Auburn, Alabama —
A collaboration has been formed between the Auburn University College of Veterinary Medicine, the University of Massachusetts Medical School and Lysogene to further develop a gene therapy treatment for GM1-gangliosidosis.
Through the collaboration, Lysogene, UMMS and AUCVM will develop pre-clinical studies in GM1 gangliosidosis, a rare, inherited disorder characterized by severe neurological impairment, using adeno-associated virus (AAV) gene therapy technology.
“This new partnership with Lysogene will undoubtedly enhance the pace of progress in this important area of research,” said Dr. Frank Bartol, associate dean for research and undergraduate studies at the College of Veterinary Medicine.
“This project builds on six years of previous gene therapy research and is the next crucial step toward human clinical trials,” said Dr. Doug Martin, a scientist at the Scott-Ritchey Research Center at the College of Veterinary Medicine. “We are very fortunate to work with Lysogene to finalize a treatment for GM1.”
A France-based leading, clinical stage gene therapy biotechnology company, Lysogene is committed to the development and commercialization of breakthrough treatments for severe orphan pathologies affecting the central nervous system.
The development of a potential treatment for GM1-gangliosidosis using AAV gene therapy was initiated in 2005 by Martin, who also is an associate professor in the Department of Anatomy, Physiology & Pharmacology at Auburn, and Dr. Miguel Sena-Esteves, associate professor in the Neurology Department and the Gene Therapy Center at UMMS.
The collaboration will combine Lysogene’s translational and clinical expertise in gene therapy for central nervous system 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 approach developed by the investigators uses AAV vectors to treat the entire brain and spinal cord. Pre-clinical studies demonstrated a remarkable extension in lifespan from eight months in untreated GM1 cats to greater than 4.5 years in AAV-treated cats, with dramatic improvements in quality of life.
“We are thrilled by our collaboration with the 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 central nervous system. 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 GM1 Gangliosidosis
GM1 gangliosidosis is a rare inherited neuro-degenerative 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 one in 100,000-200,000 newborns and is inherited in an autosomal recessive pattern. GM1 gangliosidosis can be classified into three major clinical types 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.