Ceregene Acquires Rights to ALS Gene Therapy from the Salk Institute

12-Aug-2003

Ceregene, Inc., a biotechnology company focused on gene therapies for neurodegenerative disorders, today announced that it has signed an option agreement with the Salk Institute for Biological Studies for exclusive worldwide rights to its ALS (amyotrophic lateral sclerosis) gene therapy technology. This technology was described in the August 8, 2003 issue of the journal, Science, and was reported to nearly double the life span of mice in a model of Lou Gehrig's disease.

Ceregene and the Salk Institute are working collaboratively to establish proof of principle for efficacy in ALS patients by using Ceregene's proprietary gene delivery technology, including the adeno-associated viral (AAV) vector system, to deliver therapeutic growth factor genes to selective targets with the ability to maintain long- term expression of the gene. AAV has previously been evaluated as a delivery system in humans in the treatment of genetic diseases. Ceregene has the right to pursue the development and commercialize this technology based upon the program's progress. Financial terms of the agreement were not disclosed.

"We are pleased to enter into this agreement with the Salk Institute for the use of gene therapy to treat this deadly disease. While Ceregene's internal programs will remain focused on Alzheimer's disease and Parkinson's disease, a number of other neurodegenerative conditions could benefit from our technological platforms. ALS is a good example," stated Jeffrey M. Ostrove, Ph.D., president and chief operating officer of Ceregene. "In order to not compromise our focus, while expanding product opportunities, we have entered into collaborations such as this one with the Salk Institute for ALS gene therapy with consortiums who might benefit from having access to our technologies and expertise."

"Using a single treatment, the approach evaluated by Ceregene and the Salk Institute offers the potential to enhance the function and retard the degeneration of vulnerable neurons, thereby improving the quality of life and extending survival of the victims of this disease," stated Raymond T. Bartus, Ph.D., vice president of research and development at Ceregene.

Ceregene's in vivo gene therapies utilize non-replicating viral vectors to deliver therapeutic genes to the nervous system, where they express proteins beneficial in treating neurological diseases. The AAV vector is an important gene delivery tool which has demonstrated its ability to efficiently and stably express genetic information in non-dividing target cells, such as the motor neurons degenerating in ALS, and to achieve long-term, or potentially permanent therapeutic benefit.

ALS, which is often referred to as "Lou Gehrig's disease," is a progressive neurodegenerative disease affecting nerve cells in the spinal cord and brain. The progressive degeneration of the motor neurons in ALS eventually lead to the loss of the ability of the brain to initiate and control muscle movement. With all voluntary muscle action affected, patients in the later stages of the disease become totally paralyzed, yet most patients minds remain unaffected. This devastating disease is estimated to affect approximately 30,000 Americans.

https://www.bionity.com/en/news/27564/ceregene-acquires-rights-to-als-gene-therapy-from-the-salk-institute.html