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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
NCTID
NCT06389877
(View at clinicaltrials.gov)
Description
This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).
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Development Status
Active
Indication
Alpha-1 Antitrypsin Deficiency (AATD)
Disease Ontology Term
DOID:13372
Compound Name
BEAM-302
Sponsor
Beam Therapeutics Inc.
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
106
Results Posted
Not Available
Therapy Information
Target Gene/Variant
SERPINA1 (p.Glu366Lys)
Therapy Type
Gene editing
Therapy Route
In-vivo
Mechanism of Action
Mutation correction
Route of Administration
Intravenous
Drug Product Type
MRNA, LNP
Target Tissue/Cell
Liver
Delivery System
Lipid encapsulation
Vector Type
LNP
Editor Type
ABE
Dose 1
15mg
Dose 2
30mg
Dose 3
60mg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-04-22
Completion Date
2027-08
Last Update
2025-01-10
Participation Criteria
Eligible Age
18 Years - 70 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
5
Locations
New Zealand,Netherlands,United Kingdom,Australia
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Beam Therapeutics Announces Positive Initial Data; IND cleared by FDA March 2025
Resources/Links
News and Press Releases
Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation
Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) by the United States (U.S.) Food and Drug Administration
Beam Therapeutics Announces Progress in Hematology and Genetic Disease Franchises and Outlines Key 2025 Anticipated Catalysts
Preclinical Publications
(Corporate Presentation) BEAM-302: Targeting AATD-related Liver and Lung Disease with Base Editing
(Poster) BEAM-302 decreases hepatic aggregates of mutant AAT and increases circulating functional AAT in rodent models of Alpha-1 Antitrypsin Deficiency - ESGCT 2023
(Presentation) BEAM-302 Base editing as a potential therapeutic approach for alpha-1 antitrypsin deficiency (Alpha-1) - Alpha-1 National Conference 2024