UMass Medical partners with French company on gene therapy treatment
UMass Medical School in Worcester will collaborate with Lysogene, a French biotechnology company, and Auburn University in Alabama, to develop pre-clinical studies of a rare disorder that causes neurological impairment, Lysogene announced.
The two universities will use gene therapy technology to develop studies of GM1-gangliosidosis, an inherited disorder that causes severe neurological impairment. The collaboration will combine Lysogene’s clinical expertise in gene therapy with the preclinical expertise and infrastructure of UMMS and Auburn University, according to a statement.
Development of a potential treatment for the disorder was initiated by Miguel Sena-Esteves, associate professor in the Neurology Department and Gene Therapy Center at UMMS, along with Douglas R. Martin, associate professor in the Scott-Ritchey Research Center and Department of Anatomy, Physiology, and Pharmacology at Auburn.
Preclinical studies showed a “remarkable” extension in lifespan in cat experiments, according to Lysogene, from eight months in untreated cats to more than four-and-a-half years in cats who received treatment. Results were published in the medical journal Science Translational Medicine in 2014.
Karen Aiach, president and CEO of Lysogene, said the collaboration marks a “significant step towards the development of a treatment for patients affected with GM1-gangliosidosis … For each of these patients and their families, there is currently no option and (there is) an urgent need for a safe and effective therapy.”
Image source: Freedigitalphotos.net
WBJ Web Partners
Most Recent
Most Recent
0 Comments