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OliX Pharmaceuticals Identifies Effective Inhibition of Target Gene Expression and Delivery of Candidate to Brain for CNS Therapeutics

SUWON, South Korea--(BUSINESS WIRE)--#AlzheimersDisease--OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today announced that the Company identified effective inhibition of target gene expression in rodent brain tissues for its central nervous system (CNS) treatment program.

OliX is currently developing therapeutics for CNS diseases, such as neuropathic pains and degenerative brain disorders, using its proprietary cell-penetrating asymmetric RNA platform (cp-asiRNA). With a single intrathecal injection of its optimized therapeutic candidate, the Company identified up to 90 percent knockdown of gene expression in a rodent study. The siRNA was delivered not only to the spinal cord but also to multiple areas of the brain.

According to June Park, Ph.D., head of R&D, "We adopted the intrathecal administration route to deliver the siRNA to brain without having to cross the blood-brain barrier (BBB). Effective delivery of a therapeutic candidate is a critical factor in determining the success of CNS therapeutics development. We are excited to find robust knockdown of target gene expression in brain tissues.”

The most common degenerative brain diseases include Alzheimer’s disease, which affects one in 100 seniors aged 65 years or older, and Parkinson’s disease, a motor disturbance disease caused by loss of brain nerve cells. Neuropathic pains occur due to damages or functional anomalies in the nervous system.

“We are actively discovering new CNS indications and securing candidates for further development,” said Dong Ki Lee, Ph.D., founder and chief executive officer of OliX Pharmaceuticals. “We plan to expand the CNS pipeline rapidly based on our extensive expertise in leveraging the cp-asiRNA platform.”

About OliX Pharmaceuticals

OliX Pharmaceuticals is a clinical-stage pharmaceutical company developing therapeutics against a variety of disorders by down-regulating the expression of disease-causing genes based on its own proprietary RNAi technology. The Company’s core RNAi platform, asymmetric siRNA (asiRNA), is a unique gene silencing technology based on RNA interference (RNAi), which is considered the most efficient gene silencing technology. Utilizing this proprietary asiRNA technology, OliX has developed cell penetrating asiRNA (cp-asiRNA), a therapeutic RNAi platform to effectively target diseases locally, such as hypertrophic scarring, dry and wet age-related macular degeneration (AMD), subretinal fibrosis, and neuropathic pain. OliX has also developed another therapeutic RNAi platform, GalNAc-asiRNA, to target a variety of liver diseases.

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Jiyoun Kim
OliX Pharmaceuticals PR


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Last Updated: 04-May-2022