Cells to evaluate novel compounds in neuromuscular disorders
Research type
Research Study
Full title
The use of cells to support the evaluation and development of novel compounds to treat neuromuscular disorders
IRAS ID
332769
Contact name
George Reid
Contact email
Sponsor organisation
Tay Therapeutics Ltd.
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
We are making this REC application as we need consent for a laboratory-based study by a commercial entity. We wish to use human myoblasts and fibroblasts that have undergone at least 4 divisions in vitro and are non-relevant under the Human Tissue Act. We will obtain these cells from the MRC Centre for Neuromuscular Disease Biobank, which has a clause saying that samples can only be released to ethically-approved projects for commercial research/applications.\n\nOur aim is to develop compounds that can treat a variety of neuromuscular disorders, including Duchenne muscular dystrophy by restoring critical proteins (e.g. dystrophin) that drive the disease.\n\nWe will use myoblasts and fibroblasts from patients with and without premature stop mutations in disease causing genes in laboratory studies to evaluate the ability of our novel compounds to restore normal levels of protein and to evaluate the safety window of our compounds in vitro.\n\n
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
23/EM/0210
Date of REC Opinion
14 Sep 2023
REC opinion
Further Information Favourable Opinion