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nL-PACS1-001

Phase 2Small molecule

Nn-Lorem Foundation

1

Indications

1

Total Trials

1

Active Trials

0

Competitors

Pipeline

Status by Indication[3][1]

Indication

Schuurs-Hoeijmakers SyndromeNot yet recruiting

Schuurs-Hoeijmakers SyndromePhase 2

Not yet recruiting

Timeline

Expected Milestones

Q2 2028

Phase 1/2Not yet recruiting

Schuurs-Hoeijmakers Syndrome

NCT07474298

Clinical

Trials by Indication[1]

Schuurs-Hoeijmakers Syndrome1 trial

Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS)

Not yet recruiting·Nn-Lorem Foundation·Phase 1/2·NCT07474298·1 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for nL-PACS1-001. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — nL-PACS1-001 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for nL-PACS1-001. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — n-Lorem Foundation company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: Nn-Lorem Foundation

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