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

NCTID	NCT06622668 (View at clinicaltrials.gov)
Description	This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease (Show More)
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Recruitment Status	Withdrawn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

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

Eligible Age	18 Years - 75 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion: 1. Age 18 years to 75 years 2. Diagnosis of AATD ZZ/ZNull genotypes 3. FEV1 ≥35% and ≤65% 4. No evidence of liver cirrhosis 5. Adequate chemistry and hematology measures at screening 6. Participants must agree not to participate in another interventional study for the duration of this trial. 7. Participants must be capable of providing signed informed consent Exclusion Criteria: 1. AATD genotypes outside of ZZ or ZNull 2. Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off 3. Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component. 4. Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant. 5. Unwilling to comply with study procedures. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	1
Locations	New Zealand

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Recent Updates	Sponsor discontinued this program, no subjects were enrolled and the trial was withdrawn from clinicaltrials.gov