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Clinical Trial Report

Gene Therapy Trial Report

Summary

NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

NCTID NCT06622668 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Alpha-1 Antitrypsin Deficiency (AATD)
Disease Ontology Term DOID:13372
Compound Name NTLA-3001
Sponsor Intellia Therapeutics
Funder Type Industry
Recruitment Status
Withdrawn
Results Posted Not Available

Therapy Information

Target Gene/Variant SERPINA1
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type MRNA, LNP
Target Tissue/Cell Liver
Delivery System Lipid encapsulation
Vector Type LNP
Editor Type Cas9 mRNA
Dose 1 3 undisclosed dose levels
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2024-09-30
Completion Date 2025-01-09
Last Update 2025-01-17

Participation Criteria

Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations New Zealand

Regulatory Information

Has US IND False
FDA Designations
Recent Updates Sponsor discontinued this program, no subjects were enrolled and the trial was withdrawn from clinicaltrials.gov

Resources/Links

News and Press Releases