Gene therapy specialist Oxford BioMedica plc and its partner the Amyotrophic Lateral Sclerosis (ALS) Therapy Development Institute have announced the extension of their collaboration, following successful completion of the first phase.
The extended collaboration, which is funded by the Muscular Dystrophy Association, aims to advance the development of Oxford BioMedica’s preclinical gene therapy candidate, MoNuDin, and to evaluate other gene-based strategies for the treatment of amyotrophic lateral sclerosis, or Lou Gehrig's disease.
“This collaboration adds yet another important element to our burgeoning drug development pipeline. Gene therapy is a crucial, emerging therapeutic option for diseases such as ALS and Oxford BioMedica has the experience and commitment for excellence needed in a partner,” said Steven Perrin, Chief Executive Officer and Chief Scientific Officer of the ALS Therapy Development Institute.
The collaboration brings together Oxford BioMedica’s LentiVector gene delivery technology with the Institute’s gene expression database and drug screening capabilities. The first phase of the collaboration included the development of new techniques to evaluate and identify gene therapy candidates at the Institutes research facility in Cambridge, UK.
In this second phase, the Institute will conduct preclinical efficacy studies of MoNuDin, which is designed to protect the motor neurons susceptible to degeneration from ALS by targeted delivery of a neuroprotective gene, vascular endothelial growth factor. The team also plans to evaluate other potentially therapeutic genes that inhibit or regulate specific pathways associated with disease onset or progression.