Drug intelligence / Profile preview

ATA-300

Development stage: Preclinical
Lead developer: Atamyo Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies

Overview

ATA-300 is a preclinical-stage adeno-associated virus (AAV) mediated gene replacement therapy developed by Atamyo Therapeutics for the treatment of Limb-Girdle Muscular Dystrophy Type R1 (LGMD-R1), also known as LGMD2A. The therapy is designed to deliver a functional copy of the CAPN3 gene to restore the production of Calpain-3, a protein essential for muscle cell maintenance that is deficient in patients with this condition. ATA-300 is currently undergoing IND-enabling studies, with clinical trials expected to commence in 2026.

Targets

CAPN3 (B-Raf proto-oncogene, serine/threonine kinase)

Beyond the preview

Go deeper on ATA-300.

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 ATA-300.

Explore the full profile