Characterising potential therapeutic agents of complement diseases v1
Research type
Research Study
Full title
Characterisation of potential therapeutic agents of complement mediated human diseases e.g hemolytic anemias that result in the loss of red blood cells.
IRAS ID
292130
Contact name
Susan Chapple
Contact email
Sponsor organisation
Dianthus Therapeutics, Inc
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
This project aims to screen molecules with potential therapeutic benefits in the treatment of complement mediated diseases such as hemolytic anemias (which result in serious blood disorders) e.g paroxysmal nocturnal hemoglobinuria (PNH), paroxysmal cold hemoglobinuria (PCH) paroxysmal cold hemoglobinuria, cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA).
The complement system is a tightly self-regulated internal surveillance system in the body which plays a critical role in inflammation and the defence against microbes. When errors occur in this system it can result in diseases such as hemolytic anemias. The end result of chronic loss of RBCs in patients with hemolytic anemia means an insufficient number of oxygen carrying RBCs in circulation and if left untreated can be fatal.
Red Blood cells (RBC), Type O Negative, will be obtained from surplus routine blood donations provided by healthy adults to NHS Blood and Transplant (NHSBT). As part of the donation process, blood donors are asked to consent to the possibility that part, or all, of their donation may be used for research. RBCs may also be obtained from commercial blood sources where the supplier has obtained similar consent from donors.
IONTAS will use the RBCs where permission has been granted in two assays, RBC lysis assay and detection of stained RBCs by Flow cytometry (which is a technique that allows screening of cell surface molecues). The RBC lysis assay will characterise potential inhibitor molecules for their ability to prevent lysis of the red blood cells. The Flow cytometry assay will screen for expression of a ‘marker’ (C3b) on the RBCs and characterise binding of inhibitor molecules that effectively reduce the expression of the ‘marker’.
All blood samples used for the project will be anonymized. All samples will be destroyed following use.REC name
South Central - Berkshire Research Ethics Committee
REC reference
20/SC/0453
Date of REC Opinion
7 Dec 2020
REC opinion
Favourable Opinion