Lysogene and Yeda Signs Gene Therapy Licensing Deal
Lysogene has signed an exclusive global licence agreement with Yeda Research and Development, the commercial arm of Weizmann Institute of Science to develop and market a gene therapy candidate to treat neuronopathic Gaucher disease and Parkinson’s disease (PD) with GBA1 mutations.
The new candidate is an adeno-associated virus (AAV) gene therapy approach developed to treat neuronopathic Gaucher disease and PD and other ailments linked to GBA1 gene mutations. Lysogene offered AAV vector design and production expertise while Yeda has contributed glucocerebrosidase variants with improved biological features.
Lysogene opted to exercise its option for licencing the programme based on positive initial biological proof of principle studies, following nearly 20 months of successful partnership. Lysogene will oversee the preclinical, clinical development, production, regulatory works and marketing of the drug candidate globally. Yeda is entitled to receive royalty in addition to other payments.
Ralph Laufer, CSO of Lysogene shared “We are thrilled to add to our R&D pipeline a new promising gene therapy asset, which addresses high unmet medical needs in Gaucher disease, as well as broader indications, such as PD-GBA. “The cost of these preclinical studies will not affect our cash runway. We continue to replenish our early-stage pipeline to position Lysogene as a key player in the field of gene therapy translational science.”
Lysogene is a biopharmaceutical gene therapy company with lead programs in neurodegenerative lysosomal storage disorders that affect children. More information is available here