Emory University

Regenxbio Inc.

Above and Beyond Nb, LLC

Nicholas M Boulis

Raymond T Bartus

Karen Kozarsky

John N Oshinski

Alexandre Betourne

Jonathan Patrick Riley

Jason J Lamanna

Cody Dillion Anderson

Anthony Donsante

It is disclosed herein that ssAAV and scAAV vectors of the same serotype administered by injection into the cerebrospinal fluid (CSF) via the intracerebroventricular (ICV) or intrathecal (cisternal or lumbar) route exhibit different cellular tropisms in the central nervous system. Thus, a subject can be treated by injection into the CSF of ssAAV or scAAV vector encoding a therapeutic protein, such as an ssAAV9 or scAAV9 vector. The therapeutic protein can be targeted to specific cells using these vectors. In some embodiments, scAAV9 is utilized to achieve superior transduction in the hippocampus, cerebellum and cerebral cortex where both neurons, particularly Purkinje neurons, and glial cells (such as astrocytes) are transduced. In other embodiments, ssAAV9 is utilized to minimize transduction of astrocytes. In further embodiments, an immunosuppressive agent is also administered to the subject.

Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

Devices and systems can be configured to guide an instrument to a target region. The guide system may include an imaging guide including a first segment, the first segment including a guide region and imaging coils surrounding the guide region; and a platform. The platform may include a first rail, a second rail disposed parallel to the first rail, and a positioning member disposed between the first rail and the second rail. The positioning member may include a positioning frame having an entry region. The positioning frame may be movably disposed with respect to the first and second rails in a first direction and a second direction that is perpendicular to the first direction. The platform may be disposed with respect to the imaging guide so that a position of the entry region is within the guide region.

Devices and systems for MRI-guided procedures

The present invention provides methods for parenterally administering riluzole to subjects in need of treatment.

Formulation of a riluzole solution with beta-cyclodextrins

Growing community of inventors

Atlanta, GA, United States of America

Nicholas M Boulis