Translational Research Office (TRO)


The UCL Therapeutic Acceleration Support (TAS) Fund - Rare Diseases Call result announcement

27 April 2023

The UCL Therapeutic Acceleration Support (TAS) Fund Rare Diseases Call Results

We are pleased to announce the successful awarded projects from the recent UCL Therapeutic Acceleration Support (TAS) Fund Rare Diseases Call supported by a £500K LifeArc Pathfinder award

The quality of the 19 applications was extremely high which made the panel decision very difficult, resulting in an extra £281K being used from the MRC IAA, GOSH and UCLH BRC’s to support a total of 10 projects. 

Each project awardee will have access to £60 to £80k to run their projects for up to 12 months. All projects aim to accelerate the transition from discovery science to the early stages of therapeutic, device and diagnostic development allowing them to de-risk their projects and prepare applications for further, larger translational funding schemes.

Applications were assessed based on (1) the unmet medical need and the likely hood of delivering impact to patients, (2) target validation/ prototype component validation, (3) originality, (4) feasibility and traceability, and (5) the potential for further development.

We thank the tremendous effort of the selection panel in driving the vigorous selection process.

Projects awarded:

Principal Investigator



Project Title



Great Ormond Street Institute of Child Health

Cell & Gene Therapies

Accelerating AAV gene therapy for paediatric kidney disease


Great Ormond Street Institute of Child Health


A tricyclo-based antisense oligonucleotide for the treatment of Facioscapulohumeral dystrophy

Giovanni GIOBBE

Great Ormond Street Institute of Child Health

Regenerative Medicine

Extracellular matrix hydrogel for Short Bowel Syndrome treatment


Great Ormond Street Institute of Child Health

Devices & Diagnostics

Development of a specialised, high throughput Targeted Anti-Drug Antibody Immuno Multiplex Assay (TA-DA ImmunoMA) for measuring anti-drug antibodies

Phillipa MILLS

Great Ormond Street Institute of Child Health


siRNA therapy for the treatment of inherited paediatric lysine catabolism disorders

Joanne NG

UCL Queen Square Institute of Neurology

Cell & Gene Therapies

Gene therapy for DNAJC6 juvenile parkinsonism


Medical Physics & Biomedical Engineering

Devices & Diagnostics

Oxygen-enhanced MRI for early detection of chronic lung allograft dysfunction (CLAD) in children following lung transplantation

Shamima RAHMAN

Great Ormond Street Institute of Child Health

Cell & Gene Therapies

Gene therapy for deoxyguanosine kinase deficiency


Great Ormond Street Institute of Child Health

Small Molecules

Drug development for the treatment of manganese overload to alleviate neurological disability

Haiyan ZHOU

Great Ormond Street Institute of Child Health


Preclinical study of antisense oligonucleotide therapy in a humanized mouse model of SPTLC1-related hereditary sensory neuropathy


The selection panel:

  • Dr Jane Kinghorn (Chair) - Director, UCL Translational Research Office
  • Prof Trevor Smart (Vice-Chair) - Professor of Pharmacology, Division of Bioscience & Neuroscience Domain Chair
  • Dr Rick Fagan - Director of BioPharm, UCL Business
  • Prof Geraint Thomas - Professor of Biochemistry, Division of Bioscience
  • Prof Rachel Chambers - Professor of Respiratory Cell and Molecular Biology, Director of the Centre for Inflammation and Tissue Repair (CITR)
  • Prof Thomas Voit - Director of NIHR GOSH UCL Biomedical Research Centre, Vice Dean Enterprise FPHS
  • Prof Julie Daniels - Professor of Regenerative Medicine and Cellular Therapy, BRC Theme lead for Rescue, Repair and Regeneration
  • Prof Ijeoma Uchegbu - Professor of Pharmaceutical Nanoscience, UCL School of Pharmacy
  • Prof Umber Cheema - Professor of Bioengineering, Division of Surgery & Interventional Science & Vice Dean Enterprise FMS
  • Prof Geoff Parker - Professor of Healthcare Engineering, Imaging and Enterprise, Deputy Director UCL Institute of Healthcare Engineering
  • Dr Stephen Hobbiger - Royal Society Entrepreneur in Residence - Drug Development
  • Dr Preeti Bakrania - Senior Principle Scientist, LifeArc
  • Prof Simon Waddington - Professor of Gene Therapy, UCL EGA Institute for Women’s Health

Read more:
TRO endeavours to support our academics drive change for those living with a rare disease

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