Assessment of the properties of splicing kinase inhibitors - Version 1
Research type
Research Study
Full title
In vitro and Ex Vivo Assessment of the permeability, efficacy and mechanism of splicing kinase inhibitors
IRAS ID
266249
Contact name
Kenneth McKechnie
Contact email
Sponsor organisation
Exonate
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Wet age-related macular degeneration (wAMD) and diabetic macular edema (DME) are the most common causes of blindness in the Western world. Both are caused by new and improperly formed blood vessels developing at the back of the eye. These poorly formed blood vessels leak blood and fluid into the surrounding tissues, damaging the cells needed for sight. Currently these diseases are treated by monthly injections into the eye of proteins which bind the main cell factor which causes new blood vessel formation, vascular endothelial growth factor (VEGF). These injections are time consuming for patients and medical staff and blocking all types of VEGF can cause other problems, as some is needed to maintain normal, healthy blood vessels.
Exonate are developing small molecule drugs which instead of blocking VEGF change its production by cells to a form which stops new blood vessel growth and leakage whilst keeping the normal blood vessels healthy. The other advantage of using these small molecules instead of large proteins as treatment is that these can be given as eye drops, instead of injections, saving medical resources and patient discomfort.
This study intends to use human eye tissue and cells as a model to study the effect of our small molecule drug treatments on human cells and tissue, determining how human cells respond to the compounds and how the compounds bind and penetrate the tissue. This background laboratory research will allow us to select compounds which have the most potential to work in clinical testing.REC name
HSC REC A
REC reference
20/NI/0078
Date of REC Opinion
2 Jun 2020
REC opinion
Favourable Opinion