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GalNAc Program (SERPINA1 target)

Alpha-1 Antitrypsin Deficiency (AATD)

DiscoveryActive

Key Facts

Indication

Alpha-1 Antitrypsin Deficiency (AATD)

Phase

Discovery

Status

Active

Company

Korro Bio

About Korro Bio

Korro Bio is developing transformative genetic medicines by co-opting the body's natural RNA editing machinery to correct disease-causing mutations or modulate protein function. The company's OPERA® platform and first-generation CHORD™ oligonucleotides are designed for high specificity, transient activity, and delivery via clinically validated methods like GalNAc. With a pipeline focused on liver and CNS diseases, Korro is advancing multiple wholly-owned programs, recently securing an $85 million private placement to fund its development efforts.

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Therapeutic Areas

Metabolic Disorders Neurology Rare Diseases Hepatology

Other Alpha-1 Antitrypsin Deficiency (AATD) Drugs

Drug	Company	Phase	Status
BEAM-302	Beam Therapeutics	Preclinical	IND-enabling
WVE-006	WaVe Life Sciences	Phase 1/2	Active - INHALE trial; Accelerating regulatory engagement
INBRX-101	Inhibrx Biosciences	Phase 2	Active
Glassia® (IV AAT)	Kamada	Approved	Commercial
Inhaled AAT (KMA-001)	Kamada	Phase 3	Active
Unnamed AATD Program	AlveoGene	Pre-clinical	Lead Program