Drug intelligence / Profile preview

AMT-191

Development stage: Phase 2
Lead developer: uniQure
Modality: Gene Therapies
Administration: Intravenous

Overview

AMT-191 is an adeno-associated virus serotype 5 (AAV5) gene therapy candidate developed by uniQure for the treatment of Fabry disease. It is designed to deliver a functional copy of the GLA gene, which encodes the alpha-galactosidase A (α-Gal A) enzyme. Fabry disease is a lysosomal storage disorder caused by mutations in the GLA gene, leading to the accumulation of globotriaosylceramide (Gb3) in various tissues. By providing a stable source of α-Gal A expression, AMT-191 aims to reduce Gb3 accumulation and prevent the progressive organ damage associated with the disease. It is currently being evaluated in Phase 1/2 clinical trials for adult males with classic Fabry disease.

Other names: AAV5-GLAAAV-5-GLAAAV 5-GLA

Targets

GLA (Alpha-galactosidase A)

Beyond the preview

Go deeper on AMT-191.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on AMT-191.

Explore the full profile