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Audentes Therapeutics Announces Commencement of LUSTRO, a Clinical Assessment and Phase 1 / 2 Run-in Study of Patients with Crigler-Najjar Syndrome
- LUSTRO intended to serve as a longitudinal baseline and within-patient control for VALENS, the planned Phase 1 / 2 study of AT342 for the treatment of Crigler-Najjar Syndrome

SAN FRANCISCO, March 1, 2017 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced the commencement of LUSTRO, a prospective study designed to characterize the disease course in patients living with Crigler-Najjar Syndrome.  LUSTRO will evaluate subjects prior to potential enrollment in VALENS, the planned Phase 1 / 2 clinical study to evaluate the safety and preliminary efficacy of AT342, the Audentes product candidate being developed to treat Crigler-Najjar Syndrome.

"We are pleased to announce the commencement of LUSTRO," stated Dr. Suyash Prasad, Senior Vice President and Chief Medical Officer of Audentes Therapeutics. "LUSTRO is designed to further our understanding of the baseline characteristics of patients living with Crigler-Najjar Syndrome, and the burden of disease as experienced by patients, families and caregivers.  We greatly appreciate the opportunity to work with the Crigler-Najjar community and view the initiation of LUSTRO as an important milestone as we seek to develop an innovative gene therapy for this devastating rare disease."

LUSTRO is an international study of approximately 16 to 18 Crigler-Najjar patients one year of age or older.  The primary objectives of LUSTRO are to characterize the disease course, natural history, serum bilirubin variability and phototherapy usage of Crigler-Najjar patients.  LUSTRO will also assess the burden of disease on Crigler-Najjar patients and caregivers.  In addition, LUSTRO has been designed as a run-in study to identify patients for potential enrollment in VALENS, the planned Phase 1 / 2 study of AT342, and is intended to serve as a longitudinal baseline and within-patient control for subjects who enroll in VALENS.  As LUSTRO will be conducted at the same sites and using the same assessment tools, LUSTRO will also facilitate certain operational aspects of VALENS.

The Investigational New Drug application (IND) for AT342 is active.  Audentes plans to report preliminary data from VALENS in the fourth quarter of 2017.

About AT342 to treat Crigler-Najjar Syndrome   
AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene for the treatment of Crigler-Najjar Syndrome, a rare monogenic disease characterized by severely high levels of unconjugated bilirubin in the blood and risk of irreversible neurological damage and death.  The current standard of care for Crigler-Najjar Syndrome is daily, persistent phototherapy, usually for longer than 12 hours per day. Phototherapy wanes in effectiveness as children age, and a liver transplant may be required for survival.  A single administration of AT342 has generated durable, dose-responsive and clinically-relevant decreases in total bilirubin levels in a mouse model of Crigler-Najjar. 

About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases.  We have four products in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT).  We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.

For more information regarding Audentes, please visit

Forward Looking Statements
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: our ability to enroll 12 to 16 patients into LUSTRO, whether LUSTRO will be able to meet its primary objectives, including whether it may be used as a longitudinal baseline and within patient control for VALENS, the ability of patients enrolled in LUSTRO to enroll in VALENS, the planned reporting or preliminary data from VALENS in the fourth quarter of 2017, and whether Audentes can successfully develop AT342 to treat Crigler-Najjar Syndrome.  All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercial its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other  risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Audentes Contacts:

Investor Contact:
Thomas Soloway, CFO

Media Contact:
Jeffrey Gruis



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