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- PMLiVE

haemophilia B

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

- PMLiVE

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

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

- 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

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

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