Gene therapy

After treating only one person with the ground-breaking therapy, uniqure is giving up on Glybera and shifting all its commercial manufacturing to the US

King’s College London has announced that it has recently entered into an exclusive license agreement with Pfizer Inc. for the development of a series of adeno-associated virus (AAV) gene therapy vectors

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the KU Leuven Laboratory for Molecular Virology and Gene Therapy.

Facilities are in place for early stage clinical trials but most are based in universities or hospitals, and commercial scale manufacturing is yet to come on-stream

uniqure NV, the Dutch company which won the first approval for a gene therapy, is taking its technology into the mainstream in a collaboration with Bristol-Myers Squibb to develop treatments for heart diseases