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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.

Audentes’ rare disease gene therapy programme delayed after third patient death

Audentes’ rare disease gene therapy programme delayed after third patient death

This was the third death of a patient involved in the Audentes’ ASPIRO clinical trial, which is evaluating its AT132 gene therapy in patients with X-linked myotubular myopathy (XLTM), a ... AT132 is an aden-associated virus (AAV) based gene therapy

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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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