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NGGT002

Phenylketonuria (PKU)

Phase 1/2 (inferred)Active

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Phase 1/2 (inferred)

Status

Active

Company

NGGT

About NGGT

Next Generation Gene Therapeutics (NGGT) is a private, pre-revenue biotech developing gene therapies for rare and chronic diseases. The company leverages a dual-functional vector technology platform and operates a significant 90,000 sq. ft. cGMP manufacturing facility to support its pipeline. Its most advanced program, NGGT002 for Phenylketonuria (PKU), has reported positive data, positioning the company in the competitive gene therapy space with a global operational footprint in the US and China.

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Therapeutic Areas

Rare Diseases Ophthalmology Metabolic Disorders Neurodegenerative Diseases

Other Phenylketonuria (PKU) Drugs

Drug	Company	Phase	Status
SYNB1934	Synlogic	Phase 3	Completed; Did Not Meet Primary Endpoint
PKU Treatment	Cycle Pharmaceuticals	Commercial	Active
NB-20X	Nerai Biosciences	Pre-clinical	Active
SOM1311	SOM Biotech	Preclinical	Active
Repinatrabit	Otsuka	Open-Label Extension	Active
PER303	Perseo Pharma	Preclinical	Active
Engineered Native Bacteria Platform	Endure Biotherapeutics	Pre-clinical	Active
JNT-517	JCR Pharmaceuticals	Phase 1	Active
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Approved/Marketed	Active (Label expansion)
PTC923 (sepiapterin)	PTC Therapeutics	Phase 3	Active