Lysogene, Weizmann Institute Team Up on Gene Therapy for Neuronopathic Gaucher

Lysogene, Weizmann Institute Team Up on Gene Therapy for Neuronopathic Gaucher

Lysogene has entered into a collaborative research agreement with Yeda Research and Development, a commercial branch of the Weizmann Institute of Science, to develop a gene therapy for neuronopathic Gaucher disease that affects the brain and spinal cord, the biopharmaceutical company announced.

The therapy also will be investigated as a treatment for Parkinson’s disease and other disorders caused by mutations in the GBA gene.

“This collaboration is a perfect fit with Lysogene’s strategy, providing the opportunity to develop a novel therapy for a rare lysosomal disease, with the potential to expand into neurological diseases with much larger patient populations, such as Parkinson,” Ralph Laufer, chief scientific officer of Lysogene, said in a press release.

In Gaucher, mutations in the GBA gene lower the activity of the beta-glucocerebrosidase enzyme. That enzyme is responsible for breaking down a fatty substance called glucocerebroside in cellular structures known as lysosomes. As a result, glucocerebroside accumulates to toxic levels inside immune cells called macrophages that migrate into different organs and tissues.

The inherited disorder is divided into neuronopathic — types 2 and 3 — if the brain and spinal cord are affected, and non-neuronopathic, or type 1, when no such complications are seen.

Gene therapy is a potential treatment option for Gaucher and other genetic disorders in which the faulty gene causing the disease is replaced with a healthy one. The approach uses a harmless virus, called adeno-associated virus (AAV), that delivers the normal version of the gene to patients’ cells.

The goal of the new collaboration, established between Lysogene and Anthony Futerman’s lab at the Weizmann Institute, in Israel, is to create a new gene therapy for neuronopathic forms of Gaucher. The partnership leverages the company’s expertise on the design and production of AAV vectors, and Futerman’s experience on sphingolipid biology.

Sphingolipids — found in all animals, plants, and fungi — are a class of fatty molecules that make up cells’ membranes and also play a key role as mediators in several signaling cascades. Defects in sphingolipid metabolism can lead to a series of diseases, including Gaucher, which is one of the focuses of Futerman’s research.

“We are thrilled to start this research collaboration with Prof. Futerman, a leading expert in the field of sphingolipid biology,” Laufer said.

Under the terms of the agreement, Futerman’s team will be responsible for creating new versions of beta-glucocerebrosidase with enhanced biological properties, and for carrying out all proof-of-concept studies. On its end, Lysogene has an exclusive option to license the program.