Consortium Milestones Towards our Clinical Study Programme


Consortium publication on the cover of Molecular Therapy, February 2002

2001

The Consortium is formed on the initiative of the Cystic Fibrosis Trust.

2002-2003

Reorganisation of the three groups and creation of specific working groups and core facilities at each of the three sites.

2004

Development of the first CpG-Free plasmids.

Start of the product evaluation study to assess the effectiveness of multiple gene transfer agents (GTAs) in pre-clinical studies.

2005

End of product evaluation. GL67A selected as gene transfer agents (GTAs) for clinical studies.

pGM169
The current clinical trial plasmid, pGM169

2006

Tracking Study commences.

Creation of a modular clinical plasmid design (after extensive plasmid development).

hCEFI promoter established as having superior duration of expression in pre-clinical models (after multiple promoter elements are tested).

The clinical trial plasmid pGM169 produced in Oxford.

makingtheproduct
How we make the gene therapy product. Mixing the plasmid DNA and the GL67A.

2007

Completion of the Tracking Study.

Commercial manufacturing of GL67A and pGM169 begins.

Testing and selection of clinical nebulisers.

Extensive clinical assay development ahead of clinical studies.

Design of a mixing device for correct mixing of GL67A and pGM169.

2008

Run-in Study commences.

2009

Single Dose Clinical Trial (Pilot study) commences.

 

nebulisers
The nebuliser used to adminster the gene therapy.

2010

Extensive pre-clinical multi-dose toxicology studies commence (these are required by law ahead of multi-dose studies in CF patients).

2011

 

Completion of Single Dose Clinical Trial (Pilot study).

Extensive pre-clinical multi-dose toxicology studies completed.

 

 

2012

CF Trust withdraws funding from the Consortium (limited funding provided to March 2012).

Consortium awarded grants from the MRC & NIHR to continue clinical trial and research programmes.

Start of multi-dose Clinical trial to assess efficacy of GL67/pGM169 formulation.

Rosie Barnes launches Just Gene Therapy to raise funds for the Consortium.

 

2014 - Proposed

End of multi-dose Clinical trial towards the middle of 2013.

 

 

 

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