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gene therapy trial

This page shows the latest gene therapy trial news and features for those working in and with pharma, biotech and healthcare.

FDA pauses BioMarin gene therapy trial

FDA pauses BioMarin gene therapy trial

The US Food and Drug Administration (FDA) has placed a clinical hold on the phase 1/2 Phearless study investigating BMN 307, a gene therapy from California-based rare disease biotech, ... More than 3, 000 patients have been treated with gene therapy, and

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  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    Nick Leschly talks to PME about Bluebird's remarkable gene therapy pipeline and why it’s filing first in Europe. ... Jesse Gelsinger. Probably the biggest blow came in 1999 when a young man named Jesse Gelsinger died when he took part in a gene therapy

  • Gene therapy Gene therapy

    the promise of gene therapy in the early nineties could not be remotely met. ... Therefore, the use of sub-optimal delivery vectors may continue to result in disappointing trial data and further hinder gene therapy.

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