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JNT-517

Phenylketonuria (PKU)

Phase 1Active

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Phase 1

Status

Active

Company

JCR Pharmaceuticals

About JCR Pharmaceuticals

JCR Pharmaceuticals has established itself as a premier global specialty pharmaceutical company focused on high-unmet-need rare diseases, particularly lysosomal storage disorders. Its strategic advantage is anchored in the proprietary J-Brain Cargo® platform, which enables targeted delivery of biologics across the blood-brain barrier—a key differentiator in treating neurological manifestations. The company has successfully commercialized products in multiple international markets and maintains a robust clinical pipeline, driving its significant market valuation. JCR's fully integrated model, from R&D to global commercialization, combined with a strong venture spirit, positions it for sustained growth in the orphan drug space.

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Therapeutic Areas

Lysosomal Storage Disorders Neurological Diseases Rare Genetic 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
NGGT002	NGGT	Phase 1/2 (inferred)	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
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Approved/Marketed	Active (Label expansion)
PTC923 (sepiapterin)	PTC Therapeutics	Phase 3	Active