Patent Application: Treating Brain Diseases with rAAV Particles
Summary
The USPTO has published a new patent application (US20260083862A1) from the University of Iowa Research Foundation detailing methods for treating brain diseases using rAAV particles administered via the cisterna magna. The application was filed on May 19, 2025.
What changed
This document is a published patent application from the USPTO, specifically application US20260083862A1, filed by the University of Iowa Research Foundation. It describes methods and compositions for treating brain diseases or delivering therapeutic agents by administering rAAV particles into the cisterna magna and/or ventricle. The vector within the particle contains a nucleic acid encoding a therapeutic protein designed to be expressed and secreted into the cerebrospinal fluid (CSF) for distribution throughout the brain.
As this is a patent application, it does not impose immediate regulatory obligations on companies. However, it signals potential future developments in gene therapy and treatments for neurological disorders. Companies in the pharmaceutical and biotechnology sectors, particularly those involved in gene therapy or CNS drug development, should monitor the progress of this and similar patent applications as they may indicate emerging therapeutic approaches or intellectual property landscapes.
Source document (simplified)
METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES
Application US20260083862A1 Kind: A1 Mar 26, 2026
Assignee
UNIVERSITY OF IOWA RESEARCH FOUNDATION
Inventors
Beverly L. Davidson, Luis Tecedor, Yong Hong Chen
Abstract
The present disclosure provides methods of treating a disease or delivering a therapeutic agent to a mammal comprising administering to the mammal's cisterna magna and/or ventricle an rAAV particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner such that cells with access to the cerebrospinal fluid (CSF) express the therapeutic agent and in certain embodiments secretes the therapeutic agent into the CSF for distribution to the brain.
CPC Classifications
A61K 48/0058 A61K 31/343 A61K 48/0075 A61K 48/00 C12N 2750/14143
Filing Date
2025-05-19
Application No.
19212269
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