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LB-002

Development stage: Unknown
Lead developer: AstraZeneca
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravenous

Overview

LB-002 is a drug candidate associated with LogicBio Therapeutics, a company acquired by Alexion Pharmaceuticals, which is part of AstraZeneca Rare Disease. It is designed to leverage LogicBio's nuclease-free genome editing platforms, GeneRide™ and sAAVy™, to achieve durable therapeutic protein expression levels. The drug is part of a pipeline targeting pediatric-onset disorders such as methylmalonic acidemia (MMA) and hereditary tyrosinemia type 1 (HT1).

Targets

Albumin genomic locusMMUT (Methylmalonyl-coenzyme A mutase)FAH (Fumarylacetoacetate hydrolase)

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