RIBOZYME AND OMICS
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."