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MedXY AI/MedXY News/Tag: Antisense oligonucleotide
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Antisense oligonucleotide

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AHB-137: First-in-Human Study Shows Promising Antiviral Activity and Safety in Chronic Hepatitis B Management
GastroenterologyAHB-137chronic hepatitis B

AHB-137: First-in-Human Study Shows Promising Antiviral Activity and Safety in Chronic Hepatitis B Management

By MedXY|Aug 17, 2026

AHB-137, an antisense oligonucleotide targeting HBV mRNA, demonstrated safety, favorable pharmacokinetics, and rapid HBsAg reduction in healthy volunteers and chronic hepatitis B patients, supporting further clinical development.

Decoding Tofersen’s CNS Penetrance and Efficacy in SOD1-ALS Through Autopsy Tissue Analysis
NeurologyAntisense oligonucleotide
Central Nervous System

Decoding Tofersen’s CNS Penetrance and Efficacy in SOD1-ALS Through Autopsy Tissue Analysis

By MedXY|Jul 8, 2026

This study unveils tofersen’s distribution and potent SOD1 reduction within the CNS of SOD1-ALS patients, utilizing autopsy tissues from clinical trial participants, confirming anticipated pharmacokinetic and pharmacodynamic effects.

Long-Term Tofersen Therapy in SOD1-ALS: Early Initiation Preserves Function and Extends Survival
NeurologyAmyotrophic Lateral SclerosisAntisense oligonucleotide

Long-Term Tofersen Therapy in SOD1-ALS: Early Initiation Preserves Function and Extends Survival

By MedXY|Feb 11, 2026

The integrated analysis of the VALOR trial and its open-label extension reveals that early initiation of tofersen significantly slows functional decline, preserves respiratory capacity, and improves survival in patients with SOD1-ALS over a

Functional Cure Within Reach: GSK’s Bepirovirsen Meets Primary Endpoints in Pivotal Phase III Chronic Hepatitis B Trials
GastroenterologyAntisense oligonucleotidebepirovirsen

Functional Cure Within Reach: GSK’s Bepirovirsen Meets Primary Endpoints in Pivotal Phase III Chronic Hepatitis B Trials

By MedXY|Jan 7, 2026

GSK has announced positive Phase III results for Bepirovirsen, an antisense oligonucleotide that achieves functional cure in chronic hepatitis B patients. By targeting viral RNA and HBsAg, the drug offers a finite treatment path, potentiall

AZD2693: Targeting PNPLA3 148M Variant as a Precision Therapy for Metabolic Steatohepatitis
Clinical UpdatesAntisense oligonucleotideMASH

AZD2693: Targeting PNPLA3 148M Variant as a Precision Therapy for Metabolic Steatohepatitis

By MedXY|Oct 8, 2025

AZD2693, a liver-directed antisense oligonucleotide, safely reduces PNPLA3 expression and liver fat in homozygous PNPLA3 148M carriers with metabolic dysfunction-associated steatohepatitis, signaling promise for precision treatment in this

ION224: A Promising Antisense Oligonucleotide Therapy for Metabolic Dysfunction-Associated Steatohepatitis (MASH)
Clinical UpdatesAntisense oligonucleotideMASH

ION224: A Promising Antisense Oligonucleotide Therapy for Metabolic Dysfunction-Associated Steatohepatitis (MASH)

By MedXY|Oct 5, 2025

The phase 2 ION224-CS2 trial demonstrates that ION224, a DGAT2 antisense oligonucleotide, safely improves liver histology in MASH patients independent of weight loss, offering a potential new therapeutic approach.

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