Studying Stem Cell Gene Therapy for NOD2-associated Crohn's Disease 1
Research type
Research Study
Full title
Evaluating the Application of Haematopoietic Stem-Cell Gene Therapy as a Novel Therapeutic for Crohn’s Disease patients with Loss-of Function NOD2 Polymorphisms
IRAS ID
312412
Contact name
Pervinder Sagoo
Contact email
Sponsor organisation
Orchard Therapeutics
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Approximately 20-40% of Crohn's Disease patients are known to have mutations within the NOD2 gene. Normal working copies of the NOD2 gene are needed by specialised white blood cells, to detect and alert the body to bacterial infections, including gastrointestinal infections. Several research studies now suggest that some NOD2 mutations are related to the severity of Crohn’s Disease patients may experience, and that this may also limit a patient’s ability to respond to available therapies that help alleviate symptoms of Crohn’s Disease. Our current knowledge and research suggests that mutations in the NOD2 gene, may prevent the immune system from detecting and clearing bacterial infections that occur in the gut, and that this may lead to the development of Crohn’s disease. Importantly, we have shown that by introducing a normal copy of the NOD2 gene using stem cell gene therapy, we can rescue the ability of immune cells to detect and respond to bacteria. These data therefore suggest that stem cell gene therapy may provide a new treatment for Crohn’s Disease patients with some NOD2 mutations.
To understand the potential of this new therapy, we will study the ability of blood immune cells collected from blood samples (100mLs) from healthy donors and Crohn’s Disease patients to detect and respond to bacterial proteins, and test whether gene therapy can restore this activity. This research, conducted within our laboratory in King's College London, aims to understand the role of NOD2 in different white blood cells, and its relationship with Crohn’s Disease colitis. This research will allow us to generate meaningful results to understand the potential application of stem cell gene therapy to directly benefit patients with NOD2-associated Crohn’s Disease (www.orchard-tx.com).REC name
South Central - Berkshire B Research Ethics Committee
REC reference
22/SC/0070
Date of REC Opinion
17 Mar 2022
REC opinion
Further Information Favourable Opinion