LYSOGENE, UMM and AU Collaborate To Develop IND-supporting Preclinical Studies In GM1-gangliosidosis

Collaboration entails development of gene therapy treatment for severe neurodegenerative disease GM1-gangliosidosis

LYSOGENE, a leading, clinical stage gene therapy biotechnology company committed to the development and commercialization of breakthrough treatments for severe orphan pathologies affecting the central nervous system (CNS), recently announced that it has entered into a strategic collaboration with the University of Massachusetts Medical School (UMMS) in Worcester, Massachusetts, and Auburn University (AU) in Auburn, Alabama. Through the collaboration, LYSOGENE, UMMS and AU will develop IND-supporting preclinical studies in GM1-gangliosidosis, a rare, inherited disorder characterized by severe neurological impairment, using adeno-associated virus (AAV) gene therapy technology.

The collaboration will combine LYSOGENE’s outstanding translational and clinical expertise in gene therapy for CNS disorders with the unique preclinical expertise and infrastructure of UMMS and AU to design and test innovative AAV-based gene therapy approaches to treat GM1-gangliosidosis.

The development of a potential treatment for GM1-gangliosidosis using AAV gene therapy was initiated in 2005 by Miguel Sena-Esteves, PhD, associate professor in the Neurology Department and the Gene Therapy Center at UMMS, and Douglas R. Martin, PhD, associate professor in the Scott-Ritchey Research Center and Department of Anatomy, Physiology & Pharmacology at AU. The approach developed by the investigators uses AAV vectors to treat the entire brain and spinal cord after injection of only a few intracranial sites. Preclinical studies demonstrated a remarkable extension in lifespan from 8 months in untreated GM1 cats to greater than 4.5 years in AAV-treated cats, with dramatic improvements in quality of life. Results were published in Science Translational Medicine in 2014 (McCurdy, V.J., et al., Sustained normalization of neurological disease after intracranial gene therapy in a feline model. Science Translational Medicine, 2014. 6(231): p. 231ra48).

“We are thrilled by our collaboration with University of Massachusetts Medical School and Auburn University, which constitutes a significant step towards the development of a treatment for patients affected with GM1-gangliosidosis, a severely debilitating disease. For each of these patients and their families, there is currently no option and an urgent need for a safe and effective therapy”, said Karen Aiach, founding president and CEO of LYSOGENE. “AAV-based therapies are particularly suitable for inherited disorders of the CNS. In this new program, LYSOGENE will leverage its unique capacity to develop these therapies and bring them to patients with unmet needs. We will also reinforce our scientific and technology base through our collaboration with leaders in the field.”

“Collaborating with LYSOGENE will allow us to leverage their clinical and translational expertise and advance the development of a gene transfer therapy for treating patients affected with GM1-gangliosidosis,” said Sena-Esteves. “In our minds, what ultimately matters is the ability to deliver a potential treatment to the children suffering from this horrible disease. Ultimately, that’s what drives us all.”

About Gangliosidosis with GM1
GM1-gangliosidosis is a rare inherited neurodegenerative disorder characterized by severe cognitive and motor developmental delays resulting in death of most patients at a very young age.

It is caused by mutations in the GLB1 gene, which encodes an enzyme called beta-galactosidase necessary for recycling of a molecule (GM1-ganglioside) in neurons. This brain lipid is indispensable for normal function, but its overabundance causes neurodegeneration, resulting in the severe neurological symptoms of GM1-gangliosidosis.

GM1 affects 1 in 100,000 - 200,000 newborns and is inherited in an autosomal recessive pattern. GM1-gangliosidosis can be classified into three major clinical phenotypes according to the age of onset and severity of symptoms: Type I (infantile), Type II (late infantile/juvenile) and Type III (adult). There is currently no treatment for this disease.

About LYSOGENE
LYSOGENE is a clinical stage biotechnology company committed to the development and commercialization of innovative therapies for patients affected with rare disorders and high unmet medical needs. LYSOGENE’s team translated its rAAVrh10 lead product for Sanfilippo from bench to bedside in an unprecedented fashion over the last years. Its lead product is for Sanfilippo syndrome, a neurodegenerative lysosomal storage disorder considered to be a perfect model for gene therapy. LYSOGENE is currently expanding its pipeline to additional diseases with high unmet medical needs. Lysogene was launched in 2009. It completed a Series A financing in May 2014 with leading life sciences investors Sofinnova Partners, BPI Innobio and Novo AIS.

For more information, visit www.lysogene.com.

About University of Massachusetts Medical School
The University of Massachusetts Medical School (UMMS), one of five campuses of the University system, is comprised of the School of Medicine, the Graduate School of Biomedical Sciences, the Graduate School of Nursing, a thriving research enterprise and an innovative public service initiative, Commonwealth Medicine. Its mission is to advance the health of the people of the Commonwealth through pioneering education, research, public service and health care delivery with its clinical partner, UMass Memorial Health Care. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education. The Medical School attracts more than $240M annually in research funding, placing it among the top 50 medical schools in the nation. In 2006, UMMS’s Craig C. Mello, PhD, Howard Hughes Medical Institute Investigator and the Blais University Chair in Molecular Medicine, was awarded the Nobel Prize in Physiology or Medicine, along with colleague Andrew Z. Fire, PhD, of Stanford University, for their discoveries related to RNA interference (RNAi). The 2013 opening of the Albert Sherman Center ushered in a new era of biomedical research and education on campus. Designed to maximize collaboration across fields, the Sherman Center is home to scientists pursuing novel research in emerging scientific fields with the goal of translating new discoveries into innovative therapies for human diseases.

About Auburn University and the College of Veterinary Medicine
Auburn University has provided instruction, research and outreach to benefit the state of Alabama and the nation for more than 155 years and is among a distinctive group of universities designated as Land, Sea and Space Grant institutions. Auburn makes a nearly $5B economic contribution to the state each year, has more than 250,000 graduates and provides 140 degree programs to more than 25,000 graduate and undergraduate students. For more news about Auburn University, go to www.auburn.edu. The College of Veterinary Medicine, the oldest veterinary medical program in the South, has a mission to prepare individuals for careers of excellence in veterinary medicine, including private and public practice, industrial medicine, academics, and research. The College is devoted to three strategic goals: (1) enhance student success; (2) promote discovery; and (3) practice the highest standards of veterinary medicine. For more, go to www.vetmed.auburn.edu. The Scott-Ritchey Research Center’s mission is to improve the health of companion animals and translate those findings to the clinical and scientific communities to facilitate their application in humans.