Audentes Expands Portfolio to Include AAV Gene Therapy for Rare, Genetic Cardiac Diseases including CASQ2-CPVT, an Inherited Arrhythmia with a High Risk of Mortality

SAN FRANCISCO, CA, USA I September 1, 2015 I Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced the acquisition of Cardiogen Sciences, Inc., a biotechnology company focused on the discovery and development of adeno-associated virus (AAV) gene therapy products for rare, inherited arrhythmogenic diseases.

Audentes plans to rapidly advance Cardiogen’s lead program for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT) into clinical development. The new product candidate, designated AT003, expands Audentes’ portfolio of investigational AAV gene therapy treatments for rare diseases with significant unmet medical needs, which also includes AT001 for X-Linked Myotubular Myopathy (XLMTM) and AT002 for Pompe Disease. In addition to the CASQ2-CPVT program, the acquisition includes rights to develop and commercialize AAV gene therapy products for several additional inherited arrhythmias.

Cardiogen was founded in 2014 by Louis G. Lange, MD, PhD to translate the scientific discoveries of Dr. Silvia Priori, MD, PhD and her team at the Fondazione Salvatore Maugeri (FSM) in Pavia, Italy into the development of therapeutics to address unmet medical need in inherited arrhythmias. Dr. Lange was a founder of CV Therapeutics and as Chairman, CEO and CSO, led the company through its IPO to its acquisition in 2009. Dr. Priori is the Scientific Director of the FSM, a Professor of Cardiology at the University of Pavia, and is past President of the European Heart Rhythm Association. In conjunction with the acquisition, Dr. Lange has joined the Audentes Therapeutics Board of Directors and Dr. Priori will serve as a Scientific Advisor to Audentes.

“We are thrilled to combine Cardiogen’s innovative programs with our resources and passionate team to advance AAV gene therapy for patients with inherited cardiac arrhythmias,” said Matthew Patterson, President and CEO of Audentes. “Expanding our portfolio through this acquisition aligns strongly with our strategy of developing AAV gene therapies for serious, rare diseases that have compelling pre-clinical proof-of-concept data and the potential for accelerated clinical development. In addition, we are very excited to have Drs. Lange and Priori partnering with the Audentes team as their world class expertise in inherited cardiac diseases and cardiac drug development will be invaluable in helping Audentes rapidly advance our new programs.”

“Patients with inherited cardiac arrhythmias, many of whom are children, currently lack effective, specific treatments for CASQ2-CPVT, frequently resulting in significant morbidity, poor quality of life and, sadly, early mortality,” stated Louis G. Lange, MD, PhD, former Chairman of Cardiogen. “Our mission at Cardiogen has been to eradicate life threatening arrhythmias through selective cardiac gene therapy, and we believe that we will have the best opportunity to fulfill this mission through the combined efforts of the Audentes and Cardiogen teams.”

CASQ2-CPVT is an inherited disease caused by mutations in the CASQ2 gene. CASQ2 encodes a protein called calsequestrin 2, which plays a key role in the physiology of calcium release in cardiac muscle cells, and which is required to maintain normal heart rhythm. CPVT is characterized by abnormal ventricular heartbeats (arrhythmias) precipitated by exercise or stress that can cause dizziness and fainting and may progress to cardiac arrest and sudden death. It is estimated that more than 6,000 people globally have CASQ2-CPVT, although the disease is believed to be under-diagnosed. Currently available anti-arrhythmia treatments are considered inadequate for CASQ2-CPVT and there are no therapies currently approved specifically for the treatment of this disease.

Scientific research has demonstrated that a single administration of AAV-CASQ2 restored calsequestrin 2 protein levels and prevented CASQ2-CPVT-associated arrhythmias in a mouse model of the disease (Denegri et. al., Circulation, 2014). Similar results were observed in both newborn and adult CASQ2-CPVT mice.

About Audentes Therapeutics, Inc.

Audentes is a biotechnology company committed to the development and commercialization of innovative new treatments for people with serious, rare diseases through the application of AAV gene therapy technology. We have three products in development, AT001 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT002 for the treatment of Pompe disease, and AT003 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT). Our team consists of a focused, experienced, and passionate individuals committed to forging strong, global relationships with the patient, research, and medical communities. For more information regarding Audentes, please visit us at

SOURCE: Audentes Therapeutics