ALS Therapy Development Institute And Oxford BioMedica Begin Second Phase Of Collaboration To Develop Gene Therapies For ALS, UK

Main Category: Muscular Dystrophy / ALS
Article Date: 03 Feb 2010 - 19:00 PDT

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The ALS Therapy Development Institute and Oxford BioMedica announced today 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 (ALS or Lou Gehrig's disease).

"ALS is a debilitating disease that takes so many of our best and brightest from us without warning and without reason. 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, Ph.D., Chief Executive Officer and Chief Scientific Officer of the ALS Therapy Development Institute (ALS TDI).

The collaboration combines Oxford BioMedica's advanced LentiVector® gene delivery technology with the ALS TDI's extensive 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 ALS TDI's research facility in Cambridge.

In this second next phase, the ALS TDI will conduct preclinical efficacy studies of Oxford BioMedica's LentiVector-based product, MoNuDin, which is designed to protect the motor neurons susceptible to degeneration from ALS by targeted delivery of the neuroprotective gene, vascular endothelial growth factor. Furthermore, the joint team plans to evaluate other potentially therapeutic genes that inhibit or regulate specific genetic pathways associated with disease onset or progression.

Oxford BioMedica's Chief Executive Officer, John Dawson, commented: "The successful completion of the first phase of the collaboration is testament to the quality of the scientific teams at the ALS TDI and Oxford BioMedica. Our LentiVector technology has unique capabilities to deliver genes safely and efficiently to neuronal cells and our product candidate, MoNuDin, has shown promising results in preclinical models of ALS. The extension of our collaboration with the ALS TDI enables us to accelerate MoNuDin's development and to explore new disease pathways as potential targets for genetic intervention. We are delighted to have ongoing support from the Muscular Dystrophy Association for our collaboration with the ALS TDI, which underscores our shared goal to develop new treatment options for patients suffering from ALS."

The Muscular Dystrophy Association's Augie's Quest Initiative is a major donor to the ALS TDI and provided the critical funding needed to execute the collaboration between Oxford BioMedica and the ALS TDI.

Source
ALS Therapy Development Foundation

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