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). (Show More)
Indication	Alpha 1-Antitrypsin Deficiency
Compound Name	BEAM-302
Sponsor	Beam Therapeutics Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	106

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	Undisclosed low dose
Dose 2	Undisclosed intermediate dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	3
Locations	New Zealand,United Kingdom

Has US IND	True
Recent Updates	First patient dosed 6/2024, initial clinical data expected 2025