Neurodegenerative Disorders

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The challenge in delivering therapeutics for neurodegenerative disorders lies in the physical delivery of biologics across the blood-brain barrier.  For current applications, gene therapies are being delivered by direct injection into the central nervous system. NAV® Therapeutics are significantly more efficient in delivering genes to the CNS than other AAV serotypes, and can distribute gene expression further with each injection.

Current status of gene therapy for neurodegenerative disorders:

Several groups are in clinical trials using direct injection of gene therapy vectors into the brain for the following diseases:

Batten Disease

  • The therapeutic delivers a correct copy of the mutated gene

Metachromatic Leukodystrophy

  • One-time delivery of a normal copy of the arylsulfatase A (ARSA) gene to the brain

Alzheimer’s disease

  • Neuroprotective factors to protect neurons from degeneration

Future applications:

NAV Technology (specifically, rAAV9) is showing promise in crossing the blood-brain barrier, and may lead to less invasive routes of administration. This will be applicable both for diseases in which the brain is the primary target, such as inherited neurodegenerations, Parkinson’s and Alzheimer’s diseases, and for diseases in which the spinal cord must also be targeted, such as spinal muscular atrophy.