Antisense oligonucleotide treatment (ASO)

Biologic

Antisense oligonucleotide treatment (ASO) is one of 15 biologic programs targeting Movement Disorder. It is currently in development.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Movement DisorderActive, not recruiting

Movement DisorderPhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

15 competitors in Movement Disorder

#DrugCompanyPhaseTrials

Ingrezza

Neurocrine Biosciences

Keppra

Feosol

AAuerbach Hematology Oncology Associates P C

Approved

1 4

Pepcid

KKenvue Brands

Approved

—5

Inderal

Mylan Pharmaceuticals Inc

BIIB122

1 7

UX007

Ultragenyx

Phase 3

1 8

Risperdal

WWim Janssens

Phase 1

1 9

Seroquel

EEngelhard Arzneimittel GmbH & Co.KG

Phase 1

1 10

Zyprexa

MMODAG GmbH

Phase 1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1/2Recruiting

Movement Disorder

NCT07363603

Clinical

Trials by Indication[1]

Movement Disorder1 trial

Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.

Recruiting·PPirogov Russian National Research Medical University·Phase 1/2·NCT07363603·5 enrolled

Leukodystrophy1 trial

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Enrolling by invitation·MMassachusetts General Hospital·Phase 1/2·NCT06369974·1 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for Antisense oligonucleotide treatment (ASO). U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Antisense oligonucleotide treatment (ASO) compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Antisense oligonucleotide treatment (ASO). EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Massachusetts General Hospital company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Biologic[2]
Company: MMassachusetts General Hospital

Antisense oligonucleotide treatment (ASO)

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Trials by Indication[1]

Movement Disorder1 trial

Leukodystrophy1 trial

References

Drug Info

Quick Facts

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