Development of a novel, potent, safe, long-lasting lentivirus-based gene therapy for cystic fibrosis
Lead Research Organisation:
University of Oxford
Department Name: RDM Clinical Laboratory Sciences
Abstract
Cystic Fibrosis (CF) is a common genetic disease that affects over 8000 people in the UK. It is the result of a mutation in a single gene called CFTR that causes the build up of thick sticky mucus in the lungs. Eventually the lungs become repeatedly infected and inflamed, leading to lung failure and premature death. All current therapies are aimed at coping with the symptoms; there is no cure. In 2001, three research groups in the UK formed the CF Gene Therapy Consortium to develop a new kind of treatment based on introducing new (healthy) copies of the CFTR gene into the lungs of people with CF. Many advances have been made, and we have learned a great deal about the obstacles to this approach, but we are not yet in a position to offer an effective gene therapy for CF lung disease. The aim of this research application is to use a new kind of gene therapy based on a virus called lentivirus, which we think has the potential to be more efficient at delivering the gene into the lungs and to provide an effective treatment for CF lung disease. In order to make the lentivirus carrying the CF gene as effective as possible, it is wrapped up in two new coat proteins called F and HN, a process known as 'pseudotyping'. We have shown that when the lentivirus is pseudotyped with F and HN it is efficient at entering lung cells and the gene it carries can be expressed for many months, even years in some situations. Another key feature that this pseudotyped virus displays is that it can be repeatedly administered, a property we have not seen previously with other viruses. This has an important advantage for treating chronic diseases, such as CF where the effects last for the life-time of the individual. The objectives of this application are i) to engineer the virus so it is suitable for use in patients; ii) to improve virus production methods so that it can be made in sufficient quantities to use as a therapy; and iii) to understand the potential for side-effects associated with administering the virus. The research project is 18 months long and at the end of it we hope to have selected a final version of the virus that we can take forward to test in patients. If we are successful in manufacturing the virus and selecting a suitable version to give to patients, we envisage that it could be delivered to the lungs of CF individuals using a device called a nebuliser, which generates a fine mist that can be breathed in. The increased efficiency of this virus along with the ability to have an effect that is long-lived effect means that this could form the basis of a new, more effective treatment for CF.
Technical Summary
Cystic fibrosis (CF) is the most common lethal inherited disease of Caucasian populations, affecting ~80,000 individuals worldwide. In the UK, the current median age at death is ~25 years. Current treatments are focused on symptomatic relief and are associated with a huge treatment burden, and high individual NHS patient costs. No treatment is available that targets the genetic defect.
We have developed a novel, IP-protected, lentiviral gene transfer vector, specifically pseudotyped to enable efficient airway cell uptake. We hypothesise that CFTR gene transfer into the CF lung by this vector system will provide an effective treatment for CF lung disease.
The vector directs stable gene expression in lung models at levels thought to be within the therapeutic range. Uniquely, this vector system suffers no loss of efficacy upon repeated administration; with transgene expression remaining ~2 logs higher than current competing vector technologies.
Prior to final candidate selection key aspects of the vector system must be enhanced. Firstly, the current vector system is not fully compliant with pharmacopoeial regulations; however, straightforward sequence manipulation will rectify this issue. Secondly, the current vector purification methodology is difficult to scale. Simple method development, utilising steps previously applied in the field, will provide a scaleable GMP compatible production process. Thirdly, concerns driven by clinical findings from the use of early-generation retroviral vectors, requires an understanding of the effect of the vector transgene expression cassette upon theoretical insertion site-specific clonal expansion. Consequently, insertion site profiling is prudent prior to seeking regulatory approval for clinical studies. We outline a milestone-driven, 18 months translational research programme to bridge our current pre-clinical data package to regulatory toxicology studies and first-in-man trials.
We have developed a novel, IP-protected, lentiviral gene transfer vector, specifically pseudotyped to enable efficient airway cell uptake. We hypothesise that CFTR gene transfer into the CF lung by this vector system will provide an effective treatment for CF lung disease.
The vector directs stable gene expression in lung models at levels thought to be within the therapeutic range. Uniquely, this vector system suffers no loss of efficacy upon repeated administration; with transgene expression remaining ~2 logs higher than current competing vector technologies.
Prior to final candidate selection key aspects of the vector system must be enhanced. Firstly, the current vector system is not fully compliant with pharmacopoeial regulations; however, straightforward sequence manipulation will rectify this issue. Secondly, the current vector purification methodology is difficult to scale. Simple method development, utilising steps previously applied in the field, will provide a scaleable GMP compatible production process. Thirdly, concerns driven by clinical findings from the use of early-generation retroviral vectors, requires an understanding of the effect of the vector transgene expression cassette upon theoretical insertion site-specific clonal expansion. Consequently, insertion site profiling is prudent prior to seeking regulatory approval for clinical studies. We outline a milestone-driven, 18 months translational research programme to bridge our current pre-clinical data package to regulatory toxicology studies and first-in-man trials.
Planned Impact
Who will benefit from this research?
The ultimate objective of this research is to develop a treatment for the serious life threatening disease cystic fibrosis (CF). CF is the most common lethal inherited disease of Caucasian populations, International patient registries indicate that there are ~80,000 CF subjects in the EU, North America and Australia/New Zealand. Approximately 10% of this population live in the UK. In addition, some estimates suggest that an additional ~200K CF subjects are unidentified in emerging pharmaceutical markets. Typically, CF individuals suffer from repeated bacterial infections of the conducting airways leading to lung failure. Current treatments are focused on symptomatic relief and are associated with a huge treatment burden, requiring several hours of self-administered therapy daily. In the UK, the median age at death is ~25 years. NHS costs per patient range from £15 - £50K per year, with an annual direct healthcare cost of ~£200M. No treatment is available that targets the genetic defect; bridging this gap has been shown to be the highest priority of patients and carers.
Our solution to this problem is a novel, lentiviral gene transfer vector (termed F/HN-SIV), specifically engineered to enable efficient airway cell uptake. F/HN-SIV directs stable gene expression in cells relevant to CF lung disease for ~2 years following a single application. We hypothesise that gene transfer of a copy of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene into the CF lung by this repeatedly administrable vector will provide an effective treatment for CF lung disease.
The F/HN-SIV vector is the class leading solution to the problem of delivering nucleic acid-based therapeutics to the lung. As a platform for lung delivery additional therapeutic intervention options include: (1) the delivery of CFTR or other transgenes to treat acquired or inherited lung diseases - eg alpha 1-antitrypsin to treat emphysema/ chronic obstructive pulmonary disease (COPD), (2) the delivery of inhibitory molecules to treat viral infections or pre-existing lung disease - eg RNAi/miRNA class sequences to teat influenza/asthma, and (3) the delivery of transgenes to lung cells acting as a secreted protein factory to treat systemic disease - eg Factor VIII or IX to treat haemophilias.
How will end-users benefit from this research?
While modern CF treatment regimens have extended the life expectancy of a CF child, CF remains a lethal disease with no cure. Typically, CF patients require twice-daily regimens of physiotherapy and nebuliser treatments, supplemented with numerous orally delivered medications, imposing a high therapeutic burden (3-4 hours treatment/day is not unusual) on them and their carers. Successful gene therapy will provide benefits ranging from a reduction in (i) the conventional treatment burden, and (ii) time in hospital, manifesting through an increased life expectancy, to prevention of lung disease if treatment is started early after diagnosis. Our experience is that there is enthusiasm for gene therapy amongst CF individuals, but also a sense of realism. If successful CF gene therapy will have a dramatic effect on patient well-being, and the potential benefits to end users are large.
Following this 18 month project, we estimate it will take ~2.5 years to complete the required safety toxicology studies before a first-in-man clinical trial can be initiated. If successful, a CF treatment based on F/HN-SIV technology could be marketed within 10 years.
The ultimate objective of this research is to develop a treatment for the serious life threatening disease cystic fibrosis (CF). CF is the most common lethal inherited disease of Caucasian populations, International patient registries indicate that there are ~80,000 CF subjects in the EU, North America and Australia/New Zealand. Approximately 10% of this population live in the UK. In addition, some estimates suggest that an additional ~200K CF subjects are unidentified in emerging pharmaceutical markets. Typically, CF individuals suffer from repeated bacterial infections of the conducting airways leading to lung failure. Current treatments are focused on symptomatic relief and are associated with a huge treatment burden, requiring several hours of self-administered therapy daily. In the UK, the median age at death is ~25 years. NHS costs per patient range from £15 - £50K per year, with an annual direct healthcare cost of ~£200M. No treatment is available that targets the genetic defect; bridging this gap has been shown to be the highest priority of patients and carers.
Our solution to this problem is a novel, lentiviral gene transfer vector (termed F/HN-SIV), specifically engineered to enable efficient airway cell uptake. F/HN-SIV directs stable gene expression in cells relevant to CF lung disease for ~2 years following a single application. We hypothesise that gene transfer of a copy of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene into the CF lung by this repeatedly administrable vector will provide an effective treatment for CF lung disease.
The F/HN-SIV vector is the class leading solution to the problem of delivering nucleic acid-based therapeutics to the lung. As a platform for lung delivery additional therapeutic intervention options include: (1) the delivery of CFTR or other transgenes to treat acquired or inherited lung diseases - eg alpha 1-antitrypsin to treat emphysema/ chronic obstructive pulmonary disease (COPD), (2) the delivery of inhibitory molecules to treat viral infections or pre-existing lung disease - eg RNAi/miRNA class sequences to teat influenza/asthma, and (3) the delivery of transgenes to lung cells acting as a secreted protein factory to treat systemic disease - eg Factor VIII or IX to treat haemophilias.
How will end-users benefit from this research?
While modern CF treatment regimens have extended the life expectancy of a CF child, CF remains a lethal disease with no cure. Typically, CF patients require twice-daily regimens of physiotherapy and nebuliser treatments, supplemented with numerous orally delivered medications, imposing a high therapeutic burden (3-4 hours treatment/day is not unusual) on them and their carers. Successful gene therapy will provide benefits ranging from a reduction in (i) the conventional treatment burden, and (ii) time in hospital, manifesting through an increased life expectancy, to prevention of lung disease if treatment is started early after diagnosis. Our experience is that there is enthusiasm for gene therapy amongst CF individuals, but also a sense of realism. If successful CF gene therapy will have a dramatic effect on patient well-being, and the potential benefits to end users are large.
Following this 18 month project, we estimate it will take ~2.5 years to complete the required safety toxicology studies before a first-in-man clinical trial can be initiated. If successful, a CF treatment based on F/HN-SIV technology could be marketed within 10 years.
Publications
Alton EW
(2016)
Genetic medicines for CF: Hype versus reality.
in Pediatric pulmonology
Alton EW
(2017)
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis.
in Thorax
Davidson H
(2014)
Pediatric Pulmonary
Davies, LA
(2014)
LARGE-SCALE PRODUCTION OF LENTIVIRAL VECTORS FOR CF LUNG GENE THERAPY
Gelinas J
(2016)
704. Enhanced Lentiviral Production Through Rational Design of Mammalian Host Cells
in Molecular Therapy
Gill DR
(2014)
Delivery of genes into the CF airway.
in Thorax
Griesenbach U
(2012)
Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy.
in American journal of respiratory and critical care medicine
Griesenbach U
(2013)
Moving forward: cystic fibrosis gene therapy.
in Human molecular genetics
Griesenbach U
(2016)
534. Preparation for a First-in-Man Lentivirus Trial in Cystic Fibrosis Patients
in Molecular Therapy
Griesenbach, U
(2014)
Moving towards assessment of lentiviral vector in clinical trial
Gélinas JF
(2018)
Multiple Inhibitory Factors Act in the Late Phase of HIV-1 Replication: a Systematic Review of the Literature.
in Microbiology and molecular biology reviews : MMBR
Gélinas JF
(2017)
Assessment of selected media supplements to improve F/HN lentiviral vector production yields.
in Scientific reports
Hyde S
(2014)
P203 Development Of An Optimal F/hn Pseudotyped Siv Vector For Cf Gene Therapy
in Thorax
Leoni G
(2015)
Ex Vivo and In Vivo Lentivirus-Mediated Transduction of Airway Epithelial Progenitor Cells.
in Current gene therapy
Miah K
(2019)
Emerging gene therapies for cystic fibrosis
in Expert Review of Respiratory Medicine
Nicklin SA
(2015)
Special focus issue on the annual meeting of the British Society for Gene and Cell Therapy.
in Human gene therapy
Paul-Smith MC
(2018)
The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins.
in Gene therapy
Van Haasteren J
(2018)
Lessons learned from lung and liver in-vivo gene therapy: implications for the future.
in Expert opinion on biological therapy
Description | Membership of strategy implementation board of the cystic fibrosis trust. |
Geographic Reach | National |
Policy Influence Type | Participation in a guidance/advisory committee |
Description | Airway epithelium as a factory for production of blood clotting factors |
Amount | £52,362 (GBP) |
Funding ID | P47167 |
Organisation | Imperial College London |
Sector | Academic/University |
Country | United Kingdom |
Start | 07/2013 |
End | 05/2014 |
Description | An Interim application to fund UK Gene Therapy Consortium |
Amount | £96,009 (GBP) |
Funding ID | GT013 |
Organisation | Cystic Fibrosis Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 12/2013 |
End | 12/2014 |
Description | An Interim application to fund the UK Gene Therapy Consortium |
Amount | £170,213 (GBP) |
Funding ID | GT012 |
Organisation | Cystic Fibrosis Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 01/2013 |
End | 02/2014 |
Description | An application to fund the UK CF Gene Therapy Consortium core costs |
Amount | £142,693 (GBP) |
Organisation | Cystic Fibrosis Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 03/2014 |
End | 08/2016 |
Description | An interim application to fund the UK Gene Therapy Consortium |
Amount | £41,778 (GBP) |
Funding ID | GT014 |
Organisation | Cystic Fibrosis Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 01/2013 |
End | 02/2014 |
Description | Confidence in Concept |
Amount | £49,942 (GBP) |
Organisation | University of Oxford |
Department | Medical Research Council Confidence in Concept Fund |
Sector | Academic/University |
Country | United Kingdom |
Start | 06/2014 |
End | 06/2014 |
Description | EIT Health Innovations |
Amount | € 30,000 (EUR) |
Organisation | European Institute of Innovation and Technology (EIT) |
Sector | Public |
Country | Hungary |
Start | 09/2016 |
End | 04/2017 |
Description | First-in-human trial of an optimised lentivirus vector for cystic fibrosis gene therapy |
Amount | £2,773,098 (GBP) |
Funding ID | HICF-R10-698 |
Organisation | Health Innovation Challenge Fund |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 03/2017 |
End | 11/2022 |
Description | GT consortia Core Funding |
Amount | £750,000 (GBP) |
Organisation | Medicor Foundation |
Sector | Charity/Non Profit |
Country | Liechtenstein |
Start | 03/2015 |
End | 06/2017 |
Description | Gene Therapy for Surfactant Disorders |
Amount | £3,000 (GBP) |
Funding ID | 1966167 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 08/2018 |
End | 09/2022 |
Description | NIHR RBRU Pump Priming |
Amount | £19,000 (GBP) |
Funding ID | P45585 |
Organisation | Royal Brompton & Harefield NHS Foundation Trust |
Sector | Public |
Country | United Kingdom |
Start | 11/2012 |
End | 10/2013 |
Description | RDM Pump Priming Award |
Amount | £25,000 (GBP) |
Organisation | University of Oxford |
Department | Radcliffe Department of Medicine |
Sector | Academic/University |
Country | United Kingdom |
Start | 05/2014 |
End | 05/2014 |
Description | Respiratory Gene Therapy Portfolio |
Amount | £6,402,078 (GBP) |
Funding ID | 110579/Z/15/Z |
Organisation | Wellcome Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 03/2017 |
End | 09/2022 |
Description | University Challenge Seed Fund |
Amount | £20,000 (GBP) |
Funding ID | Enhancement of Lentiviral Production |
Organisation | University of Oxford |
Department | Oxford University Innovation |
Sector | Private |
Country | United Kingdom |
Start | 12/2016 |
Title | Novel integrating & non-integrating Lentiviral vectors |
Description | Plasmids designed to generate recombinant Lentiviral vectors |
Type Of Material | Technology assay or reagent |
Year Produced | 2012 |
Provided To Others? | Yes |
Impact | N/a at this stage |
Title | cGMP-compliant Lentiviral production methods |
Description | methods to manufacture cGMP Lentiviral vectors |
Type Of Material | Technology assay or reagent |
Provided To Others? | No |
Impact | Multiple lots of Lentiviral vectors manufactured for non clinical toxicological studies. |
Description | DNAVEC and UK Cystic Fibrosis Gene Therapy Consortium |
Organisation | DNAVEC Corporation |
Country | Japan |
Sector | Private |
PI Contribution | * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. |
Collaborator Contribution | DNAVEC: * Technology Transfer to the Consortium University of Edinburgh and ICSTM. * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. Imperial innovations: * IP Management |
Impact | Publication number; WO2007049752 Publication number; WO2005001082 Publication number; WO0238786 |
Start Year | 2010 |
Description | DNAVEC and UK Cystic Fibrosis Gene Therapy Consortium |
Organisation | Imperial College London |
Department | National Heart & Lung Institute (NHLI) |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. |
Collaborator Contribution | DNAVEC: * Technology Transfer to the Consortium University of Edinburgh and ICSTM. * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. Imperial innovations: * IP Management |
Impact | Publication number; WO2007049752 Publication number; WO2005001082 Publication number; WO0238786 |
Start Year | 2010 |
Description | DNAVEC and UK Cystic Fibrosis Gene Therapy Consortium |
Organisation | Imperial Innovations |
Country | United Kingdom |
Sector | Private |
PI Contribution | * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. |
Collaborator Contribution | DNAVEC: * Technology Transfer to the Consortium University of Edinburgh and ICSTM. * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. Imperial innovations: * IP Management |
Impact | Publication number; WO2007049752 Publication number; WO2005001082 Publication number; WO0238786 |
Start Year | 2010 |
Description | DNAVEC and UK Cystic Fibrosis Gene Therapy Consortium |
Organisation | University of Edinburgh |
Department | Institute of Genetics & Molecular Medicine |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. |
Collaborator Contribution | DNAVEC: * Technology Transfer to the Consortium University of Edinburgh and ICSTM. * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. Imperial innovations: * IP Management |
Impact | Publication number; WO2007049752 Publication number; WO2005001082 Publication number; WO0238786 |
Start Year | 2010 |
Description | DNAVEC and UK Cystic Fibrosis Gene Therapy Consortium |
Organisation | University of Oxford |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. |
Collaborator Contribution | DNAVEC: * Technology Transfer to the Consortium University of Edinburgh and ICSTM. * Vector improvements/modifications * Assessment of genotoxicity and biodistribution * Vector production and scale-up. Imperial innovations: * IP Management |
Impact | Publication number; WO2007049752 Publication number; WO2005001082 Publication number; WO0238786 |
Start Year | 2010 |
Description | Development of Lentiviral platform for lung gene transfer to treat Cystic Fibrosis lung disease |
Organisation | Boehringer Ingelheim |
Country | Germany |
Sector | Private |
PI Contribution | Provision of recombinant lentiviral platform and expertise, for delivery of CFTR to lungs of Cystic Fibrosis patients |
Collaborator Contribution | Provision of expertise in development of treatments for respiratory disease |
Impact | Collaboration only started very recently. No outputs so far. |
Start Year | 2018 |
Title | GENE TRANSFER INTO AIRWAY EPITHELIAL STEM CELL BY USING LENTIVIRAL VECTOR PSEUDOTYPED WITH RNA VIRUS SPIKE PROTEIN |
Description | The present inventors successfully introduced genes into stem cells of airway epithelial tissues using simian immunodeficiency virus vectors pseudotyped with F and HN, which are envelope glycoproteins of Sendai virus. Gene transfer into airway epithelial tissue stem cells using a vector of the present invention is useful for gene therapy of genetic respiratory diseases such as cystic fibrosis. Furthermore, it is possible to select respiratory organs such as the lungs as production tissues for providing proteins that are deficient due to genetic diseases. |
IP Reference | EP1950307 |
Protection | Patent granted |
Year Protection Granted | 2008 |
Licensed | Yes |
Impact | This patent is owned by DNAVEC Corp (now ID Pharma Co.). and forms part of the Core Technology and Background IP. A research and sub-licensable commercial use licence under this patent has been granted to Imperial College, Universities of Oxford and Edinburgh, and Imperial Innovations Ltd. Granted in US, EP (parent and divisional, validated in BE, AT, CH, CZ, DE, ES, FR, GB, IE, IT, NL), CA and pending in US (continuation), China and S Korea |
Title | LENTIVIRAL VECTORS |
Description | This invention relates to lentiviral gene transfer vectors pseudotyped with hemagglutinin- neuraminidase (HN) and fusion (F) proteins from a respiratory paramyxovirus, comprising a promoter and a transgene; and methods of making the same. The present invention also relates to the use of said vectors in gene therapy, particularly for the treatment of respiratory tract diseases such as Cystic Fibrosis (CF). |
IP Reference | WO2015177501 |
Protection | Patent application published |
Year Protection Granted | 2015 |
Licensed | Yes |
Impact | This patent is owned jointly between DNAVEC Corp (now ID Pharma Co.). and Imperial Innovations Ltd, and forms part of the Core Technology and Background IP. A research and sub-licensable commercial use licence under this patent has been granted to Imperial College, Universities of Oxford and Edinburgh, and Imperial Innovations Ltd. Patent applications are granted in US, EP, AU, JP and pending in EA, CA |
Title | PSEUDO-TYPE RETROVIRUS VECTOR CONTAINING MEMBRANE PROTEIN HAVING HEMAGGLUTININ ACTIVITY |
Description | A retrovirus vector containing a membrane protein having a hemagglutinin activity. By using the membrane protein having a hemagglutinin activity, the retrovirus vector of the pseudo-type is constructed. This virus vector shows a high gene transfer efficiency into host cells. It is clarified that genes can be transferred thereby at a high efficiency into cells into which genes can be hardly transferred by the conventional techniques, for example, blood cells and hematopoietic cells involving hematopoietic stem cells and mucous cells involving mucosa epithelial cells. This virus vector is highly useful as a vector for gene therapy. |
IP Reference | WO0192508 |
Protection | Patent granted |
Year Protection Granted | 2001 |
Licensed | Yes |
Impact | This patent is owned by DNAVEC Corp (now ID Pharma Co.). and forms part of the Core Technology and Background IP. A research and sub-licensable commercial use licence under this patent has been granted to Imperial College, Universities of Oxford and Edinburgh, and Imperial Innovations Ltd. The patent has also been granted in Canada ( CA2413995), Europe (EP1291419), US (US7510706), Japan ( JP4700888), China (CN100531802) and Korea (KR100807016). |
Title | RECOMBINANT SENDAI VIRUS VECTOR FOR INTRODUCING EXOGENOUS GENES TO AIRWAY EPITHELIA |
Description | Provided are a recombinant Sendai virus vector for introducing exogenous genes to airway epithelia and a method for introducing exogenous genes using the vector. The recombinant Sendai virus vector enables efficient gene transfer to native mucus-layered airway epithelial cells by briefly contacting the vector with the cells. Furthermore, the vector can introduce genes to not only apical surfaces but also submucosal glands where CFTR primarily expresses. The vector can thus be used for gene therapy of CF, a CFTR-deficient disease. |
IP Reference | WO0132898 |
Protection | Patent granted |
Year Protection Granted | 2001 |
Licensed | Yes |
Impact | This patent is owned by DNAVEC Corp. (now ID Pharma Co.) and forms part of the Core Technology and Background IP. A research and sub-licensable commercial use right under this patent has been granted to Imperial College, Universities of Oxford and Edinburgh, and Imperial Innovations Ltd. The patent has also been granted in US (US7314614), Canada ( CA2389743) and Japan ( JP4838962). |
Description | 38th European Cystic Fibrosis Conference |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Other audiences |
Results and Impact | Extensive interest in world 1st non-viral trial of Gene Therapy aimed at Clinical improvement. Discussions underway with Pharma for follow on study. |
Year(s) Of Engagement Activity | 2015 |
Description | 3rd Annual CF Science Appreciation Dinner (2015) |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Patients, carers and/or patient groups |
Results and Impact | 3rd Annual CF Science Appreciation Dinner. 25 fund raisers, care givers and scientists involved with cystic fibrosis research. Networking and speaking event which sparked questions and discussions. |
Year(s) Of Engagement Activity | 2015 |
Description | ASSCR/AGCTS breakfast session supported by Miltenyi Biotec - Clinical translation of CAR T and iPS cell therapies: Sydney Australian Gene Therapy Meeting |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public. |
Year(s) Of Engagement Activity | 2017 |
Description | Academy of Medical Sciences and the Cell and Gene Therapy Catapult on 'The future of gene therapies next - steps for the UK' |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2019 |
Description | Advanced Therapies Network Launch event |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Industry/Business |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2018 |
Description | Alport Society Annual Meeting - CF Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | Alports Syndrome International Workshop (2015) |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Presentation to ~150 people attending international workshop on Alports syndrome. Individuals included patients, carers and scientific researchers. Introduced new therapeutic concepts. Wide ranging discussions followed. |
Year(s) Of Engagement Activity | 2015 |
Description | Animal models Conference - Cystic fibrosis |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | Annual A-Level GCSE student Practical |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Type Of Presentation | Workshop Facilitator |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | Annually 5-6 attend the workshop, often inspired to take up careers in Science and Medicine. Increase in applications to Science and Medical Universities. |
Year(s) Of Engagement Activity | 2009,2010,2011,2012,2013 |
Description | Annual American Society for Gene and Cell Therapy - Selection of Regulator-compliant Optimal Lentiviral Vector Configurations for Progression into CF Clinical Trials |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Type Of Presentation | keynote/invited speaker |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public Stimulated enthusiasm aorund GT trial |
Year(s) Of Engagement Activity | 2014 |
Description | Annual North American CF Conference - British Society for Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlight awareness of GT and CF to the wider public |
Year(s) Of Engagement Activity | 2013 |
Description | BBC Radio 5Live Breakfast Show (2015) |
Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | BBC Radio 5Live Breakfast show. Broadcast interview to discuss clinical trial results |
Year(s) Of Engagement Activity | 2015 |
Description | BBC Radio 5Live Your Call Show (2016) |
Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | Was asked to act as scientific expert regarding cystic fibrosis during national radio phone in show. |
Year(s) Of Engagement Activity | 2016 |
URL | http://www.bbc.co.uk/programmes/b0717346 |
Description | BBC Radio London (2015) |
Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | BBC Radio London Breakfast Show. Broadcast interview to discuss clinical trial results. |
Year(s) Of Engagement Activity | 2015 |
Description | BBC Radio Oxford Drivetime Show (2015) |
Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | BBC Radio Oxford Drivetime Show. Broadcast interview to discuss clinical trial results. |
Year(s) Of Engagement Activity | 2015 |
Description | BBC Radio2 Jeremy Vine Show (2015) |
Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | BBC Radio2 Jeremy Vine Show. Broadcast interview to discuss clinical trial results. |
Year(s) Of Engagement Activity | 2015 |
Description | BBC World Service - World Have Your Say - 2017/03/03 |
Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Public/other audiences |
Results and Impact | BBC World Service Discussion Programme regarding gene therapy in general, specially the use of lentiviral vectors and the correction of sickle cell anaemia. |
Year(s) Of Engagement Activity | 2017 |
URL | http://www.bbc.co.uk/programmes/p04tv5n9 |
Description | BBC2 TV Victoria Derbyshire Show (2015) |
Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | BBC2 TV Victoria Derbyshire Show. Broadcast interview to discuss clinical trial results. |
Year(s) Of Engagement Activity | 2015 |
Description | BSGCT Public Engagement Day Oxford 2014 |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | Local event at Oxford Natural History museum to debate gene therapy for a variety of diseases, attended by local school children |
Year(s) Of Engagement Activity | 2014 |
Description | BSGCT Public Engagement Day Oxford 2015 |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Schools |
Results and Impact | One day of engagement (workshops and talks and discussions) which local schools, also open to general public |
Year(s) Of Engagement Activity | 2015 |
Description | BTS Winter Meeting (London) |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Extensive interest in world 1st non-viral trial of Gene Therapy aimed at Clinical improvement. Discussions underway with Pharma for follow on study. |
Year(s) Of Engagement Activity | 2015 |
Description | Biology in Action |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Schools |
Results and Impact | Highlighting the awareness of CF and GT to the wider public. |
Year(s) Of Engagement Activity | 2017 |
Description | CATO Research Symposium |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2018 |
Description | CF Unite, Nottingham - CF Genetics and Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2013 |
Description | CF gene therapy (Probus: Nov 2017) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | CB was invited to talk to the Bishopbriggs Probus Club about the group's research into CF gene therapy. (Probus clubs have regular programmes of talks on a variety of topics.) The audience was a general one, and the presentation sparked a lively debate. |
Year(s) Of Engagement Activity | 2017 |
Description | Cafe Scientific Talk (Bradfield College 2015) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | Science discussion evenning event at Bradfield College. Wide ranging audience from school children to CF care givers and relatives. Talk stimulated extensive discussion and questions. |
Year(s) Of Engagement Activity | 2015 |
Description | Cafe Scientific Talk Bradfield College 2015 |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | Science discussion evening event Extensive Q & A session |
Year(s) Of Engagement Activity | 2015 |
Description | Cystic Fibrosis Trust: Lungs for the Life Conference (Manchester) |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Extensive interest in world 1st non-viral trial of Gene Therapy aimed at Clinical improvement. Discussions underway with Pharma for follow on study. |
Year(s) Of Engagement Activity | 2015 |
Description | Dr Stephen Hyde attended Cystic Fibrosis Foundation to conduct grant reviews |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Dr Stephen Hyde attended Cystic Fibrosis Foundation to conduct grant reviews |
Year(s) Of Engagement Activity | 2015 |
Description | Dr Stephen Hyde contributed to grant discussions for the Cystic Fibrosis Foundation |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Dr Stephen Hyde contributed to expect discussions for the Cystic Fibrosis Foundation to conduct grant reviews |
Year(s) Of Engagement Activity | 2016 |
Description | ECFS Diagnostic Network Working Group - CFTR gene therapy: What does the future hold |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2016 |
Description | ESGCT - BSGCT Spring School, Oxford |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2017 |
Description | ESGCT Congress Berlin |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public. |
Year(s) Of Engagement Activity | 2017 |
Description | Emerging gene therapies for CF |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Professional Practitioners |
Results and Impact | Presentation to a group of Health professionals informing on Emerging Gene Therapies for CF, which sparked questions and discussion afterwards. |
Year(s) Of Engagement Activity | 2018 |
Description | Eoin MacReamoinn presented at talk on Gene Therapy for Cystic Fibrosis |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Other audiences |
Results and Impact | Eoin MacReamoinn one of our Research Assistants presented a talk on Gene Therapy for Cystic Fibrosis to a group of Scouts which resulted in a donation to the group from their fundraising efforts throughout the year |
Year(s) Of Engagement Activity | 2018 |
Description | European Respiratory Society Annual Meeting - Update on CF Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | European Young Investigators Meeting, Paris |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2019 |
Description | Gene Therapy: A Brave New World |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Public/other audiences |
Results and Impact | Purpose was to enlighten audience about Gene Therapy and its potential which resulted in discussion and personal donations from members of the public. |
Year(s) Of Engagement Activity | 2018 |
Description | German Mukoviscidose Gesellschaft Annual Meeting - Moving forward with CF Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | German Society for Gene Therapy (Vienna) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Extensive interest in world 1st non-viral trial of Gene Therapy aimed at Clinical improvement. Discussions underway with Pharma for follow on study. |
Year(s) Of Engagement Activity | 2015 |
Description | Guest speaker at BSGCT conference London |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Postgraduate students |
Results and Impact | Presented a talk titled: Scale Up challenges - an academic view. |
Year(s) Of Engagement Activity | 2018 |
Description | Guest speaker at CFF Research conference at Jackson Hole Wyoming USA |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a talk on Sendai Virus Pseudotyped Lentiviral Vectors for CF Therapy. |
Year(s) Of Engagement Activity | 2018 |
Description | Guest speaker at ESGCT Spring School Oxford |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Postgraduate students |
Results and Impact | Presented a talk titled: How to produce high quality vectors in lab scale. |
Year(s) Of Engagement Activity | 2018 |
Description | Guest speaker at European Bioanalytical forum in Barcelona |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Presented a talk titled: Cell & Gene Therapy - Where are we and where are we going? |
Year(s) Of Engagement Activity | 2018 |
Description | IGMM Doors Open Day 2018 |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | The research building that houses Chris Boyd's lab group was opened to the public as part of the Cockburn's Doors Open Day on 29th Sept 2018. Over 200 members of the public enjoyed a programme of building tours, drop in activities, scientific talks and poster presentations. A poster entitled 'Cystic Fibrosis Gene Therapy' gained a lot of interest and many participants spent time engaging in lengthy Q&A sessions relating to this work. |
Year(s) Of Engagement Activity | 2018 |
Description | Imperial College Fringe Event |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | Yes |
Type Of Presentation | Workshop Facilitator |
Geographic Reach | Local |
Primary Audience | Public/other audiences |
Results and Impact | Communication of science to the public Wide spread enthusiasm amongst participates. |
Year(s) Of Engagement Activity | 2013 |
Description | Imperial Festival |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | Yes |
Type Of Presentation | Workshop Facilitator |
Geographic Reach | Local |
Primary Audience | Public/other audiences |
Results and Impact | Communication of Science related topics to the wider public. Widespread enthusiasm amongst participants. |
Year(s) Of Engagement Activity | 2013 |
Description | Innovate UK: Manufacturing ATMPs - moving from approval to commercial success, London |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Industry/Business |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2018 |
Description | Invitation to Lecture at the Institut de Recherches Cliniques de Montreal |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | This conference is an institute-wide conference where a significant number of PI and students of the institute were present. Our work on gene therapy was of particular interest to the institute researchers. |
Year(s) Of Engagement Activity | 2017 |
Description | King's College London DGCST Postgraduate Research Symposium - Keynote speaker |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2018 |
Description | Media Interview at Bioanalysis Zone conference in Barcelona |
Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Media (as a channel to the public) |
Results and Impact | Not sure Steve to complete |
Year(s) Of Engagement Activity | 2018 |
Description | Meet the Expert - ESGCT Spring School |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Postgraduate students |
Results and Impact | Students had the opportunity to talk with Experts in their field |
Year(s) Of Engagement Activity | 2018 |
Description | Meet the Experts- ESGCT Spring School |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Postgraduate students |
Results and Impact | Students had the opportunity to talk with experts in their field of science. |
Year(s) Of Engagement Activity | 2018 |
Description | Navigating The Future Of Healthcare - Academy Of Medical Sciences - 2016/07/08 |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Postgraduate students |
Results and Impact | Science education workshop. Encouraging career participation and development especially for women scientist. |
Year(s) Of Engagement Activity | 2016 |
URL | https://acmedsci.ac.uk/more/events/navigating-the-future-of-healthcare |
Description | Open day for Flutterby Fundraisers for CF charity. |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | It was an open day for Flutterby fundraisers for CF to increase awareness of Cystic Fibrosis that attracted approx 140 members of the public asked questions . |
Year(s) Of Engagement Activity | 2017 |
Description | Oxford University Presentation 2014 |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Professional Practitioners |
Results and Impact | Presentation of research activities with local (Oxford University) researchers to share knowledge and promote collaboraiton |
Year(s) Of Engagement Activity | 2014 |
Description | Phacilitate Navigating the path to commercialisation of ATMP's in Europe: funding, pricing & reimbursement & patient access, London |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Industry/Business |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2018 |
Description | Pirbright Research Institute - Lung Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | Presentation Karolinska Institute 2015 |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Dissemination of scientific findings in a general CF gene therapy context to range of practitioners |
Year(s) Of Engagement Activity | 2015 |
Description | Presentation to EIT Health Innovation Summit: Enhancement of lenitivral vector production through alteration of virus-cell interactions |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Industry/Business |
Results and Impact | Presentation by student/new fellow (Dr JF Gelinas) to EIT Health Innovation Summit: Enhancement of lenitivral vector production through alteration of virus-cell interactions. Approx 350 participants from Research Institutions, Universities and Govt bodies attended the Summit. The presentation was on Lentiviral vector production and the enhancements made which is a project funded by EIT Health Innovations. |
Year(s) Of Engagement Activity | 2016 |
Description | Presentation to Parents of Children with CF (RHSC Edinburgh) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Patients, carers and/or patient groups |
Results and Impact | Chris Boyd and a member of his lab team gave presentations on the clinical trial work and ongoing research being undertaken by the UKCFGT Consortium. Approx 15 parents and carers of children with cystic fibrosis attended the talks and were able to informally ask questions regarding present and future studies that their children may be eligible to take part in. |
Year(s) Of Engagement Activity | 2015 |
Description | Presentation to Probus group |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Public/other audiences |
Results and Impact | Chris Boyd was invited to present his work 'Gene Therapy and Cystic Fibrosis' to the Lenzie Probus Club. This general audience have a regular programme of talks on a variety of subjects. |
Year(s) Of Engagement Activity | 2018 |
Description | Presentation to University College (Wadham College Oxford) on Gene Therapy for Cystic Fibrosis |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Undergraduate students |
Results and Impact | Presentation to University College (Wadham College Oxford) on Gene Therapy for Cystic Fibrosis. 30 under graduate students attended a presentation on Gene Therapy for Cystic Fibrosis: Finally Turning the Hype into Clinical Progess. This sparked questions and discussion afterwards and gave students more awareness of Gene Therapy and how it can help sufferers of Cystic Fibrosis |
Year(s) Of Engagement Activity | 2016 |
Description | Quintiles - Success and challenges in CF gene therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Industry/Business |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | RSM Cystic Fibrosis Symposium |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Extensive interest in world 1st non-viral trial of Gene Therapy aimed at Clinical improvement. Discussions underway with Pharma for follow on study. |
Year(s) Of Engagement Activity | 2015 |
Description | Rare Disease Awareness Day |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | Yes |
Type Of Presentation | Workshop Facilitator |
Geographic Reach | Regional |
Primary Audience | Schools |
Results and Impact | Highlighting the awareness of CF and GT to the wider public. Stimulated enthusiasm for Science and Medical topics to the wider public. |
Year(s) Of Engagement Activity | 2011,2012,2013 |
Description | Royal Society of Medicine 9th Annual Charles Darwin Lecture |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighting the awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2017 |
Description | Science in Health |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | Highlighting the awareness of CF and GT to the wider public Stimulated enthusiasm around the Gene Therapy trial. |
Year(s) Of Engagement Activity | 2014 |
Description | Sixth Form Talk Oxford 2014 |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | Presentation of science research to sixth formers and GCSE students at local school, also discussed careers in science. |
Year(s) Of Engagement Activity | 2014 |
Description | Studium Invitation to Lecture at Winchester College |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Undergraduate students |
Results and Impact | Highlighting the awareness of CF and GT to the wider public. |
Year(s) Of Engagement Activity | 2017 |
Description | TRT World - News Report - 2017/03/03 |
Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | TV interview discussing gene therapy, lentiviral vectors and genetic disease |
Year(s) Of Engagement Activity | 2017 |
URL | http://www.trtworld.com |
Description | The Institute of Genetic Medicine Seminar - Lung Gene Therapy |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | Highlighted awareness of CF and GT to the wider public |
Year(s) Of Engagement Activity | 2015 |
Description | Where next for Gene Therapy of Cystic Fibrosis lung disease at UCL GOSICH - BDRC Seminar |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Professional Practitioners |
Results and Impact | 30-40 Professional Scientists attended my presentation on where next for Gene Therapy of Cystic Fibrosis Lung Disease, which sparked questions and discussion afterwards |
Year(s) Of Engagement Activity | 2018 |