Date: 2015-09-01
Type of information: Company acquisition
Acquired company: Cardiogen Sciences (USA - CA)
Acquiring company: Audentes Therapeutics (USA - CA)
Amount: undisclosed
Terms: * On September 1, 2015, Audentes Therapeutics, a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, 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.
Details: 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. 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.
Related: Gene therapy Rare diseases Cardiovascular diseases
