AveXis
AveXis is a clinical-stage gene therapy biotech developing AAV-based treatments for rare neurological genetic diseases, primarily Zolgensma for spinal muscular atrophy. Acquired by Novartis in 2018 for $8.7 billion, it now operates as part of Novartis Gene Therapies with consolidated San Diego research operations.
- Company typePrivate
- Founded2013
- HeadquartersDallas, United States
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What AveXis does
AveXis is a biotechnology company focused on developing adeno-associated virus (AAV)-based gene therapies for patients with rare and life-threatening neurological genetic diseases, particularly spinal muscular atrophy (SMA). Founded in 2013 and headquartered in Dallas, Texas, AveXis developed Zolgensma (onasemnogene abeparvovec), an FDA-approved one-time intravenous gene therapy that delivers a functional copy of the SMN1 gene to address the genetic root cause of SMA. The company's core technology platform combines AAV capsid engineering for targeted gene delivery to muscle, heart, and CNS tissues with proprietary vector manufacturing capabilities, positioning Zolgensma as one of the only commercially available gene therapies for SMA globally. Zolgensma is priced at approximately $2.125 million per single-dose treatment, making it among the most expensive therapies in the world.
In May 2018, Novartis acquired AveXis for approximately $8.7 billion to gain access to its AAV gene therapy platform and lead asset Zolgensma. Following the acquisition, AveXis was integrated into Novartis Gene Therapies and its operations were consolidated into Novartis's San Diego genetic medicine research hub, alongside subsequent acquisitions of DTx Pharma (2023), Kate Therapeutics (2024), and Regulus Therapeutics (2024). The combined entity operates as part of Novartis's broader $23 billion US infrastructure commitment, which includes a $1.1 billion San Diego biomedical research hub announced in April 2025.
AveXis operates as a wholly-owned subsidiary of Novartis AG, with standalone financials no longer publicly reported. Its revenue model is built around one-time curative gene therapy treatments sold to healthcare providers and payers, supplemented by outcomes-based payment contracts, annuity-based installments, and state subscription programs designed to manage the upfront cost shock of million-dollar therapies. Customers include hospitals, specialty clinics, and treatment centers capable of administering intravenous gene therapy infusions, with the underlying patient population being infants and children diagnosed with SMA. Pre-acquisition, AveXis was listed on NASDAQ under ticker AVXS and raised approximately $75 million in venture funding prior to its acquisition.
AveXis firmographics
Firmographics- Name
- AveXis
- Legal name
- AveXis Inc.
- Website
- https://avexis.com
- Company type
- Private
- Founded year
- 2013
- Operating status
- Acquired
- Headcount range
- 11–50 employees
- Short description
- AveXis is a clinical-stage gene therapy biotech developing AAV-based treatments for rare neurological genetic diseases, primarily Zolgensma for spinal muscular atrophy. Acquired by Novartis in 2018 for $8.7 billion, it now operates as part of Novartis Gene Therapies with consolidated San Diego research operations.
- Ownership category
- akta.pro rank
AveXis industry classification
Industry- Product category
- Gene Therapy Biopharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Pharmaceutical and Medicine Manufacturing (3254)
- SIC
- Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- AAV Vector Gene Therapy Developers (HLAAACAA)
- akta.pro secondary industries
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Neurology/CNS Gene Therapies (HLAAACAH)
Keywords
Where AveXis is headquartered
LocationHeadquarters
- HQ city
- Dallas
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
AveXis business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Supply Chain, Infrastructure, Marketing or Sales, Operations
Revenue model
- Gene Therapy Product Sales: One-time curative gene therapy treatments sold to healthcare providers and payers, with Zolgensma priced at $2.125 million per treatment. Novartis Gene Therapies offers outcomes-based payment models to manage the upfront cost shock of these therapies.
- Outcomes-Based Payment Contracts: Alternative payment models including annuity-based installments, outcomes-based contracts, and state subscription programs to help health systems manage budget impacts of million-dollar gene therapies.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| One time/ perpetual license | Multi-year contract | Zolgensma one-time treatment: $2.125 million per patient |
Go-to-market motion2 records
Distribution channels1 record
Marketing channels2 records
AveXis product offering
Product offeringCore offering
AveXis develops and manufactures AAV-based gene therapies for patients with rare and life-threatening neurological genetic diseases. Its flagship product Zolgensma (onasemnogene abeparvovec) is an FDA-approved one-time intravenous gene therapy that delivers a functional copy of the SMN1 gene to treat spinal muscular atrophy (SMA). Following its 2018 acquisition by Novartis, AveXis now operates as part of Novartis Gene Therapies, with its pipeline and technology consolidated under Novartis's San Diego genetic medicine research operations.
Product overview
AveXis is a gene therapy company that was acquired by Novartis in 2018 and now operates as Novartis Gene Therapies. The company's core product is Zolgensma, an FDA-approved gene therapy for spinal muscular atrophy (SMA). AveXis developed AAV (adeno-associated virus) vector-based gene therapies focused on treating severe genetic diseases, particularly neurological conditions. The company operates as part of Novartis's consolidated genetic medicine research in San Diego, which includes capabilities in gene therapy and xRNA technologies.
Differentiator
Problem solved
Functional benefit
Brands
- Novartis Gene Therapies: Novartis's gene therapy business unit incorporating AveXis programs including Zolgensma (onasemnogene abeparvovec-xioi), approved for spinal muscular atrophy (SMA).
Products and services
- Zolgensma (onasemnogene abeparvovec) A one-time intravenous AAV gene therapy that delivers a functional copy of the SMN1 gene to address the genetic root cause of spinal muscular atrophy (SMA). Zolgensma is AveXis's flagship product, priced at approximately $2.125 million per patient, and is administered in clinical settings to infants and children with SMA.
Quantifiable outcome
- Zolgensma achieves FDA approval and has become standard of care for SMA treatment globally
Companies that use AveXis
Customer profileNamed customers1 record
Segments2 records
Ideal customer profiles2 records
AveXis technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature2 records
AveXis partnerships and signals
Strategic signalPartnerships
Four partnerships are on record, tiered core and minor.
- Kate TherapeuticscoreNovartis acquired Kate Therapeutics in 2024 to expand its gene therapy and xRNA portfolio. The company brought additional delivery technologies and programs targeting muscle, heart, and CNS tissues.
- Regulus TherapeuticsminorNovartis expanded its San Diego footprint through the acquisition of Regulus Therapeutics, strengthening capabilities in gene therapy and xRNA research.
- DTx PharmacoreNovartis acquired DTx Pharma in 2023 to strengthen its xRNA capabilities. The acquisition brought additional expertise and programs to the consolidated San Diego genetic medicine research team.
- Novartis (acquirer)coreNovartis acquired AveXis in 2018 for its gene therapy capabilities, integrating the company's AAV technology platform and Zolgensma product into Novartis Gene Therapies. The acquisition brought programs and expertise targeting neuromuscular and neurodegenerative diseases.
Scale indicators4 records
Recent moves6 records
Expansion highlights5 records
AveXis competitors and assessment
Company assessmentDirect peers
- Sarepta Therapeutics: Sarepta is the most directly comparable AAV gene therapy company, with FDA-approved Elevidys for Duchenne muscular dystrophy (DMD) and an AAV platform targeting neuromuscular disease — directly overlapping AveXis's neuromuscular/CNS AAV gene therapy focus and competing for the same specialized treatment-center infrastructure.
- BioMarin Pharmaceutical: BioMarin is a rare-disease specialist with an AAV gene therapy (Roctavian for hemophilia A) and a deep pipeline of high-cost one-time treatments, directly comparable to AveXis/Novartis on commercial model, rare-disease commercial infrastructure, and AAV gene therapy manufacturing.
- Regenxbio: Regenxbio develops AAV-based gene therapies (e.g., ABECMA-derivative programs, ABTYS) using proprietary NAV capsid technology — a direct AAV platform competitor to AveXis, sharing the same delivery-technology category and serving overlapping rare-disease indications.
- uniQure: uniQure is a clinical-stage AAV gene therapy company developing treatments for CNS, cardiovascular, and metabolic rare diseases (e.g., AMT-130 for Huntington's). Its AAV platform and neuromuscular/neurodegenerative focus overlap directly with AveXis's pipeline breadth beyond SMA.
- Voyager Therapeutics: Voyager is an AAV gene therapy company developing treatments for CNS and neurodegenerative diseases (e.g., Alzheimer's, Parkinson's, tauopathies). Its CNS-focused AAV platform directly competes with AveXis's CNS capsid engineering programs.
Broad incumbents
- Biogen: Biogen markets Spinraza, the leading antisense oligonucleotide treatment for SMA and Zolgensma's primary competitor in the SMA market. While not an AAV platform peer, Biogen directly contests AveXis's core commercial franchise and operates an overlapping rare/neurological disease commercial infrastructure.
- Roche (Genentech): Roche markets Evrysdi (risdiplam), the leading oral SMA therapy and Zolgensma's principal competitive threat. Roche also owns Spark Therapeutics (Luxturna), giving it breadth across both small-molecule and AAV gene therapy modalities for rare diseases.
- Pfizer: Pfizer invested aggressively in AAV gene therapy for DMD and hemophilia, and despite setbacks remains a broad incumbent competitor in the rare-disease gene therapy space with overlapping commercial and manufacturing capabilities.
Emerging players
- Solid Biosciences: Solid Biosciences is a clinical-stage AAV gene therapy company focused on DMD and other rare neuromuscular diseases. While smaller and narrower in indication, its AAV platform and neuromuscular focus overlap with AveXis's core technology.
- Taysha Gene Therapies: Taysha is a clinical-stage AAV gene therapy company developing treatments for CNS and rare neurological diseases, often in partnership with larger pharma. Its AAV platform and CNS focus overlap directly with AveXis's neurodegenerative pipeline direction.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat5 records
Key risks5 records
Key highlights7 records
Customer concentration
AveXis social profiles
Digital presenceAveXis financial estimates
Financial estimateRevenue estimate
Valuation estimate
AveXis leadership team
Management profileNumber of profiles
Profiles4 records
AveXis funding detail
Funding detailFunding overview
Funding rounds5 records
Investors13 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
AveXis M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about AveXis
What does AveXis do?
AveXis develops and manufactures AAV-based gene therapies for patients with rare and life-threatening neurological genetic diseases. Its flagship product Zolgensma (onasemnogene abeparvovec) is an FDA-approved one-time intravenous gene therapy that delivers a functional copy of the SMN1 gene to treat spinal muscular atrophy (SMA). Following its 2018 acquisition by Novartis, AveXis now operates as part of Novartis Gene Therapies, with its pipeline and technology consolidated under Novartis's San Diego genetic medicine research operations.
Is AveXis a public or private company?
AveXis is a private company. It is classified as corporate owned and is currently acquired.
When was AveXis founded?
AveXis was founded in 2013. It employs 11 to 50 people.
Where is AveXis based?
AveXis is headquartered in Dallas, United States, in the North America region.
How does AveXis make money?
Two revenue lines are on record. Gene Therapy Product Sales are the primary driver. The others are outcomes-Based Payment Contracts.
Who are AveXis's main competitors?
Direct peers on record are Sarepta Therapeutics, BioMarin Pharmaceutical, Regenxbio, uniQure and Voyager Therapeutics. Broad incumbents are Biogen, Roche (Genentech) and Pfizer. Emerging players are Solid Biosciences and Taysha Gene Therapies.
Does AveXis have an API?
No public API is recorded for AveXis.
What industry is AveXis in?
AveXis's product category is Gene Therapy Biopharmaceuticals. Its primary akta.pro industry code is HLAAACAA, AAV Vector Gene Therapy Developers, with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 2834.