Audentes Therapeutics said today it has acquired adeno-associated virus (AAV) gene therapy developer Cardiogen Sciences for an undisclosed price.

The deal will expand Audentes’ portfolio by adding to it a third preclinical program—Cardiogen’s lead program for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT). No therapies have yet been approved for treating CASQ2-CPVT, an inherited disease caused by mutations in the CASQ2 gene.

Audentes said it plans to “rapidly” advance into clinical development the CASQ2-CPVT program, which is centered on a new product renamed AT003, without specifying a timeframe.

The company cited mouse model research showing 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. Similar results were seen in both newborn and adult CASQ2-CPVT mice, Audentes said.

In addition to the CASQ2-CPVT program, the acquisition includes rights to develop and commercialize AAV gene therapy products for several additional inherited arrhythmias.

AT003 will expand Audentes’ portfolio of investigational AAV gene therapy treatments for rare diseases with significant unmet medical needs—a portfolio that includes AT001 for X-linked myotubular myopathy (XLMTM); and AT002 for Pompe disease.

“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,” Audentes president and CEO Matthew Patterson said in a statement.

Cardiogen specializes in developing AAV gene therapies for rare, inherited arrhythmogenic diseases. Cardiogen was founded last year by Louis G. Lange, M.D., Ph.D., to translate the scientific discoveries of Silvia Priori, M.D., Ph.D., 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.

As Chairman, CEO, and CSO, Dr. Lange 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.

Dr. Lange, a founder of CV Therapeutics, has joined Audentes’ board of directors, while Dr. Priori will serve as a scientific advisor to Audentes.

“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,” Dr. Lange stated.

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