ABL Bio said on the 15th that its U.S. partner Ionis Pharmaceuticals confirmed through preclinical research that the blood-brain barrier (BBB) shuttle platform "Grabody-B" enables delivery of oligonucleotide therapeutics to the brain.
Ionis announced the findings at the global ribonucleic acid (RNA) therapeutics conference "TIDES USA 2026: Oligonucleotide & Peptide Therapeutics," held in Boston on the 13th (local time).
Hien Zhao, a vice president at Ionis who delivered the presentation, introduced Grabody-B as one of the promising technologies for BBB penetration of oligonucleotide therapeutics.
According to joint research by ABL Bio and Ionis, when a therapy combining small interfering RNA (siRNA), a type of oligonucleotide therapeutic, with Grabody-B was administered intravenously, it reduced target gene expression in key brain regions including the cerebrum, striatum, thalamus, and cerebellum. The effect increased with higher doses.
In particular, the cerebellum is important for degenerative brain diseases and motor control, but conventional oligonucleotide therapeutics have struggled to deliver drugs deep enough into brain regions. The company said the study confirmed the potential to deliver drugs to major brain regions including the cerebellum, and noted it expects the platform could be used to develop treatments for degenerative brain diseases.
ABL Bio is developing a range of pipelines based on its bispecific antibody platform "Grabody." Clinical projects are underway in the United States, China, Australia, and Korea.
Among them, follow-up clinical trials for the Parkinson's disease drug candidate "ABL301" are set to be conducted by Sanofi in France, and "ABL001," under development as a cholangiocarcinoma treatment, has received priority review (fast track) and orphan drug designation from the U.S. Food and Drug Administration (FDA).
In addition, "ABL111," being co-developed with Novabridge, has entered a phase 2 trial in combination with nivolumab and chemotherapy, and the company plans to release additional phase 1b data at a global conference in the second half of this year.
Chief Executive Officer Lee Sang-hoon of ABL Bio said, "Through joint research with Ionis, we confirmed the potential of Grabody-B to serve as a core delivery platform for developing oligonucleotide-based treatments for degenerative brain diseases," and added, "We are currently advancing it as a platform optimized for oligonucleotide therapeutics."
Lee added, "In the same study, we also confirmed the potential for delivery to peripheral tissues such as muscle and the heart, and we are considering expanding the range of applicable disease areas."