The multicenter, single-blind, randomized placebo-controlled trial involves a single intravenous administration of the gene therapy drug to pediatric patients with progressive Duchenne muscular dystrophy. This Phase 1/2 study aims to evaluate the tolerability, safety, and efficacy of the drug GNR-097.
GNR-097 is a recombinant adeno-associated viral vector of serotype 9, encoding a truncated dystrophin gene.
Daniil Talyansky, CEO of GENERIUM:
"Gene therapy is the youngest and most groundbreaking field in global pharmaceuticals and medical science. The unique expertise of Russian scientists has enabled the development of a recombinant adeno-associated viral vector encoding the microdystrophin gene. We are now initiating clinical trials of this original drug, which holds great promise".
The trial of this innovative drug is international and will be conducted in the Russian Federation and the Republic of Belarus. In Russia, the study will take place at five medical centers: Research Clinical Institute of Pediatrics and Pediatric Surgery named after Academician Yu.E. Veltishchev, the Pirogov Russian National Research Medical University, the St. Petersburg State Pediatric University, the Regional Children's Clinical Hospital in Yekaterinburg, and the National Medical Research Center for Children's Health. In Belarus, the trial will be conducted at the Republican Scientific and Practical Center "Mother and Child."
About Duchenne Muscular Dystrophy (DMD):
Duchenne muscular dystrophy is a severe, progressively debilitating disease that primarily affects boys. It is a genetically inherited disorder caused by a mutation in the DMD gene, leading to irreversible destruction of muscle cells. The disease is characterized by progressive weakness and atrophy of skeletal muscles, resulting in cardiovascular, respiratory, and orthopedic complications. On average, by the age of 11, patients lose the ability to walk independently. The clinical trial design includes studying the drug's application in boys aged 4–9 years with DMD through a single intravenous administration. The trial will assess the drug's tolerability, safety, and efficacy.