Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
In the first large-scale stem cell clinical trial in epidermolysis bullosa (EB), the “MissionEB” study, CORDStrom was investigated in a double-blinded, randomized, placebo-controlled pivotal trial to evaluate the safety and efficacy of CORDStrom in 30 pediatric patients in the UK with intermediate and severe RDEB using a novel crossover clinical trial design. Topline results showed CORDStrom was easily administered, well tolerated, with no serious adverse effects related to treatment, and there were beneficial effects with respect to itch, pain, disease severity and quality of life measures.
CORDStrom
An investigational cell medicine comprising allogeneic, pooled, human umbilical cord -derived mesenchymal stromal cells (hucMSCs) in suspension for injection or infusion. Our CORDStrom hucMSCs platform of cell medicines leverages proprietary patent-pending formulations and techniques related to donor cell selection, pooling and expansion to offer batch-to-batch consistent, drug-product grade, cGMP, scalable hucMSCs cell medicines designed to treat complex inflammatory diseases. The initial CORDStrom product is agnostic to disease indication; however, the platform offers ability to tune CORDStrom product variants for optimization of anti-inflammatory, immunomodulatory, wound healing, and other characteristics, which can be strategically utilized to design products for indication-specific optimization. CORDStrom is a first of a kind systemic hucMSCs therapy in development for treatment of recessive dystrophic epidermolysis bullosa (RDEB) in pediatric patients. Unlike other MSC products, CORDStrom leverages pooling of hucMSCs from multiple donors to provide a batch-to-batch consistent off-the-shelf therapy. Moreover, CORDStrom, as a systemic therapy, is uniquely positioned to target inflammation and immunomodulatory limitations in RDEB, intended to drive clinically important benefits in itch, pain, disease severity and quality of life.
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