Drug intelligence / Profile preview

AGIL-AS

Development stage: Preclinical
Lead developer: PTC Therapeutics
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies

Overview

AGIL-AS is an investigative gene therapy candidate developed to treat Angelman syndrome by restoring expression of the ubiquitin-protein ligase E3A (UBE3A) gene in the central nervous system of affected patients. It is designed to deliver a functional copy of UBE3A using a viral vector to neurons, with the goal of rescuing neurological deficits such as developmental delay, impaired motor function, seizures, and cognitive dysfunction characteristic of Angelman syndrome.[3][5]

Targets

UBE3A (Ubiquitin-protein ligase E3A)

Beyond the preview

Go deeper on AGIL-AS.

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 AGIL-AS.

Explore the full profile