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NB-20X

Phenylketonuria (PKU)

Pre-clinicalActive

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Pre-clinical

Status

Active

Company

Nerai Biosciences

About Nerai Biosciences

Nerai Biosciences is a private, pre-clinical stage biotech founded in 2021 in Zurich, Switzerland. The company's core innovation is the MORPHEME platform, which integrates high-throughput directed evolution, functional screening, and machine learning to engineer novel CRISPR enzymes with superior specificity and activity. Nerai is advancing a proprietary pipeline of genome editing therapies for rare genetic diseases, with its lead program for Citrullinemia type 1 in in vivo lead optimization. The company is backed by non-dilutive grants and awards, positioning it to address previously inaccessible genetic mutations.

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Therapeutic Areas

Rare Genetic Diseases Metabolic Disorders Ophthalmology

Other Phenylketonuria (PKU) Drugs

Drug	Company	Phase	Status
SYNB1934	Synlogic	Phase 3	Completed; Did Not Meet Primary Endpoint
PKU Treatment	Cycle Pharmaceuticals	Commercial	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
JNT-517	JCR Pharmaceuticals	Phase 1	Active
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Approved/Marketed	Active (Label expansion)
PTC923 (sepiapterin)	PTC Therapeutics	Phase 3	Active