pfizer pauses enrolment for trial evaluating anti-BCMA bispecific antibody

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

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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

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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

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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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