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Patent Application: Treating Brain Diseases with rAAV Particles

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Published May 19th, 2025
Detected March 26th, 2026
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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)

← USPTO Patent Applications

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

View original document →

Classification

Agency
USPTO
Published
May 19th, 2025
Instrument
Notice
Legal weight
Non-binding
Stage
Draft
Change scope
Minor
Document ID
US20260083862A1

Who this affects

Applies to
Drug manufacturers Pharmaceutical companies
Industry sector
3254 Pharmaceutical Manufacturing 3254.1 Biotechnology
Activity scope
Drug Development Gene Therapy
Geographic scope
United States US

Taxonomy

Primary area
Pharmaceuticals
Operational domain
Legal
Compliance frameworks
FDA 21 CFR Part 11 GxP
Topics
Biotechnology Medical Research

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