Novel Therapies 2019 Funding Call

CLOSED. This funding call makes available up to £342k (£171k per year for up to 2 years) to support several projects that align best with Novel Therapies' strategy.

Internal GOSH and ICH applicants only.

Why is it important?

In this theme we will build our capacity to offer clinical trial options to children affected by rare and complex conditions who have limited treatment options. The NIHR Great Ormond Street Hospital Biomedical Research Centre Novel Therapies theme is offering this funding as part of its commitment to better understand disease progression and respond to treatments more effectively.

Who can apply?

Only one application per PI will be accepted. Co-applicants from different disciplines are encouraged and collaborative applications from two different fields where a more senior applicant can provide mentorship to the more junior applicant will be given preference. Funding will be awarded to meet the costs outlined in the application only. All projects will be considered, but priority will be given to those which most closely align with the Novel Therapies Theme’s strategy, and which could clearly lead to patient benefit within the NHS, or which are most likely to lead to either external funding being leveraged. Preclinical studies involving animal models are not eligible studies for any NIHR funding.

How much money is available?

Novel Therapies has £342,000 in funding to be split across a number of awarded applications. This total is split into 2 years and whilst applicants can apply for the full funding, it is highly unlikely a single project will receive the entire £342,000 budget as we hope to support 2-4 projects. Successful applicants will need to ensure they can spend their Y1 allocation of funds by 31st March 2020 and Y2's allocation by 31st March 2021.

Where possible, ideas should centre on the following priorities from the Novel Therapies' strategy:

- Improving deep phenotyping and applying state-of-the-art biomarker stratification;

- Exploit advances in understanding pathogenesis and molecular basis of rare diseases to identify novel therapeutic targets for diseases currently not accessible to experimental therapies;

- Building on our extensive experience in innovative trial design, and by developing the capacity for complex trial interventions;

- Increase the opportunities to recruit children with rare diseases into clinical trials and systematically collect and assess the value of longitudinal biological samples;

- To increase industry collaboration on discovery and validation pipeline by establishing novel rare disease specific cellular models.

Application process:

Applications open: 01 October 2018

Applications deadline: 03 December 2018

Award letters: Mid-January 2019

Funding available: 01 April 2019

Applicants must complete both forms below. All applicants must update (if they have not done so) their cohorts catalogue data for their application to be considered. A copy of the cohorts catalogue will be sent to applicants to populate when they send their application.

Novel Therapies Funding Call 2019 Application Form (83.46 KB)

Registration form (147.59 KB)

Contact details: Submit applications to: and completed Project Registration forms to

For any general queries regarding your application please contact the Marta Zancolli ( If PIs have any questions regarding the eligibility of their application please feel free to contact Francesco Muntoni ( or Paul Gissen (

Previous awards

In November 2017 we announced our first funding call and are pleased to announce the successful applicants. After careful consideration, the panel selected to fund two projects:

Principal Investigator Project Title Award
Dr Hannah Mitchison Novel genetic therapies for motile cilia disease £102,000
Dr Veronica Kinsler Gene therapy for congenital melanocytic naevi £107,000
Professor Chris Clark Fractional diffusion imaging as a biomarker for pathology in Duchenne Muscular Dystrophy £86,000
Professor Francesco Muntoni Developing microRNAs as biomarkers and therapeutic targets in children with spinal muscular atroph £132,170