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Adjuvant IM Therapy

PreclinicalSmall molecule

1

Indications

2

Total Trials

2

Active Trials

0

Competitors

Pipeline

Status by Indication[3][1]

Indication

MPS IIIARecruiting

MPS IIIAPreclinical

Recruiting

Timeline

Expected Milestones

Q1 2029

PreclinicalRecruiting

MPS IIIA

NCT02716246

Clinical

Trials by Indication[1]

MPS IIIA1 trial

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Recruiting· Ultragenyx logo

Ultragenyx·Preclinical·NCT02716246·36 enrolled

Mucopolysaccharidosis III-A1 trial

A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

Enrolling by invitation· Ultragenyx logo

Ultragenyx·Phase 3·NCT04360265·41 enrolled

Sources

References

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

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Preclinical[1]
Company: Ultragenyx

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