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May 22, 2024 : The American Society of Gene and Cell Therapy (ASGCT) annual meeting recently featured promising developments in brain-penetrant adeno-associated viral (AAV) capsids. These engineered capsids hold immense potential for gene therapies targeting neurological disorders.
The presentations highlighted advancements by various companies and research institutions in developing AAV capsids specifically designed to cross the blood-brain barrier (BBB). The BBB is a highly selective membrane system that protects the brain from foreign substances in the bloodstream. However, this protective barrier also presents a significant obstacle to delivering gene therapies to the central nervous system.
One presentation focused on Voyager Therapeutics’ TRACER™ platform, which utilizes a high-throughput screening method to identify novel capsids with enhanced BBB penetration capabilities. Data presented demonstrated that these capsids effectively delivered transgenes (therapeutic genes) to multiple brain regions in non-human primates following intravenous administration. This approach offers a minimally invasive method for potentially treating various neurological conditions.
Another presentation discussed research by Sangamo Therapeutics, focusing on the evolution of blood-brain barrier penetrant AAV capsids. Their work explores methods for optimizing capsid design through engineering techniques to further enhance BBB permeability and achieve targeted delivery to specific brain regions.
Developing efficient and safe brain-penetrant AAV capsids represents a significant step forward for gene therapy targeting neurological diseases. This technology could potentially benefit several currently incurable conditions, including Alzheimer’s, Parkinson’s, and Huntington’s disease.
Challenges remain. Researchers must ensure the safety and specificity of these capsid designs while optimizing their ability to deliver gene therapies effectively. Additionally, large-scale clinical trials will be necessary to validate the efficacy and safety of these novel approaches in human patients.
Despite these hurdles, the progress reported at the ASGCT meeting signifies a renewed sense of optimism in gene therapy for neurological disorders. The development of brain-penetrant AAV capsids holds the potential to revolutionize treatment options for a wide range of devastating neurological conditions.
