Therapy Areas: Central Nervous System
OliX Pharmaceuticals Identifies Effective Inhibition of Target Gene Expression and Delivery of Candidate to Brain for CNS Therapeutics
4 May 2022 - - South Korea-based RNAi therapeutics developer OliX Pharmaceuticals, Inc. (KOSDAQ:226950) has identified effective inhibition of target gene expression in rodent brain tissues for its central nervous system treatment program, the company said.

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 % 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.

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.

OliX Pharmaceuticals is a clinical-stage pharmaceutical company developing therapeutics against a range 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, 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, 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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