Key Patent Granted For Aavlife’s Gene-Therapy Program To Treat Cardiomyopathy In Friedreich’s Ataxia

AVLife SAS, a company developing gene-therapy products, today announces the grant of a key United States patent for a therapy under development at AAVLife for the treatment of cardiomyopathy associated with the genetic disorder known as Friedreich’s ataxia.

“As this recently issued patent indicates, AAVLife is building strong intellectual-property protection at the same time we are steadily progressing our R&D toward an urgently needed therapy”

The patent broadly protects a promising method for treating cardiomyopathy by using an adeno-associated virus (AAV) vector to carry into cells a gene expressing the protein frataxin. The patent will run until 2033 or longer in the event of a successful application for an extension. Corresponding patent applications are pending in major markets globally.

The patent protects the commercial potential of AAVLife’s lead development program, which focuses on Friedreich’s ataxia. This disorder is caused by a genetic mutation resulting in low expression of frataxin, a protein essential to the normal function of mitochondria and their role in cellular metabolism. Friedreich’s ataxia is characterized by the loss of full control of bodily movements. However, most deaths due to the disorder result from progressive degradation of cardiac function.

AAVLife holds an exclusive worldwide license to the patent (US 9,066,966 as issued by the United States Patent and Trademark Office) under an agreement with Inserm Transfert, the technology-transfer arm of Institut National de la Sante et de la Recherche Medicale (Inserm).*

“As this recently issued patent indicates, AAVLife is building strong intellectual-property protection at the same time we are steadily progressing our R&D toward an urgently needed therapy,” said Amber Salzman, Chief Executive Officer of AAVLife.

Published research in an animal model of Friedreich’s ataxia demonstrated that delivery of a normal frataxin gene by means of an AAV vector restored cardiac function and reversed pathological heart enlargement.** In addition to continuing preclinical research, AAVLife is conducting observational studies in Friedreich’s ataxia patients to determine appropriate efficacy measures for use in an anticipated clinical trial.

Friedreich’s ataxia affects 10,000 to 20,000 people in the United States and Europe. There are no approved therapies.

About AAVlife

AAVLife, headquartered in Paris, is a privately held company dedicated to advancing gene therapy for rare diseases. It is funded by Versant Ventures, an international life-sciences venture-capital firm, and Inserm Transfert Initiative, a private venture-capital subsidiary of Inserm. Further information is available at www.aavlife.com.

*The patent holders are Inserm, Centre de la Recherche Scientifique, Université de Strasbourg, Cornell University, Université Paris-Sud XI, and Assistance Publique-Hopitaux de Paris.

**Perdomini M, Belbellaa B, Monassier L, Reutenauer L, Messaddeq N, Cartier N, Crystal RG, Aubourg P, Puccio H. Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich’s ataxia.Nature Medicine, 20, 542–547 (2014) doi:10.1038/nm.3510.