Drug intelligence / Profile preview

AAV9-Des-UFµDys2

Development stage: Preclinical
Lead developer: University of Florida
Modality: Gene Therapies
Administration: Intravenous

Overview

AAV9-Des-UFµDys2 is an adeno-associated virus serotype 9 (AAV9) gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes a muscle-specific desmin (Des) promoter to drive the expression of a microdystrophin (µDys2) transgene, aiming to restore functional protein expression in cardiac and skeletal muscles. In preclinical studies, it is often evaluated alongside immunomodulatory strategies, such as liver-directed tolerogenic vectors, to overcome immune responses against the AAV capsid and the dystrophin transgene, thereby enhancing therapeutic durability and efficacy.

Other names: AAV9-Des-UFmicroDys2AAV-9-Des-UFmicroDys2AAV 9-Des-UFmicroDys2

Targets

F-actin (Actin, alpha skeletal muscle (ACTA1))Gal (Terminal galactose residues on N-linked glycans)DAG1 (Dystroglycan 1)RPSA (37/67 kDa laminin receptor)

Beyond the preview

Go deeper on AAV9-Des-UFµDys2.

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 AAV9-Des-UFµDys2.

Explore the full profile