nL-TUBB4-001

Phase 2Small molecule

UUniversity of California, San Diego

nL-TUBB4-001 is one of 2 small molecule programs targeting Leukodystrophy. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

LeukodystrophyActive, not recruiting

LeukodystrophyPhase 2

Active, not recruiting

Competition

Competitive Landscape

2 competitors in Leukodystrophy

#DrugCompanyPhaseTrials

Antisense Oligonucleotide Treatment (ASO)

MMassachusetts General Hospital

Phase 2

1 2

nL-TUBB4-001

UUniversity of California, San Diego

Phase 2

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

Timeline

Expected Milestones

Q3 2027

Phase 1/2Active, not recruiting

Leukodystrophy

NCT07222371

Clinical

Trials by Indication[1]

Leukodystrophy1 trial

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

Active, not recruiting·UUniversity of California, San Diego·Phase 1/2·NCT07222371·1 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for nL-TUBB4-001. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — nL-TUBB4-001 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for nL-TUBB4-001. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — University of California, San Diego company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: UUniversity of California, San Diego

nL-TUBB4-001

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2027

Trials by Indication[1]

Leukodystrophy1 trial

References

Drug Info

Quick Facts

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