[Edaily Reporter KIM SAE-MI ] Olix Pharmaceuticals, Inc.(226950)announced the development progress of its age-related macular degeneration (AMD) treatment “OLX301A” at a conference specializing in oligonucleotide therapeutics co-hosted by the U.S. Food and Drug Administration (FDA) and the Drug Information Association (DIA).

Olix Pharmaceuticals, Inc. announced on the 29th that it was invited to the “2026 DIA/FDA Conference on Oligonucleotide-Based Therapies,” held in Arlington, Virginia, from the 23rd to the 25th (local time), where it shared its experience in developing RNA interference (RNAi) therapies for ophthalmic diseases, with a focus on OLX301A (molecule name: OLX10212).

This event, a specialized conference on oligonucleotide therapeutics jointly organized by the FDA and the DIA, serves as a forum for experts from regulatory agencies, the pharmaceutical and biotech industries, and academia to discuss research and development as well as regulatory issues related to oligonucleotide therapeutics.

Dr. Pooja Dua of Olix Pharmaceuticals, Inc.’s U.S. subsidiary, Olix US, participated as a speaker on the 25th in the session titled “Extra-Hepatic Delivery Strategies to Optimize Distribution and Exposure for Improved Efficacy and Safety in Translational Models.”

The session covered technologies for delivering oligonucleotides to tissues other than the liver, as well as nonclinical trial design, pharmacology, absorption, distribution, metabolism, and excretion (ADME), and toxicity. Kim Tae-won, Vice President of Preclinical Development at Ionis Pharmaceuticals, and James Wilde, a pharmacologist at the FDA’s Center for Drug Evaluation and Research (CDER), served as co-chairs.

Dr. Doo-A delivered a presentation titled “Inhibition of Ocular Degeneration and Neovascularization via MyD88-Targeting Cell-Penetrating Asymmetric siRNA.” OLX301A is an RNAi therapeutics that targets MyD88, an inflammatory signaling protein involved in the onset and progression of age-related macular degeneration (AMD).

In preclinical studies, OLX301A demonstrated efficacy in inhibiting retinal damage and functional decline, as well as reducing abnormal neovascularization. In a Phase 1 clinical trial conducted in the United States, no drug-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) were observed, and early signs of efficacy—including improvements in best-corrected visual acuity (BCVA)—were observed in some patients, including those with geographic atrophy (GA).

Based on the Phase 1 results, Olix Pharmaceuticals, Inc. is currently conducting a global Phase 2a clinical trial to evaluate the efficacy of OLX301A in patients with age-related macular degeneration (AMD) accompanied by geographic atrophy (GA).

At this event, Olix Pharmaceuticals, Inc. also presented preliminary research results on the application of its dual-target RNAi platform, “OASIS-D,” to ophthalmic diseases. OASIS-D is a platform designed to simultaneously suppress two disease-related genes within the same tissue using a single therapeutic agent. The presentation shared preliminary research results demonstrating the potential to simultaneously suppress both genes in ocular tissue.

An Olix Pharmaceuticals, Inc. representative stated, “At the joint FDA and DIA conference, we shared the development progress of OLX301A and the preliminary research results of the dual-target RNAi platform applied to ophthalmic diseases,” adding, “Since we have already submitted an Investigational New Drug (IND) application to enter a global Phase 2a clinical trial, we will prepare for the subsequent clinical trial without delay.”