rAAV9-CMV-hNAGLUop

Preclinical

NNeuroGT

rAAV9-CMV-hNAGLUop is one of 3 therapeutic programs targeting Sanfilippo Syndrome. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[2][1]

Indication

Sanfilippo SyndromeNot yet recruiting

Sanfilippo SyndromePreclinical

Not yet recruiting

Competition

Competitive Landscape

2 competitors in Sanfilippo Syndrome

#DrugCompanyPhaseTrials

Epidiolex

Jazz Pharmaceuticals

Preclinical

1 2

Epidiolex

Jazz Pharmaceuticals

Preclinical

1 3

rAAV9-CMV-hNAGLUop

NNeuroGT

Preclinical

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

Timeline

Expected Milestones

Q4 2029

PreclinicalNot yet recruiting

Sanfilippo Syndrome

NCT07818759

Clinical

Trials by Indication[1]

Sanfilippo Syndrome1 trial

An Open-Label, Single Injection, Ascending Dose Phase I/II Clinical Study of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

Not yet recruiting·NNeuroGT·Preclinical·NCT07818759·9 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for rAAV9-CMV-hNAGLUop. U.S. National Library of Medicine, National Institutes of Health.
[2]
Open Targets Platform — Drug–disease associations for rAAV9-CMV-hNAGLUop. EMBL-EBI & Wellcome Sanger Institute.
[3]
SEC EDGAR — NeuroGT company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Highest Phase: Preclinical[1]
Company: NNeuroGT

rAAV9-CMV-hNAGLUop

Status by Indication[2][1]

Competitive Landscape

Expected Milestones

Q4 2029

Trials by Indication[1]

Sanfilippo Syndrome1 trial

References

Drug Info

Quick Facts

Related News