Optimized Prophylactic IM Therapy

PreclinicalSmall molecule

Optimized Prophylactic IM Therapy is one of 2 small molecule programs targeting MPS IIIA. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

MPS IIIARecruiting

MPS IIIAPreclinical

Recruiting

Competition

Competitive Landscape

3 competitors in MPS IIIA

#DrugCompanyPhaseTrials

Adjuvant IM Therapy

Ultragenyx

Preclinical

1 2

Optimized Prophylactic IM Therapy

Ultragenyx

Preclinical

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

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

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for Optimized Prophylactic IM Therapy. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Optimized Prophylactic IM Therapy compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Optimized Prophylactic 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

Optimized Prophylactic IM Therapy

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2029

Trials by Indication[1]

MPS IIIA1 trial

References

Drug Info

Quick Facts

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