[Press released] Rznomics selected for ‘National new drug development project for rare genetic diseases’

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Rznomics announced on the 16th that a candidate substance as an innovative new drug for Rett Syndrome under development was selected for national drug development project by the National New Drug Development Foundation.

With this selection, Rznomics will receive a national research grant for 24 months for research on lead material derivation and verification.

Rett syndrome is a hereditary neurodevelopmental disorder with an incidence of 1 in 10,000 to 15,000. To date, there is no approved treatment.

For this reason, patients have been treated with physical therapy or nutritional support.

More than 90% of Rett syndrome is known to be caused by mutations in the 'MeCP2' gene.

Disease occurs when MeCP2 does not function normally. It develops relatively normally from 6 to 18 months of age, and then shows a decrease in brain development, loss of acquired cognitive and motor skills, and loss of language function.

The treatment under development by Rznomics is the world's first gene treatment with single dose. The 'RNA replacement enzyme', a platform technology of Rznomics that removes mutated genes and replaces them with normal genes at the same time, is applied. By replacing the mutant MeCP2 gene with a normal gene, it is expected that a fundamental treatment of the disease will be possible.

Seong-wook Lee, CEO of Rznomics, said, "It is the result of a positive evaluation of the potential of Rznomics' gene therapy by the National New Drug Development Foundation”,. Director Han Seung-ryeol, who was selected as the research director, said, "since Si-yoon Kim, a professor of veterinary medicine at Konkuk University participate as a co-researcher, synergy is expected through industry-university collaboration."