The early phase 1 study (NCT07188012) of SPOT-mRNA03 gene therapy for Duchenne Muscular Dystrophy (DMD) developed by Shanghai Siponuoyin Biotechnology is a pilot study of the safety and expression of dystrophin in skeletal muscles after administration.
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What is SPOT-mRNA03 Gene Therapy
SPOT-mRNA03 is the gene therapy product for Duchenne Muscular Dystrophy (DMD). Following administration to DMD patients, the molecular targeting peptide on the membrane of the EVs can direct SPOT-mRNA03 into the patient’s muscle cells, enabling the translation and secretion of endogenous full-length intact dystrophin by dystrophin deficient receptor cells using intracellular ribosomes and amino acids.
SPOT-mRNA03 Gene Therapy Early Phase 1 – NCT07188012
This is a single-arm, open-label, FIH, single-center exploratory clinical research using SPOT-mRNA03 given intravenously to patients with DMD. Full-length dystrophin mRNA is contained in muscle-targeted extracellular vesicles (EVs) called SPOT-mRNA03. Molecular targeting peptides on the EVs membrane give SPOT-mRNA03 its targeting capability, which makes it possible to distribute dystrophin mRNA as a gene therapy product for DMD.
Study Start (Estimated): 2025-08-06
Primary Completion (Estimated): 2026-12
Study Completion (Estimated): 2026-12
Number of Participants (Estimated): 6
Study Type: Early Phase 1
Location: Shanghai Children’s Medical Center
Ages Eligible for Study: 2 Years to 6 Years (Ambulant Males)
Principal inclusion and exclusion criteria. – NCT07188012 –
How to Participate in the SPOT-mRNA03 Clinical Trial
To participate in the SPOT-mRNA03 gene therapy early phase 1 clinical trial, you can use the contact information below.
Name: Winston Town
Phone Number: 212-920-5501
Email: [email protected]
