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AveXis

Bannockburn, Illinois, United Statesnovartis.com ↗

Ownership: Private
Employees: ~200
Development stage: Phase 3

Overview

AveXis is now Novartis Gene Therapies, focused on AAV-mediated gene therapy treatments for rare diseases. Its lead product, onasemnogene abeparvovec (Zolgensma), is the first approved gene therapy for pediatric spinal muscular atrophy. Other programs target Rett syndrome and ALS with SOD1 mutations.

Therapeutic areas: Neurology
Modalities: Gene therapy
Industry: Pharma

Drug pipeline

1 asset

AVXS-101

Phase 3

Indications: Spinal muscular atrophy Type 1

Partnerships

Academic collaborators (e.g., Nationwide Children's Hospital)

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