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Audentes Therapeutics, an Astellas company, is a biotechnology company that was built around the robust data in two animal models for gene transfer therapy for XLMTM and Intellectual Property developed by the Joshua Frase Foundation after pitching Matt Patterson, former CEO in 2012. The company is focused on the development and commercialization of innovative new gene therapy treatments for people with serious rare diseases. Audentes Therapeutics is developing AT132 for the treament of X-Linked Myotubular Myopathy. This company is based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina.


Below you will find press releases and annoucments made for the Patient community by Audentes Therapeutics surrounding ASPIRO, the Clinical Trial evaluating AT132 in Patients with X-Linked Myotubular Myopathy (XLMTM).  


Statement from Audentes ~ August 20, 2020

 Audentes Therapeutics Provides Update on the ASPIRO Clinical Trial Evaluating AT132 in Patients with X-linked Myotubular Myopathy San Francisco, Calif., August 20, 2020 /BUSINESSWIRE/ -- Audentes Therapeutics, Inc., an Astellas company, today announced that a third study patient has passed away in the ASPIRO clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy (XLMTM), which is a serious, lifethreatening neuromuscular disease characterized by extreme muscle weakness, respiratory failure, and early death. Preliminary findings indicate that the immediate cause of death was gastrointestinal bleeding. This patient was one of three study patients previously disclosed to have received AT132 at the dose of 3x1014 vg/kg who began to demonstrate signs of liver dysfunction within 3 to 4 weeks after dosing. All three patients demonstrated evidence of pre-existing hepatobiliary disease. More than 50% of patients enrolled in the ASPIRO trial to date show evidence of pre-existing hepatobiliary disease, but it has not been associated with similar progressive liver dysfunction in any of the patients who received AT132 at the 1x1014 vg/kg dose nor in the other patients who received the 3x1014 vg/kg dose. To date, 23 ASPIRO patients have received AT132: six at the 1x1014 vg/kg dose and 17 at the 3x1014 vg/kg dose. Although the ASPIRO study is currently on clinical hold, there are no other patients in the study known to be currently experiencing similar liver dysfunction. Audentes, together with the ASPIRO investigators and independent Data Monitoring Committee, continue to closely monitor all patients enrolled in the study. Additionally, Audentes’ investigation regarding why these three patients developed progressive liver dysfunction is ongoing. Audentes extends its deepest sympathies to this patient’s family; the company remains committed to the AT132 development program and the XLMTM patient community. The company plans to provide further information on the ASPIRO program based on both ongoing data collection and future regulatory status updates.  

Statement from Audentes ~ June 23rd, 2020 

Dear XLMTM Patient Community,

 A second patient in the ASPIRO clinical trial investigating AT132 (the investigational gene therapy product candidate) for X-linked myotubular myopathy (XLMTM) has tragically passed away. We are deeply saddened by this loss and our hearts go out to the family.

Here is what we can share at this time:

  • Preliminary findings indicate that the immediate cause of death was sepsis. This patient had progressive liver dysfunction characterized by hyperbilirubinemia that occurred within the first 4-6 weeks following AT132 dosing, and which did not respond to standard treatment. This patient received aggressive medical treatment for his liver dysfunction, including hospitalization and close clinical observation. Unfortunately his condition worsened, and he ultimately succumbed to bacterial infection and sepsis. Additional information surrounding the death is being collected at this time.
  • As was the case with the patient death we shared last month, this patient was also one of three older patients who received AT132 at the dose of 3x1014 vg/kg in whom we have recently observed new serious adverse events (SAEs) of hepatobiliary disease. Although the investigations around both deaths are ongoing, preliminary reports indicate that the clinical course was similar in the two patients who passed away.
  • Notable features among the three patients with these SAEs include older age, heavier weight, evidence of pre- existing hepatobiliary disease, and dosing with the higher dose of 3x1014 vg/kg. It should be noted that among the six patients treated at 1x1014 vg/kg, including four with a previous history of hepatobiliary disease, none have developed liver SAEs, despite being years out from treatment.

Next steps:

Our ongoing investigation includes, among other potential factors, an examination of whether the factors described above contributed to these SAEs. The investigation of these cases in the ASPIRO clinical trial continues to be ongoing.

 We continue to be in regular contact with the ASPIRO clinical investigators and the ASPIRO Data Monitoring Committee (DMC) as new information becomes available. Regulatory authorities are being notified via their standard reporting procedures.

 Prior to both of these deaths, Audentes, in consultation with the DMC, had halted further dosing of patients currently enrolled in the clinical trial. Subsequently, following interactions with the FDA (US Food and Drug Administration), the study was put on formal clinical hold. Per FDA’s guidance, we continue to follow and actively monitor those patients who are enrolled in the trial.

 We are taking all necessary steps to understand these events and incorporate what we learn into our development plan going forward. We are currently assessing the impact on potential regulatory filing timelines, however we will not be filing in mid-2020 as previously communicated.

 We will continue to provide immediate updates on any significant developments related to patient safety in the AT132 development program, regardless of the status of the investigation or clinical trial. We will continue to provide “data cut” updates at appropriate scientific meetings as the progress of ASPIRO and the COVID-19 related delays in data collection allow.

Contact information

 Important note

It is important to understand that regulatory agencies have not approved the Audentes investigational gene therapy product candidate (AT132) or determined that it is safe or effective, as it is still undergoing formal assessment in clinical trials. The investigational gene therapy product candidate is not approved for commercial sale and is only available in clinical trial settings.

We remain committed to developing AT132 for the families and patients living with XLMTM.


Natalie Holles, President and Chief Executive Officer and 

Edward Conner, M.D., Senior Vice President and Chief Medical Officer

Statement from Audentes ~ MAY 6TH, 2020

 Yesterday morning we learned that a patient in the ASPIRO clinical trial investigating AT132 for X-linked myotubular myopathy (XLMTM) tragically passed away. We extend our deepest sympathies to the family during this time of immeasurable loss and sorrow.

 Preliminary findings indicate that the patient passed away as a result of sepsis, and a full investigation is being conducted to determine the extent to which AT132 may have contributed to this event.  The company has informed all relevant study investigators and regulatory authorities about this event.

 This patient was one of three older patients treated with AT132 at the dose of 3 X 1014 vg/kg in which we have recently observed new serious adverse events. Together with the ASPIRO clinical investigators, we are closely monitoring these patients to understand these adverse events as they continue to evolve.

We will provide relevant updates as appropriate. Patient safety continues to be our highest priority and our commitment to advancing the development of AT132 for XLMTM remains unchanged.

 Kimberly Trant RN, MBA

Senior Director, Patient Advocacy and Engagement

Audentes Therapeutics, an Astellas company

600 California Street, 17th Floor I San Francisco, CA I 94108

Office: (415) 805.1049


 August 26, 2019

May 1st, 2019 

January 31, 2019

November 6, 2018

October 5, 2018


August 7th, 2018


May 16th, 2018 

 “It is important to note we cannot make any conclusions on the interim findings of the clinical trial until after all enrolled subjects are dosed and evaluated for the duration of the study, and the full scope of data is collected and analysed making conclusions about interim data may not accurately predict the  full risk/benefit profile of an investigational product after all enrolled subjects are dosed and evaluated for the duration of the study, and the full scope of data is collected and analyzed, more complete information about safety and efficacy of this investigational gene therapy product will become available to the community.” released Audentes 

 At this phase of the clinical trial many in our community are asking the question about Audentes' approach to Expanded Access to this investigational product. Please read here


Jan 4th, 2018


March 20th, 2017  


 April 3rd, 2017  

AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), an ultra-rare, severe, debilitating disease that affects skeletal muscles in an estimated one in 50,000 newborn males worldwide, and is caused by mutations in the MTM1 gene


 What is INCEPTUS? What is ASPIRO?   

Both INCEPTUS and ASPIRO are clinical studies that are being or will be conducted by Audentes Therapeutics. The Audentes gene therapy product for XLMTM is called AT132. INCEPTUS and ASPIRO form part of the clinical development program for AT132. The overall aim of the clinical development program is to evaluate if AT132 is safe and effective in XLMTM, and to get the drug approved quickly in order to benefit patients with XLMTM as rapidly as possible. Audentes will collect the necessary data in the clinical studies and then, if the outcomes from the clinical studies are positive, file an application for potential approval to sell AT132 commercially with regulatory agencies around the world (e.g. FDA in the United States, and EMA in Europe).  


Audentes Therapeutics has initiated its first study of X-Linked Myotubular Myopathy (XLMTM), entitled RECENSUS: A Medical Chart Review of Patients with XLMTM.  RECENSUS is a Latin word which means to review and investigate. The goal of this study is to collect information from medical charts in order to characterize aspects of the condition and associated medical care for males with XLMTM.

 This is a retrospective, non-interventional study, meaning there is a review of past care. It does not require visits to a study site or any medical procedures.

We hope that this study will contribute considerably to the understanding of XLMTM. If you are interested in having your or your child’s medical chart included in the study, please follow the link below for more information about the study and participating sites:  

Clinical Trials RECENSUS study 

 Those patients enrolled in the Begg’s registry do NOT need to do anything else. Their data is already being included in the study. Participation in this study does not influence the selection of patients for the clinical trial.

For any questions, please contact

Kimberly Trant RN, MBA
Director, Patient Advocacy
Audentes Therapeutics, Inc.
600 California Street, 17th Floor I San Francisco, CA I 94108
Office: (415) 805.1049