Pariglasgene brecaparvovec

DB16801ApprovedGene Therapies

Pariglasgene brecaparvovec is an AAV8-based gene therapy indicated as an adjunct to nutritional management in patients 8 years and older with glycogen storage disease type Ia, delivering a functional G6PC gene to hepatocytes to restore glucose-6-phosphatase activity. Pariglasgene brecaparvovec-opnr is an adeno-associated virus serotype 8 (AAV8)–based gene therapy for glycogen storage disease type Ia (GSDIa), a disorder caused by deficiency of the enzyme glucose-6-phosphatase (G6Pase).

Mechanism: Pharmacologically active on Glucose-6-phosphatase catalytic subunit 1
Curator reviewed
Primary indication: Pariglasgene brecaparvovec-opnr is indicated to reduce daily cornstarch intake as an adjunct to nutritional management in adult and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa).Curator reviewed · 1 structured indication
First approval: United States, 2026
Also known as: Pariglasgene brecaparvovec-opnr
Code names: AAV8G6PASE · DTX-401

Resolves to

Compound

UNII7LP92X4WPECAS2259927-72-3

Targets

P35575G6PC1

What you can answer from here — as of September 21, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

557Drug interactionsStructured to mechanism, not free text

2Clinical trialsPhase, status and sponsor resolved per trial

1Structured indicationCondition, population, route and combination as fields, not prose

2ContraindicationsEach with its own population and attribute set

13Marketed productsAcross 1 country and 1 labeller

3+ReferencesStructured and connected to the statements they support

Which drugs share a target with Pariglasgene brecaparvovec, and which of those have an active Phase 3 trial?

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