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NEWS: Monday, 6th December 2021


As you may have seen from the press release, the Consortium is now entering an exciting new phase of its partnership with Boehringer Ingelheim, which will take our novel lentivirus gene therapy platform technology to a first-in-man clinical trial in those with cystic fibrosis (CF).  We are contributing our 20+years of experience in the CF gene therapy field to help accelerate the various key elements of the programme that need to be covered before the trial. 
These include:

a.       Manufacturing the very large quantities of this lentivirus needed to efficiently treat the large surface area of the lungs into which we will nebulise the gene therapy

b.       Undertaking the toxicology studies required by the regulators to embark on the clinical trial

c.       Establishing new assays that will be required to detect the effects of the gene therapy in the trial

d.       Devising the clinical trial protocol
The first trial will focus on those with CF who are not sensitive to current modulators.  We will make further announcements regarding timing and clinical trial sites as soon as these are known; both are currently under active discussion.


Eric Alton, on behalf of the UK Respiratory Gene Therapy Consortium

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