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New industry partnership to develop gene therapy for cystic fibrosis

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A new partnership that brings together academics and pharmaceutical expertise has been established to focus on developing a gene therapy for cystic fibrosis patients. Cystic fibrosis (CF) is an inherited disease caused by defects in a single gene. The gene affected by CF controls the movement of salt and water in and out of cells. People with CF experience a build-up of thick sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms affecting the entire body. CF affects more than 10,400 people in the UK.

Researchers from the , which includes Imperial College London and the Universities of Oxford and Edinburgh, have been working towards making gene therapy for CF a clinical reality and have been at the forefront of this field for over 20 years. The GTC will now join with Boehringer Ingelheim and Oxford BioMedica to develop a new viral vector-based gene therapy to take forward to clinical trials.

This commercial collaboration is a great example of the GTC’s work that has been developed through collaborative support from the À¶Ý®ÊÓƵ À¶Ý®ÊÓƵ, À¶Ý®ÊÓƵ Respiratory Biomedical Research Unit at the Royal Brompton and Harefield NHS Foundation Trust, and awards by the À¶Ý®ÊÓƵ Efficacy and Mechanism Evaluation (EME) Programme and Medical Research Council. Read the full story by Ryan O’Hare (Imperial College London) .

People
  • Professor Eric Alton
    Professor Eric Alton
Publications
  • The Lancet
Partners
  • Medical Research Council
  • À¶Ý®ÊÓƵ Efficacy and Mechanism Evaluation Programme
  • À¶Ý®ÊÓƵ Royal Brompton Respiratory Biomedical Research Unit
  • UK Cystic Fibrosis Gene Therapy Consortium

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