MICA: The role of utrophin in DMD and its therapeutic potential
Lead Research Organisation:
University of Oxford
Department Name: Physiology Anatomy and Genetics
Abstract
Duchenne muscular dystrophy (DMD) is a devastating progressive muscle wasting disease caused by the absence of a large protein, dystrophin, in all muscle cells of patients. Generally, only boys are affected, and girls are carriers of the disease because the gene is located on the X chromosome. (Boys have only one X chromosome whereas girls have two, and therefore have a normal X chromosome to compensate). Patients are typically wheelchair bound by the age of 12 and die from respiratory failure or cardiomyopathy in their twenties. More than 65% of DMD patients have portions of the gene missing. Normally, dystrophin associates with other proteins at the muscle membrane to form the dystrophin-associated protein complex (DAPC). In the absence of dystrophin, the DAPC fails to form, and the muscle membrane becomes more susceptible to contraction-induced injury. As a consequence of this, muscle fibres die and are replaced by fibre-like tissue.
There is currently no effective treatment for DMD, and because of its frequency in all populations, there is a real unmet clinical need. It is estimated that in the developed territories of the world, there are at least 50,000 DMD patients.
Pharmacological treatments are being developed, and although many of them will slow the progression of the disease, there may be significant long term side effects. Genetic approaches target the mutation directly and are showing very promising progress. Examples include viral delivery of dystrophin, exon skipping and termination codon read-through. These treatments are in clinical trials but challenges remain in efficacy, delivery to all muscles including the heart,and the latter two approaches are mutation-dependent. For example, exon-51 skipping targets 13% of patients and stop codon read-through only 12% of boys with DMD. We discovered some years ago through research funded by MRC that there is another protein, utrophin, which is normally present at low levels in adult muscle. We demonstrated that if utrophin levels are increased in the mdx mouse model of the disease, the muscle pathology is improved. Our strategy for DMD therapy is therefore to modulate the expression of this dystrophin-related protein, utrophin, using small chemical molecules. This approach is applicable to all patients because it is not mutation-dependent. Furthermore, an orally-administered drug can potentially target all affected muscle types, including heart and diaphragm.
We are now at an exciting stage where we have proof of principle of the utrophin modulation approach. We have several chemical series which increase utrophin levels from our high through-put screens. However, these drugs need to be optimised and their effects characterised in detail in the mdx mouse. The programme requires close collaboration with chemists, molecular biologists and Summit Therapeutics. Summit Therapeutics are currently performing Phase 1b trials in patients with our first candidate drug, SMT C1100. However, we now need to identify and develop follow-up compounds to improve on current drug effectiveness and we need to understand more about this therapy works.
This work will be part of the UtroDMD Alliance of which MRC is a member and which includes the Muscular Dystrophy Association USA and Muscular Dystrophy UK. This Alliance of funders allows us to work seamlessly with patients groups and Summit Therapeutics to deliver these molecules to the clinic.
There is currently no effective treatment for DMD, and because of its frequency in all populations, there is a real unmet clinical need. It is estimated that in the developed territories of the world, there are at least 50,000 DMD patients.
Pharmacological treatments are being developed, and although many of them will slow the progression of the disease, there may be significant long term side effects. Genetic approaches target the mutation directly and are showing very promising progress. Examples include viral delivery of dystrophin, exon skipping and termination codon read-through. These treatments are in clinical trials but challenges remain in efficacy, delivery to all muscles including the heart,and the latter two approaches are mutation-dependent. For example, exon-51 skipping targets 13% of patients and stop codon read-through only 12% of boys with DMD. We discovered some years ago through research funded by MRC that there is another protein, utrophin, which is normally present at low levels in adult muscle. We demonstrated that if utrophin levels are increased in the mdx mouse model of the disease, the muscle pathology is improved. Our strategy for DMD therapy is therefore to modulate the expression of this dystrophin-related protein, utrophin, using small chemical molecules. This approach is applicable to all patients because it is not mutation-dependent. Furthermore, an orally-administered drug can potentially target all affected muscle types, including heart and diaphragm.
We are now at an exciting stage where we have proof of principle of the utrophin modulation approach. We have several chemical series which increase utrophin levels from our high through-put screens. However, these drugs need to be optimised and their effects characterised in detail in the mdx mouse. The programme requires close collaboration with chemists, molecular biologists and Summit Therapeutics. Summit Therapeutics are currently performing Phase 1b trials in patients with our first candidate drug, SMT C1100. However, we now need to identify and develop follow-up compounds to improve on current drug effectiveness and we need to understand more about this therapy works.
This work will be part of the UtroDMD Alliance of which MRC is a member and which includes the Muscular Dystrophy Association USA and Muscular Dystrophy UK. This Alliance of funders allows us to work seamlessly with patients groups and Summit Therapeutics to deliver these molecules to the clinic.
Technical Summary
Duchenne Muscular Dystrophy (DMD) is an X-linked recessive muscle wasting disorder. Patients are wheelchair-bound by the age of 12 and die from respiratory failure or cardiomyopathy in their twenties. DMD is caused by the absence of the cytoskeletal protein dystrophin at the sarcolemma which makes it more susceptible to contraction-induced injury.
There is currently no effective treatment for DMD. The strategy we are taking is to modulate the expression of the dystrophin related protein utrophin which can compensate for the missing dystrophin. This approach is applicable to all patients whatever their mutation.
We will identify lead candidate small molecules for the modulation of utrophin expression by high through-put screening of dystrophin deficient immortalised mouse muscle cell lines. Our initial work has led to a first-in-class agent, SMT C1100, for the pharmacological modulation of utrophin which is entering a Phase Ib clinical trial. However, we now need to identify and develop best in class compounds to improve on current drug effectiveness. We will optimise any hits from the cell lines using medicinal chemistry and in human DMD cell lines. We will then assess lead candidates in vivo in the mdx mouse model of the disease. Promising candidate molecules will be assessed for their effect on the muscle pathology using histopatholgical and molecular analysis, assays of eccentric contraction in EDL muscle and MRI studies of the heart. We will analyse biomarkers for the disease such as miRNAs, FN1 and MMP9. We will also determine the mechanism of action of drug candidates which may allow the development of other molecular targets. Our aim is to have generated lead drug candidates that by the end of the five years of this basic preclinical work can be taken into the clinic by Summit Therapeutics.
There is currently no effective treatment for DMD. The strategy we are taking is to modulate the expression of the dystrophin related protein utrophin which can compensate for the missing dystrophin. This approach is applicable to all patients whatever their mutation.
We will identify lead candidate small molecules for the modulation of utrophin expression by high through-put screening of dystrophin deficient immortalised mouse muscle cell lines. Our initial work has led to a first-in-class agent, SMT C1100, for the pharmacological modulation of utrophin which is entering a Phase Ib clinical trial. However, we now need to identify and develop best in class compounds to improve on current drug effectiveness. We will optimise any hits from the cell lines using medicinal chemistry and in human DMD cell lines. We will then assess lead candidates in vivo in the mdx mouse model of the disease. Promising candidate molecules will be assessed for their effect on the muscle pathology using histopatholgical and molecular analysis, assays of eccentric contraction in EDL muscle and MRI studies of the heart. We will analyse biomarkers for the disease such as miRNAs, FN1 and MMP9. We will also determine the mechanism of action of drug candidates which may allow the development of other molecular targets. Our aim is to have generated lead drug candidates that by the end of the five years of this basic preclinical work can be taken into the clinic by Summit Therapeutics.
Planned Impact
The aim of this programme is to develop new drugs which modulate the expression of utrophin for the therapy of Duchenne muscular dystrophy (DMD). This is important because there is currently no effective treatment for DMD. In view of the fact that this disease is one of the most common recessive disorders in all populations, there is a real unmet clinical need. (It is estimated that in the developed territories of the world, there are 50,000 patients). One such drug which we have developed, SMT C1100, is already in Phase 1 trial, and it is our endeavour to progress additional drugs, potentially with better attributes such as potency or more favourable drug like properties, to move into clinical development by the end of the funding period of the grant in five years' time. This treatment has the potential to improve the quality of life of many DMD patients worldwide since it is applicable to all DMD patients regardless of their dystrophin mutation.
The treatment will be more effective in young patients, therefore the development of new-born screening for DMD could be of major importance. The PI is working with Muscular Dystrophy UK to lobby policy makers to help ensure that this could be introduced once an effective therapy becomes available.
Part of this work is funded in partnership with Summit Therapeutics and Muscular Dystrophy UK, Muscular Dystrophy Association USA, covered by an Alliance agreement set up through Isis Innovation and Research Services at the University of Oxford. This arrangement ensures that all on-going research activities are coordinated for maximum productivity and that patients and patient organisations are kept informed of developments. The development of new drug candidates and further clinical trials emanating from the UtroDMD Alliance may have significant impact on the lives of DMD patients. The programme is MRC-led and would not be possible without this Alliance of funders as it depends on a collaboration of expertise from gene to clinical trial.
The intellectual property arrangements are handled by Isis Innovation at the University. There is currently an exclusive arrangement of commercial exploitation through Summit Therapeutics (see MICA form). Summit Therapeutics is a UK-based biotechnology company, and this work would therefore benefit the UK economy in the longer term.
The staff working on the grant will be trained not only in the molecular techniques associated with the research programme but also in the rigour of reporting required for the development of a new drug. This will lead to employment prospects not only in academia but also in the biotechnology and large pharma sector.
The treatment will be more effective in young patients, therefore the development of new-born screening for DMD could be of major importance. The PI is working with Muscular Dystrophy UK to lobby policy makers to help ensure that this could be introduced once an effective therapy becomes available.
Part of this work is funded in partnership with Summit Therapeutics and Muscular Dystrophy UK, Muscular Dystrophy Association USA, covered by an Alliance agreement set up through Isis Innovation and Research Services at the University of Oxford. This arrangement ensures that all on-going research activities are coordinated for maximum productivity and that patients and patient organisations are kept informed of developments. The development of new drug candidates and further clinical trials emanating from the UtroDMD Alliance may have significant impact on the lives of DMD patients. The programme is MRC-led and would not be possible without this Alliance of funders as it depends on a collaboration of expertise from gene to clinical trial.
The intellectual property arrangements are handled by Isis Innovation at the University. There is currently an exclusive arrangement of commercial exploitation through Summit Therapeutics (see MICA form). Summit Therapeutics is a UK-based biotechnology company, and this work would therefore benefit the UK economy in the longer term.
The staff working on the grant will be trained not only in the molecular techniques associated with the research programme but also in the rigour of reporting required for the development of a new drug. This will lead to employment prospects not only in academia but also in the biotechnology and large pharma sector.
Publications
Chatzopoulou M
(2020)
Decreasing HepG2 Cytotoxicity by Lowering the Lipophilicity of Benzo[d]oxazolephosphinate Ester Utrophin Modulators.
in ACS medicinal chemistry letters
Davies KE
(2016)
2015 William Allan Award.
in American journal of human genetics
Wilkinson IVL
(2020)
Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid.
in Angewandte Chemie (International ed. in English)
Davies K
(2020)
The Long Journey from Diagnosis to Therapy
in Annual Review of Genomics and Human Genetics
Babbs A
(2020)
From diagnosis to therapy in Duchenne muscular dystrophy.
in Biochemical Society transactions
Chatzopoulou M
(2022)
Structure-activity relationships of 2-pyrimidinecarbohydrazides as utrophin modulators for the potential treatment of Duchenne muscular dystrophy.
in Bioorganic & medicinal chemistry
Muntoni F
(2019)
A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet.
in Clinical pharmacology in drug development
Guiraud S
(2017)
Pharmacological advances for treatment in Duchenne muscular dystrophy.
in Current opinion in pharmacology
Himic V
(2021)
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy.
in European journal of human genetics : EJHG
Perkins KJ
(2018)
Alternative utrophin mRNAs contribute to phenotypic differences between dystrophin-deficient mice and Duchenne muscular dystrophy.
in FEBS letters
Description | Accelerated optimisation of next generation utrophin modulators in readiness for clinical trials |
Amount | £163,887 (GBP) |
Organisation | Duchenne UK |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 08/2016 |
End | 08/2018 |
Description | Development of utrophin modulators for DMD |
Amount | £4,300,000 (GBP) |
Organisation | Summit Plc |
Sector | Private |
Country | United Kingdom |
Start | 11/2013 |
End | 11/2019 |
Description | The role of utrophin in DMD and its therapeutic potential |
Amount | £2,018,279 (GBP) |
Funding ID | MR/N010698/1 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 03/2016 |
End | 03/2021 |
Description | Utrophin modulation for the therapy of Duchenne muscular dystrophy (DMD) |
Amount | £196,593 (GBP) |
Funding ID | RA3/3073 |
Organisation | Muscular Dystrophy Association |
Sector | Charity/Non Profit |
Country | United States |
Start | 03/2016 |
End | 03/2019 |
Description | Medicinal chemistry |
Organisation | University of Oxford |
Department | Department of Chemistry |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | My team screened the compounds in cell lines and then any positive hits in the mdx mouse. |
Collaborator Contribution | They designed and synthesised the chemical compounds |
Impact | Clinical trial with Summit Therapeutics in 2018 |
Start Year | 2006 |
Description | Utrophin modulation for the therapy of DMD |
Organisation | Summit Plc |
Country | United Kingdom |
Sector | Private |
PI Contribution | We have been performing the preclinical testing of drugs for the modulation of utrophin for the therapy of DMD. Recently one of the drugs has shown signs of success in Phase 2 clinical trials. |
Collaborator Contribution | The partners have given advice on biology and chemistry and also covered the costs of out sourcing for toxicity studies and preclinical mouse trials. They have also funded biomarker studies. This collaborative agreement was renewed until 2019 as a result of MRC funding. |
Impact | This has resulted in a partnership deal with the University of Oxford to carry our preclinical work to the clinic. Phase 1 trials of one of our drugs has shown it to be safe and well tolerated. Phase 2 trials have just reported intial positive data which are very encouraging. |
Start Year | 2013 |
Description | Action Duchenne Day with Staff and Parents with boys with DMD |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | 15 people attended the lab with talks and a tour |
Year(s) Of Engagement Activity | 2017 |
Description | Attendance for the IF Oxford Science and Ideas Festival, Explorazone: Adults Only |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Public/other audiences |
Results and Impact | Attendance for the IF Oxford Science and Ideas Festival, Explorazone: Adults Only. Oxford Town Hall, Oxford |
Year(s) Of Engagement Activity | 2018 |
Description | Biochemistry Society Centenary Award Lecture |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Public/other audiences |
Results and Impact | "From diagnosis to therapy in Duchenne muscular dystrophy" - Biochemistry Society Centenary Award Lecture - 10th June (Webinar) |
Year(s) Of Engagement Activity | 2020 |
Description | Croonian Medal and Lecture |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Other audiences |
Results and Impact | Croonian Medal and Lecture, Royal Society of London |
Year(s) Of Engagement Activity | 2019 |
Description | Facebook Questions and Answers "on the development of utrophin upregulation therapies for Duchenne" for the Muscular Dystrophy Campaign Charity |
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 | I did a question and answer session on facebook for an hour on utrophin modulation therapy for therapy of DMD |
Year(s) Of Engagement Activity | 2016 |
Description | First Women in Science Workshop |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Public/other audiences |
Results and Impact | First Women in Science Workshop, Sidra Medicine, Doha, Qatar |
Year(s) Of Engagement Activity | 2018 |
Description | Interview with Frontline Genomics |
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 | Postgraduate students |
Results and Impact | The interview was about my life in science and my career. I described my achievements and challenges. |
Year(s) Of Engagement Activity | 2021 |
Description | Interview with Kevin Davies for Human Germline Genome Editing |
Form Of Engagement Activity | A magazine, newsletter or online publication |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Public/other audiences |
Results and Impact | Interview with Kevin Davies for Human Germline Genome Editing |
Year(s) Of Engagement Activity | 2020 |
Description | Interview with Voice, Balliol College, Oxford. Advice to Young People |
Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Undergraduate students |
Results and Impact | x |
Year(s) Of Engagement Activity | 2016 |
Description | Keynote Speaker for Duchenne Muscular Dystrophy Awareness Day |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | An overview of Duchenne Muscular Dystrophy Talk for an Awareness Day, Lodnon |
Year(s) Of Engagement Activity | 2017 |
Description | Lecture for the Croonian Medal |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Media (as a channel to the public) |
Results and Impact | "From diagnosis to therapy in Duchenne muscular dystrophy" - Croonian Lecture - Royal Society - 10th April. The was a special lecture about the development of therapy for Duchenne muscular dystrophy. The talk was aimed at the educated layperson and was broadcast on U-tube. |
Year(s) Of Engagement Activity | 2019,2020 |
URL | http://royalsociety.org/.../2019/04/croonian-lecture |
Description | Lecture, Doha, Qatar |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | "Genetic approaches to therapy for Duchenne Muscular Dystrophy" Sidra Medicine Lecture in Human Genetics, Functional Genomics 2018, Big Data to Clinic - QNCC, Doha, Qatar I gave the opening talk to a Functional Genomics Symposium |
Year(s) Of Engagement Activity | 2018,2019 |
Description | MD UK and parents Day at the lab |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Public/other audiences |
Results and Impact | 15 people attended my laboratory with talks and a tour |
Year(s) Of Engagement Activity | 2017 |
Description | Opened Avonwood Primary School (with a Science Lab), Bournemouth |
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 | x |
Year(s) Of Engagement Activity | 2016 |
Description | Oxford Medical Lecture Club Talk |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Professional Practitioners |
Results and Impact | "Genetic approaches to therapy for Duchenne muscular dystrophy" - Oxford Medical Lecture Club- Oxford Medical Alumni - Osler House, John Radcliffe Hospital - 29th April. Update to senior medical doctors on the progress of therapy for DMD. |
Year(s) Of Engagement Activity | 2019 |
Description | Oxfordshire Science Festival Talk |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | "Muscular dystrophy in the new genomic era" Talk at the Oxfordshire Science Festival held at the Museum of Natural History |
Year(s) Of Engagement Activity | 2016 |
Description | Parental Organization DMD Serbia Workshop |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Supporters |
Results and Impact | "Genetic approaches to therapy of Duchenne muscular dystrophy" Talk, Parental Organization DMD Serbia, Belgrade |
Year(s) Of Engagement Activity | 2018 |
Description | Radio Oxford and Local TV Interview on DMD |
Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Public/other audiences |
Results and Impact | Interview |
Year(s) Of Engagement Activity | 2016 |
Description | Research Strategy Workshop |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | Key Discussants on two topics at a Research Strategy Workshop for Muscular Dystrophy UK in London |
Year(s) Of Engagement Activity | 2017 |
Description | Rosetrees Trust Symposium, London |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | Talk at a Symposium, London |
Year(s) Of Engagement Activity | 2017 |
Description | Speech |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Supporters |
Results and Impact | Speech for the Foodbuy Charity Fundraising Dinner in aid of the Duchenne UK and Chasing Connor's Cure |
Year(s) Of Engagement Activity | 2018 |
Description | Talk at MIT Biology Colloquium, Cambridge, MA (part of the Vallee Foundation Award) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Postgraduate students |
Results and Impact | "Utrophin modulation for therapy of Duchenne muscular dystrophy" - MIT Biology Colloquium, Cambridge, MA - 17th September (part of the Vallee Foundation Award) This was a public lecture delivered to a mixed audience of patients, students and researchers. |
Year(s) Of Engagement Activity | 2019 |
Description | Talk at Sarepta Biosciences, Cambridge, MA (part of the Vallee Foundation Award) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Industry/Business |
Results and Impact | "Utrophin modulation for therapy of Duchenne muscular dystrophy" - Sarepta Biosciences, Cambridge, MA - 17th September (part of the Vallee Foundation Award). Talk to our collaborators on the progress we have made in DMD therapy. This biotechnology company has funded in the past through supporting Summit Therapeutics. |
Year(s) Of Engagement Activity | 2019 |
Description | Talk at the Case Western University, Cleveland, USA (part of the Vallee Foundation Award) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Postgraduate students |
Results and Impact | "Utrophin modulation for therapy of Duchenne muscular dystrophy" - Case Western Reserve University, Cleveland - 11th September (part of the Vallee Foundation Award) This was a research seminar for research students and faculty. |
Year(s) Of Engagement Activity | 2019 |
Description | Talk at the WIMM, Oxford |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Postgraduate students |
Results and Impact | Talk "The trials and tribulations of a career in molecular medicine" - WIMM Postdoc Association with an Evening with Professor Dame Kay E Davies - about lift, the Universe and everything else I would like to say to young scientists. WIMM, Oxford. |
Year(s) Of Engagement Activity | 2018,2019 |
Description | Talk for Duke University, Durham, North Carolina (part of the Vallee Foundation Award) |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Postgraduate students |
Results and Impact | "Utrophin modulation for therapy of Duchenne muscular dystrophy" - Duke University, Durham, North Carolina - 24th September (part of the Vallee Foundation Award) This was a research talk to students. |
Year(s) Of Engagement Activity | 2019 |
Description | Talk for Solid Biosciences , Cambridge, MA |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Industry/Business |
Results and Impact | "Utrophin modulation for therapy of Duchenne muscular dystrophy" - Solid, Cambridge, MA - 19th September This was a talk to a biotech company with whom we have collaborated in the past. |
Year(s) Of Engagement Activity | 2019 |
Description | Talk for families/carers at the Vice-President's lunch at the House of Lords for the Muscular Dystrophy UK |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Supporters |
Results and Impact | "Strategic plans for DMD" for families/carers. Vice-President's lunch at the House of Lords, London for the Muscular Dystrophy UK - 21st November. I presented the main overview of how research is feeding into the clinical trial landscape for muscular dystrophy sufferers. |
Year(s) Of Engagement Activity | 2019,2020 |
Description | Talk for the Oxford Medical School Gazette 70th Anniversary, Worcester College, Oxford |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Postgraduate students |
Results and Impact | "Utrophin modulation in the therapy of Duchenne muscular dystrophy" - Oxford Medical School Gazette 70th Anniversary, Worcester College, Oxford - 5th November. I gave a talk to mdeical students about the importance of translation research and how genetics will change their ability to improve health care in the future. |
Year(s) Of Engagement Activity | 2019,2020 |
Description | Talk, Action Duchenne Conference |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Supporters |
Results and Impact | Talk "Small molecule utrophin modulators for the therapy of Duchenne muscular dystrophy" - Action Duchenne, Hilton Birmingham Metropole Hotel, Birmingham |
Year(s) Of Engagement Activity | 2018 |
Description | Talk, MRC DTP Symposium, Oxford |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Postgraduate students |
Results and Impact | Talk "Genetic approaches to therapy of muscular dystrophy" MRC DTP Symposium, Oxford Talk to researchers in Oxford on therapy for DMD |
Year(s) Of Engagement Activity | 2018,2019 |
Description | The 15th Action Duchenne International Conference, Birmingham |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Media (as a channel to the public) |
Results and Impact | Talk at a Conference |
Year(s) Of Engagement Activity | 2017 |