Audentes Therapeutics

What Audentes Therapeutics does

Audentes Therapeutics is a San Francisco-based biotechnology company founded in 2013 that develops adeno-associated virus (AAV) gene therapies for rare neuromuscular diseases. Its core technology platform centers on AAV-mediated delivery of functional gene payloads to skeletal muscle, with a primary focus on X-linked myotubular myopathy (XLMTM), an ultra-rare genetic disorder affecting approximately 1 in 50,000 males that causes severe muscle weakness, respiratory failure, and reduced life expectancy. The company's lead clinical asset, AT132 (an AAV8 vector delivering the MTM1 gene), advanced into the ASPIRO trial before being placed on FDA clinical hold following four patient deaths from liver failure, leading to program termination and a ~$103 million impairment charge.

In January 2019, Astellas Pharma acquired Audentes for approximately $3 billion, making it a wholly-owned subsidiary operating under Astellas Gene Therapies. Following the AT132 setback, Audentes pivoted its XLMTM program by licensing ASP2957 (MyoAAV3.8-MHCK7-hMTM1) from Kate Therapeutics in 2023, a next-generation construct using a proprietary MyoAAV capsid designed for enhanced muscle tropism at approximately 100-fold lower doses to address the prior liver toxicity profile. The company is also pursuing broader capsid engineering collaborations with Sangamo Therapeutics and Dyno Therapeutics to expand its AAV platform capabilities.

As a clinical-stage subsidiary, Audentes generates no commercial product revenue; its economics flow through Astellas' consolidated R&D and impairment reporting. The company's go-to-market model relies on Astellas' hospital pharmacy and specialty treatment center distribution infrastructure for eventual commercialization of AAV gene therapies, with pricing expected to follow the seven-figure list price convention typical of gene therapies though no specific pricing has been publicly disclosed for AT132 or ASP2957.

Name

Audentes Therapeutics

Website

https://audentestx.com

Company type

Private

Founded year

2013

Operating status

Acquired

Headcount range

251–500 employees

Short description

Audentes Therapeutics is a San Francisco-based gene therapy company developing AAV-based treatments for rare neuromuscular diseases, notably X-linked myotubular myopathy. Acquired by Astellas Pharma for $3 billion in 2019, it now operates as Astellas Gene Therapies and is advancing next-generation capsid-based candidates.

Ownership category

akta.pro rank

Product category

Gene Therapy Biologics

NAICS

Other Scientific and Technical Consulting Services (54169)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Health Services (8000)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

Neurology/CNS Gene Therapies (HLAAACAH), Gene Therapy Regulatory, Pharmacovigilance & Clinical Operations Services (HLAAACAM)

Keywords

• Gene therapy development
• Rare neuromuscular diseases
• AAV vector platform
• X-linked myotubular myopathy
• Biologics manufacturing

Headquarters

HQ city

San Francisco

HQ country

United States

HQ region

North America

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Supply Chain

Core offering

Audentes Therapeutics is a clinical-stage biotechnology company that develops AAV (adeno-associated virus) gene therapies for rare neuromuscular diseases, with a primary focus on X-linked myotubular myopathy (XLMTM). The company's pipeline centers on delivering functional copies of disease-causing genes to affected muscle tissue, including the first-generation AT132 program and the next-generation ASP2957 candidate that uses proprietary MyoAAV capsid technology licensed from Kate Therapeutics.

Product overview

Audentes Therapeutics, acquired by Astellas Pharma in 2019 for $3 billion, operated as a gene therapy company focused on rare neuromuscular diseases. The company's lead product AT132 (an AAV8-based gene therapy for XLMTM) was discontinued due to safety concerns. Astellas has since transitioned to ASP2957, a next-generation gene therapy using MyoAAV capsid technology licensed from Kate Therapeutics, which demonstrates improved safety profile through lower dosing requirements. The pipeline also included ASP5502 and ASP1570, which were terminated along with AT132.

Differentiator

Problem solved

Functional benefit

Products and services

• AT132 AAV8-based gene therapy delivering a functional copy of the MTM1 gene to muscle tissue for the treatment of X-linked myotubular myopathy (XLMTM). Development was discontinued following patient deaths linked to liver failure.
• ASP2957 (MyoAAV3.8-MHCK7-hMTM1) Next-generation AAV gene therapy for X-linked myotubular myopathy using proprietary MyoAAV capsid technology licensed from Kate Therapeutics. Demonstrates improved muscle targeting at approximately 100-fold lower doses than AT132 to mitigate liver toxicity concerns.

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Three partnerships are on record, tiered core and supporting.

• Kate TherapeuticscoreOEM/ Whitelabel/ Licensing Partner · 1 January 2023Licensed ASP2957 (MyoAAV3.8-MHCK7-hMTM1), a next-generation gene therapy for XLMTM using proprietary MyoAAV capsid technology. Kate Therapeutics was subsequently acquired by Novartis in 2024. The MyoAAV capsid demonstrates improved muscle targeting at approximately 100-fold lower doses than the original AT132 therapy, addressing liver toxicity concerns identified in prior clinical trials.
• Sangamo TherapeuticssupportingTechnology or IntegrationContinuing partnership for next-generation gene therapy capsid development as part of Astellas' broader AAV platform strategy. Sangamo contributes zinc finger protein engineering capabilities for capsid optimization.
• Dyno TherapeuticssupportingTechnology or IntegrationOngoing collaboration for next-generation AAV capsid development leveraging Dyno's AI-powered capsid engineering platform to enhance tissue targeting and reduce immunogenicity.

Scale indicators2 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Sarepta Therapeutics: Commercial-stage AAV gene therapy company targeting Duchenne muscular dystrophy and other neuromuscular indications. Direct peer to Audentes given parallel AAV delivery to skeletal muscle and identical regulatory pathway for rare pediatric myopathies.
• Solid Biosciences: Clinical-stage AAV gene therapy developer for Duchenne muscular dystrophy using microdystrophin delivery to skeletal muscle. Closely comparable technology platform, indication focus, and development stage to Audentes.
• Regenxbio: AAV gene therapy platform company developing treatments for rare diseases including neuromuscular and retinal disorders. Comparable AAV vector technology and rare-disease focus, with broader pipeline diversification.
• uniQure: Clinical-stage AAV gene therapy company developing treatments for rare diseases including neuromuscular and CNS indications. Directly comparable AAV platform, rare-disease positioning, and clinical development stage.
• Voyager Therapeutics: AAV gene therapy company focused on CNS and neurological diseases including severe neuromuscular conditions. Comparable AAV platform with emphasis on CNS tropism, similar to Audentes' muscle-targeted approach.
• PTC Therapeutics: Biotech developing treatments for rare diseases including Duchenne muscular dystrophy, with both small-molecule and gene therapy programs. Comparable neuromuscular rare-disease focus and competing therapeutic modalities.

Broad incumbents

• Novartis: Owns Zolgensma (AAV gene therapy for SMA) and acquired Kate Therapeutics, Audentes' MyoAAV capsid licensor. Direct strategic overlap with Audentes through Kate and through Zolgensma's commercial precedent in AAV neuromuscular therapy.
• Pfizer: Major pharma with active AAV gene therapy programs including Duchenne muscular dystrophy candidate. Broad incumbent with overlapping neuromuscular gene therapy interest, deeper resources, and competing programs.
• BioMarin Pharmaceutical: Established rare-disease biotech with AAV gene therapy programs (hemophilia A) leveraging similar regulatory pathway and rare-disease commercialization model. Adjacent platform with comparable ultra-rare indication strategy.

Emerging players

• Taysha Gene Therapies: Clinical-stage AAV gene therapy company focused on CNS and rare neurological diseases including neuromuscular indications. Emerging peer with partial overlap in AAV delivery to muscle/CNS and rare-disease strategy.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights5 records

Customer concentration

• Xx.com/audentestx
• Facebookfacebook.com/audentestx
• LinkedInlinkedin.com/company/audentes-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds8 records

Investors16 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Audentes Therapeutics