Capsida Biotherapeutics Announces Publication in Nature Neuroscience From Caltech Demonstrating Robust, Non-invasive IV Gene Delivery Targeted to the Brain While Avoiding Liver
Retrieved on:
Thursday, December 9, 2021
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For a genetic intervention to be safe and effective, a gene therapy should express a transgene in the affected brain cells while minimizing off-target expression.
Key Points:
- For a genetic intervention to be safe and effective, a gene therapy should express a transgene in the affected brain cells while minimizing off-target expression.
- The research published in Nature Neuroscience describes how combinatorial AAV engineering of multiple loops was utilized to select capsids with brain-wide gene expression and liver detargeting after IV delivery in mouse and marmoset models.
- These findings extended to non-human primates following IV administration, allowing for robust, non-invasive gene delivery to the central nervous system (CNS).
- Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders.