NEWS: January 2021

For the last two decades, the teams from Oxford and Edinburgh Universities and Imperial College, London have been working together to bring gene therapy to clinical practice for patients with cystic fibrosis (CF) and other respiratory conditions.

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Following our demonstration in 2015 that delivery of the CF gene using a non-viral carrier (liposome) can stabilise lung function in CF patients, when compared with placebo (Lancet Respir 2015;3:684-691), the Consortium has made exciting progress.

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Our attention has focused on producing the next generation carrier that we hope will allow a step-up in the efficiency of gene delivery to the lungs. Based on more than a decade of work, which ran in parallel with the non-viral approach, we have produced a novel viral carrier (lentivirus). This has some unique properties including:

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• High efficiency of gene transfer (orders of magnitude greater than the non-viral approach)

• Long duration of action (months to years in preclinical studies)

• The ability to demonstrate these properties on multiple applications

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Through experience, we have learnt that partnering with the pharmaceutical industry provides the fastest and most efficient route to bringing products to the clinic. We were, therefore, very pleased to announce in August 2018 that the lentivirus product had been partnered with the global pharmaceutical company Boehringer Ingelheim and the world leader in manufacturing of such viruses Oxford BioMedica.

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This unique collaboration has enabled the lentivirus product to move forward at pace towards a first clinical trial of gene therapy for patients with CF. The partnership is conducting an extensive programme, including optimising the manufacturing processes for the lentivirus and examining its safety profile in considerable detail.

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We can’t yet provide a firm date for a clinical trial because the ongoing programme clearly needs to generate the supporting data. If all continues to proceed well, the clinical programme is likely to start with a Phase I clinical trial including those with different types of CF gene mutations; the lentiviral approach is agnostic to which mutation is present.

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In parallel with our CF programme, through a £6.4M grant from the Wellcome, we are pushing forward the application of this novel lentivirus for other respiratory diseases. These include rare respiratory disorders such as surfactant deficiencies for which there is no treatment, α1-antitrypsin deficiency and interstitial lung diseases as well as a novel approach towards vaccination against respiratory conditions. We also have a programme of work using the lungs as a ‘factory’ to secrete proteins into the bloodstream.

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We will be regularly updating this new website with information for patients, scientists and the public, including a summary of current progress. Finally, may we warmly thank our many supporters and funders without whom we would not be at this exciting stage.

 

With best wishes

Eric

 

Eric Alton on behalf of the UK Respiratory Gene Therapy Consortium

January 2021

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