Drug intelligence / Profile preview

GTP-206

Development stage: Preclinical
Lead developer: Passage Bio
Modality: Gene Therapies
Administration: Intravenous

Overview

**GTP-206** is an investigational in vivo gene therapy designed to treat Krabbe disease, a rare lysosomal storage disorder caused by mutations in the GALC gene leading to galactocerebroside accumulation and demyelination. It utilizes adeno-associated virus (AAV) vectors to deliver a functional GALC gene, aiming to restore enzyme activity and halt disease progression. Developed by Passage Bio in collaboration with the University of Pennsylvania's Gene Therapy Program, it received FDA rare pediatric disease designation.[7][9][11]

Targets

GALC (Galactosylceramidase)

Beyond the preview

Go deeper on GTP-206.

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 GTP-206.

Explore the full profile