About

About Us

In 2001 the UK's three leading cystic fibrosis gene therapy groups joined together as one organisation, the UK CF Gene Therapy Consortium (GTC), to pursue a single goal, namely to establish whether gene therapy can become a clinically viable option for patients with CF.

 

We developed a Wave 1 product (the CF gene delivered via a liposome), and were able for the first time to demonstrate a significant benefit in lung function compared with placebo in the world’s largest CF gene therapy trial. Since the trial ended, we have been exploring possibilities to produce higher levels of efficiency with the Wave 1 product. If this proves feasible we will push towards a further clinical trial. 

 

In parallel, building on knowledge from the Wave 1 programme, we have developed a novel viral vector to deliver the CF gene to the lungs (Wave 2 product). Translation into a first-in-man clincial trial has been supported by a Wellcome HICF grant (£2.7M). We have also joined forces with two world class organisations (Boehringer Ingelheim and Oxford BioMedica) in a major tripartite collaboration to catalyse translation of the Wave 2 product into clinical trials, and if successful, into routine clinical practice.

 

During our CF programme we noted that our novel lentivirus was capable of producing high levels of secreted proteins, of relevance to non-CF diseases. Following a successful award from the Wellcome Portfolio scheme (£6.4M) we are pursuing gene therapy for α1-antitrypsin deficiency, surfactant protein B deficiency, interstitial lung diseases and the production of antibodies of relevance to influenza and other infections.

 

About the Consortium Teams

 

As with any large organisation our biggest asset is our people and their skills, experience and expertise. The Consortium members are made up of the following; 

The Consortium is managed by our strategy group with representatives from all three sites bringing together the clinical, regulatory and scientific skills required to maximise the chances of translation into the clinic.

Our Story So Far...

Consortium Timeline

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

Consortium publication on the cover of Molecular Therapy, February 2002

Consortium publication on the cover of Molecular Therapy, February 2002

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

The clinical trial plasmid pGM169

The clinical trial plasmid pGM169

2005

  • End of product evaluation

  • GL67A selected as the optimal non-viral gene transfer agent (GTA) for clinical studies

2006

  • Tracking Study commences assessing the optimal outcome markers following an exacerbation

  • 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

  • Completion of the Tracking Study

2007

  • 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 assessing the optimal biomarkers during longitudinal follow-up over a two year period

  • Developing collaborations with DNAVEC (Tsukuba, Japan) over Wave 2 product

2009

The clinical trial mixing device

The clinical trial mixing device

2010

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

  • Change in CF Trust funding priorities

2011

  • Completion of Single Dose Clinical Trial (Pilot study)

  • Extensive pre-clinical multi-dose toxicology studies completed

2012

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

2013

  • Multidose clinical trial

Nebuliser_action.jpg

Nebuliser

2014

  • End of Multidose clinical trial

  • Extensive collaborations defined with ID Pharma (Tsukuba, Japan) over Wave 2 product

2015

  • Publication of the clinical trial results showing for the first time that repeated doses of gene therapy can produce clinically relevant benefit in the lungs of CF patients when compared with placebo

2016

  • Development of novel Wave 2 lentiviral product accelerated

2017

  • The GTC awarded a grant from the Wellcome/Health Innovation Challenge Fund (HICF) for £2.7M to undertake a Phase 1/2a nose trial in CF patients using our Wave 2 product, delivering the CFTR gene using a novel lentivirus

  • Cystic Fibrosis Trust awards an additional £0.5M to continue to support the scientific work of the GTC over the next two years

  • Flutterby Fundraisers sponsor Dr Aarash Saleh, Clinical Research Fellow, Imperial College London

  • The GTC secure a grant (£6.4M) from the Wellcome Innovation funding stream to use our underpinning rSIV.F/HN lentiviral vector platform applied to five non-CF projects

2018

  • The UK Cystic Fibrosis Gene Therapy Consortium, Boehringer Ingelheim, and Oxford BioMedica announce a major tripartite partnership to develop a first-in-class gene therapy for cystic fibrosis.

2019

  • The new partnership makes rapid progress with manufacturing of the novel lentiviral platform, begins the necessary toxicology studies that will underpin the clinical trial and develops further assays that will be needed to assess safety and efficacy in the trial.

2020

  • Clinical trial protocol developed

  • Toxicology and manufacturing studies continue