Contact SCGE
Your email
Message
Send
SCGE Consortium Home
|
About SCGE TCDC
|
Contact Us
|
License
Home
Gene Therapy Trial Browser
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
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)
Indication
Alpha 1-Antitrypsin Deficiency
Compound Name
NTLA-3001
Sponsor
Intellia Therapeutics
Funder Type
Industry
Status
Recruiting
Enrollment Count
30
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
Undisclosed low dose
Dose 2
Undisclosed intermediate dose
Dose 3
Undisclosed high dose
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-09-30
Completion Date
2030-05
Last Update
2024-10-30
Participation Criteria
Eligible Age
18 Years - 75 Years
Standard Ages
Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
1
Locations
New Zealand
Regulatory Information
Has US IND
False
Recent Updates
Resources/Links
Resources/Links
No External Links Available.