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uniQure to Acquire Corlieve Therapeutics

Source: press release

uniQure N.V.(NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced it has entered into a definitive agreement to acquire Corlieve Therapeutics and its lead program, which will be known as AMT-260, to treat temporal lobe epilepsy, the most common form of focal epilepsy.

Corlieve’s lead gene therapy program employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (TLE). TLE affects approximately 1.3 million people in the U.S. and Europe alone, of which approximately 800,000patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life.

Valérie Crépel and Christophe Mulle

AMT-260 was originally developed by Corlieve in collaboration with Christophe Mulle, Ph.D., CNRS Research Director, at the Interdisciplinary Institute of Neurosciences, CNRS, University of Bordeaux, Valerie Crepel, Ph.D., Inserm Research Director at the Institut de Neurobiologie de la Méditerranée, INSERM, Aix-Marseille University, and REGENXBIO Inc. Drs. Mulle and Crepel will continue their collaborations with uniQure.

“The acquisition of Corlieve provides an extraordinary opportunity to transform the lives of hundreds of thousands of patients around the world suffering from epilepsy and aligns with our vision of pursuing unmet medical needs for disorders that impact large populations and can be addressed with gene therapies directed to the CNS and liver,” stated Matt Kapusta, Chief Executive Officer of uniQure. “The groundbreaking work of the Corlieve team, in collaboration with Drs. Mulle and Crepel, has led to compelling preclinical results in temporal lobe epilepsy that we believe can strategically leverage uniQure’s leading position in developing and delivering gene therapies that employ miRNA silencing technology.We look forward to welcoming the Corlieve team into the uniQure family as we join forces to advance this important andpotentially transformative therapy into clinical studies.

I am very proud of what Corlieve has achieved in such a short amount of time. With the dedication and focus of our teamand our collaboration partners, we have taken a promising therapeutic approach discovered by our scientific founders and created a potential transformative therapeutic opportunity for patients with refractory TLE,” said Richard Porter, Ph.D., founder and Chief Executive Officer of Corlieve. “As leaders in the field for miRNA gene therapy for neurological conditions,uniQure is the ideal long-term partner for us, and we look forward to working together to advance our program rapidly to the clinic for the benefit of the patients we serve.

About Corlieve Therapeutics

Corlieve Therapeutics is a biotechnology company focused on bringing novel therapeutic options to patients with severe neurological disorders. The lead project is targeting aberrantly expressed kainate receptors in the hippocampus of patients with refractory TLE using a gene therapy approach. Corlieve was founded by Kurma Partners 4Q2019 on the basis of a partnership with REGENXBIO Inc., SATT Aquitaine Science Transfert, and Inserm Transfert. Corlieve is supported by its investors and partners Kurma Partners, Eurazeo, Pureos Bioventures, SATT Aquitaine Science Transfert, Inserm Transfert, Inserm CNRS, and REGENXBIO.

www.corlieve.com

About  uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipelineof proprietary gene therapies to treat patients with hemophilia B, Huntington’s disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases

www.uniQure.com

01/07/21