BEAM-302

N/A

Report date: 2026/03/21 20:00:21

Base Information

Drug NameBEAM-302
Drug Alias NameAlpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency Therapeutic drugsa lipid nanoparticle-based therapy consisting of a guide RNA that targets the SERPINA1-c.1096G>A allele and a messenger RNA that encodes an ABE proteinα1-ADα-1-AD +[6] 
ClassificationCell and gene therapy > Gene therapy > Gene editing > Base editingCell and gene therapy > Gene therapy > LNP
Mechanisms of ActionAlpha-1-antiproteinase modulators
TargetsAlpha-1-antiproteinase
Therapy Area/Indicationsalpha 1-Antitrypsin Deficiency
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesNetherlandsNew Zealand +[6] 
CombinationsNo
Global Highest StatusPhase 2 Clinical
Highest Status in ChinaN/A
Originator CompaniesBeam Therapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesBeam Therapeutics Inc
Inactive CompaniesN/A
Active Indicationsalpha 1-Antitrypsin Deficiency
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A