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KB408

Alpha-1 Antitrypsin Deficiency (AATD)

PreclinicalActive

Key Facts

Indication

Alpha-1 Antitrypsin Deficiency (AATD)

Phase

Preclinical

Status

Active

Company

Krystal Biotech

About Krystal Biotech

Krystal Biotech's mission is to develop and deliver redosable gene therapies for severe, life-threatening rare diseases. Its landmark achievement is the 2023 FDA approval of VYJUVEK®, the first and only redosable gene therapy, which validates its proprietary HSV-1 vector platform. The company's strategy is to commercialize VYJUVEK globally while rapidly advancing a deep internal pipeline across multiple therapeutic areas, supported by in-house GMP manufacturing capabilities. This integrated approach positions Krystal as a leader in next-generation, repeat-administration genetic medicines.

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Therapeutic Areas

Dermatology Pulmonology Rare Genetic Diseases

Other Alpha-1 Antitrypsin Deficiency (AATD) Drugs

Drug	Company	Phase	Status
Unnamed AATD Program	AlveoGene	Pre-clinical	Lead Program
NB-101	Nerai Biosciences	Pre-clinical	Active
Undisclosed	GondolaBio	IND Enabling	Active
TSRA-196	Tessera Therapeutics	Phase 1	Active
BEAM-302 (for Beam Therapeutics)	Richmond Pharmacology	Phase 1/2	Active
Alpha-1 Antitrypsin (AAT)	Grifols	Marketed	Lifecycle Management
BEAM-302	Beam Therapeutics	Phase 1/2	Active
GalNAc Program	Korro Bio	Preclinical	Active