Assessing the permeability of Bruch's membrane to complement proteins
Research type
Research Study
Full title
Study of the permeability of post mortem Bruch's membrane to complement proteins and its use in the study of age-related macular degeneration
IRAS ID
289704
Contact name
Katharina Lo
Contact email
Sponsor organisation
Gyroscope Therapeutics Ltd
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
Age related macular degeneration (AMD) is the commonest cause of blindness in the UK with no known treatment for the underlying disease and no treatment for the common and blinding dry form. It is a disease characterised by death of light sensitive cells at the back of the eye, leading to permanent vision loss. AMD is strongly linked to overactivity in the complement system, a part of the immune system. Gyroscope Therapeutics has developed a sustained method of gene therapy treatment delivered through a harmless virus that enters the tissue at the back of the eye and designed to control the overactive complement system. Currently, the gene therapy has been evaluated in single layers of laboratory grown eye cells but this does not represent the true complexity of the eye. There is no animal model of AMD meaning that therapy cannot be fully evaluated and best dose and way of giving it cannot determined until given in humans. Within this study, we are proposing to produce a better model of AMD using post mortem eye tissue. To do this we will remove all the cells from left over donated eye tissue after corneal transplantation and then recellularise it using two types of human stem cell derived eye cells on either side of a key part of the back of the eye called Bruchs membrane. In some instances we may use BrM without any additional cells. Once we have developed this model, we will utilise it to understand how the drugs cross the structures at the back of the eye. This will mean that we can focus development on those drugs that are moot likely to provide effective treatment.
REC name
North East - York Research Ethics Committee
REC reference
21/NE/0068
Date of REC Opinion
12 Mar 2021
REC opinion
Favourable Opinion