Respiratory gene therapy. Preclinical large animal safety/efficacy studies Image Dr Gerry McLachlan Research in a Nutshell The main long-term focus of my research has been on developing lung-directed gene therapy as a viable clinical entity. My group is part of the UK Respiratory Gene Therapy Consortium (GTC). The main focus of research has been developing the sheep and pig as models for pre-clinical development of lung-directed gene therapy protocols and includes both expertise in non-viral and viral gene delivery. At Roslin, we have delivered an MHRA-approved toxicology study of repeated administration of a cationic lipid-based formulation in the sheep lung which subsequently reached its primary clinical endpoint in a Phase IIb trial. GTC have also developed a lentiviral gene delivery platform based on an SIV virus (rSIV.F/HN) pseudotyped with the F and HN proteins from Sendai virus (SeV) for lung gene transfer. rSIV.F/HN transduces murine and sheep lungs and human ex vivo models efficiently and leads to gene expression at least 100-fold higher than the cationic lipid formulation GL67A. The GTC CF programme partnered with Boehringer Ingelheim in 2018 and in 2021, BI made the decision to exercise their option to licence the product and progress to clinical trial. GTC also has a well-established programme for the translation of gene therapy for a portfolio of diseases by exploiting the synergies provided by our respiratory gene delivery platform technology, a critical mass of researchers with complementary extensive expertise, the use of common resources, and respiratory gene transfer expertise. People Dr Gerry McLachlan Group Leader Liz Thornton Core Scientist/Lab Manager Dr Craig McDougall Postdoctoral Fellow Cathy McLaughlin Research Associate Contact gerry.mclachlan@roslin.ed.ac.uk HTML Collaborations Dr Chris Boyd, University of Edinburgh Prof Eric Alton, Imperial College Prof Uta Griesenbach, Imperial College Prof Deborah Dill , University of Oxford Prof Steve Hyde, University of Oxford Dr James Dixon, University of Nottingham Boehringer Ingelheim Oxford Biomedica Scientific Themes Respiratory gene therapy, large animal models, preclinical evaluation, respiratory disease, ex vivo models, image analysis for quantification of transduction efficiency & localisation of transduced cells. Technology Expertise Respiratory gene therapy, bronchoscopy, inhalation, large animal models, lung disease, lentiviral vectors, cationic liposomes, precision cut lung slice culture, ex vivo models. This article was published on 2024-09-23
