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Subject OliX Pharmaceuticals Expands Ocular Disease Pipeline; Adds OLX304A for the Treatment of Retinitis Pi
Writer 관리자 Date 2019.05.10 See 1605
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OliX Pharmaceuticals Expands Ocular Disease Pipeline; Adds OLX304A for the Treatment of Retinitis Pigmentosa

May. 10, 2019

OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, announced today that an expansion of its ocular disease pipeline. Specifically, the company announced that OLX304A has been added to develop an RNAi therapeutic with a novel target (undisclosed) for treatment of Retinitis Pigmentosa (RP) in a presentation at the Investor Relations Meeting in Seoul on May 10th.

“We have confirmed the strong potential of treating RP using our cp-asiRNA platform technology which is an optimal siRNA technology for developing ocular therapeutics. We look forward to advancing OLX304A as a highly innovative approach to benefit RP patients.” said Dong-ki Lee, Ph.D., the founder and CEO of OliX.

OLX304A is a program to develop a treatment that targets a single gene for RP patients regardless of their disease-causing gene mutation. This strategy is different from the conventional treatment development strategies for RP which target individual disease-causing genes.

An IND application of OLX304A program is planned to be submitted to the US FDA to initiate a Phase 1 within the first half of next year.

What is Retinitis Pigmentosa?
Retinitis pigmentosa (RP) is the name given to a group of inherited eye diseases that affect the retina (the light-sensitive part of the eye). RP causes the breakdown of photoreceptor cells (cells in the retina that detect light). Photoreceptor cells capture and process light helping us to see. As these cells breakdown and die, patients experience progressive vision loss.
The most common feature of all forms of RP is a gradual breakdown of rods (retinal cells that detect dim light) and cones (retinal cells that detect light and color). Most forms of RP first cause the breakdown of rod cells. These forms of RP, sometimes called rod-cone dystrophy, usually begin with night blindness. Night blindness is somewhat like the experience normally sighted individuals encounter when entering a dark movie theatre on a bright, sunny day. However, patients with RP cannot adjust well to dark and dimly lit environments.
RP is an inherited disorder that results from harmful changes in any one of more than 50 genes. These genes carry the instructions for making proteins that are needed in cells within the retina, called photoreceptors. Some of the changes, or mutations, within genes are so severe that the gene cannot make the required protein, limiting the cell's function. Other mutations produce a protein that is toxic to the cell. Still other mutations lead to an abnormal protein that doesn't function properly. In all three cases, the result is damage to the photoreceptors.
RP is considered a rare disorder. Although current statistics are not available, it is generally estimated that the disorder affects roughly 1 in 4,000 people, both in the United States and worldwide.
https://nei.nih.gov/health/pigmentosa/pigmentosa_facts
Source by NIH

OliX Pharmaceuticals, Inc.
OliX is a clinical stage pharmaceutical company developing therapeutics against a variety of disorders by down-regulating expression of disease-causing genes, based on its own proprietary RNAi technology. The company is currently developing novel therapeutic programs for treatment of various diseases with high unmet medical needs, including hypertrophic scar, dry & wet age-related macular degeneration (AMD), subretinal fibrosis, Retinitis Pigmentosa and idiopathic pulmonary fibrosis (IPF).

Source by OliX Pharmaceuticals, Inc.