Rznomics, Selection of national new drug development projects for gene therapy

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Rznomics, trans-splicing ribozyme based gene therapy technology platform company, announced on the 12th that their innovative new drug treatment, which is being developed for hereditary retinal pigmentation, was selected as a support project for the National New Drug Development Project's first "New Drug R&D Ecosystem Construction Research" in 2022.

The national new drug development project is a pan-ministerial national R&D project, which is promoted by the Ministry of Science and ICT, the Ministry of Trade, Industry and Welfare, and has been supporting the whole cycle of new drug development since 2021 with the goal of strengthening global competitiveness of the national pharmaceutical and bio industries.

Rznomics' selected research on building a national new R&D ecosystem is a task to support candidate materials and non-clinical stages, and this selection will receive a total of KRW 2 billion in national research funds for 22 months with the goal of non-clinical research and IND approval.

Retinal pigmentation is a blindness disease that occurs in one out of every 4,000 to 5,000 people worldwide and loses central vision due to abnormal photoreceptor cells distributed in the retina.

Up to now, there is no fundamental treatment method, and it has relied on some adjuvant treatments that prevent vision from being damaged by ultraviolet rays or slow vision loss by taking nutritional supplements.

The cause of retinal pigmentation is a defect in the genes involved in the mechanism of converting light into electrical signals in visual cells.

Among several gene mutations, mutations in the RHO gene are known to be the most common form of mutations in the disease, accounting for 20 to 30% of autosomal dominant retinal pigmentation.

The progress rapidity of vision damage depends on individual, but it usually starts with night blindness, gradually narrowing the field of vision, and losing the central field of vision, leading to complete blindness.

The treatment under development by Rznomics is an RNA-based gene therapy that is being developed for a single administration, and it is expected that the disease can be fundamentally treated by replacing the mutant RHO gene with the normal RHO gene using "Trans-splicing ribozyme" technology, which removes and corrects the mutant gene.

Dr. Ji-hyun, Kim who was selected as the research director of the project, said, "Rznomics' platform technology, which has accumulated technology for a long period of time, has been recognized for its excellence, and we will do our best to approve IND with the goal of clinical progress."

Seong-wook, Lee, CEO of Rznomics, said, "This is the second national new drug project since the development of Rett’s syndrome treatment was selected as a national new drug project at the end of last year, and it is believed to be a good example of new drug development as a bio-venture in Korea."