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BioMarin and the Allen Institute Explore Use of Novel Scientific Technologies to Interrogate Rare Brain Diseases

It is increasingly understood in the scientific research community that many central nervous system disorders affect individual brain circuits or cell types rather than the entire brain. BioMarin, a pioneer in the development of targeted therapeutics, has formed a new partnership with the Allen Institute to further explore the fundamental mechanisms of these individual circuits and cell types in rare brain diseases.

BioMarin is a demonstrated leader in the development of treatments for rare conditions that affect multiple body systems, including the central nervous system. BioMarin also remains at the forefront of scientific technology design, leveraging the potential of adeno-associated virus (AAV) gene therapy to investigate treatments for multiple genetic conditions. The Allen Institute for Brain Science, a division of the Allen Institute, has developed modified AAVs that have been engineered to impact specific classes of cells in the brain. Scientists from BioMarin and the Allen Institute will now collaborate to investigate whether these novel AAVs can potentially enable the creation of a new class of gene therapies to treat diseases of the central nervous system.

Researchers at the Allen Institute originally developed the AAV tools with the goal of studying and classifying individual brain cell types. The tools are built by engineering AAVs to carry genes that switch on in specific types of neurons or other cells in the brain. A key component of these engineered AAVs is a unique molecular “zip code” or enhancer, which ensures that viral gene expression is restricted to only the correct cellular address in the brain. To date, scientists have used the tools to ferry fluorescent labels to the brain, lighting up single brain cell types or subclasses of brain cells under the microscope to enable better studies of individual cell types. Researchers have also shown that the same tools can selectively label comparable cell types.

“At BioMarin, we welcome the chance to collaborate with fellow researchers who can help to further expand our scientific perspective around intractable conditions,” said Lon Cardon, Ph.D., Senior Vice President and Chief Scientific Strategy Officer. “Our collaboration with the Allen Institute now offers us great opportunities to partner with other leaders in brain research, laying the scientific foundation to potentially deliver multiple investigational gene therapies for CNS conditions.”

“BioMarin scientists have a long history of trying to do things that haven’t been done before—and we embrace innovative tools and technologies that accelerate our discoveries,” said Kevin Eggan, Ph.D., Group Vice President and Head of Research and Early Development. “Our partnership with the Allen Institute is in direct alignment with this scientific tradition, as we interrogate rare brain diseases that impact people around the world.”

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