Enhanced Treatment and Prediction of Therapeutic Responses in Friedreich Ataxia Patients

Publication ID: 24-11857532_0006_PTD
Published: October 28, 2025
Category:Direct Improvements & Enhancements

Legal Citation

pr1or.art Inc., “Enhanced Treatment and Prediction of Therapeutic Responses in Friedreich Ataxia Patients,” Published Technical Disclosure No. 24-11857532_0006_PTD, Published October 28, 2025, available at https://archive.pr1or.art/24-11857532_0006_PTD
This technical disclosure describes improvements that would be readily apparent to a Person Having Ordinary Skill In The Art (PHOSITA) when considered in combination with the foundational architecture disclosed in U.S. Patent No. 11,857,532.

Summary of the Inventive Concept

This inventive concept improves the treatment and prediction of therapeutic responses in Friedreich ataxia patients by introducing sustained-release formulations, computational models, synergistic co-administration, and personalized monitoring, enhancing patient compliance, treatment efficacy, and overall iron homeostasis.

Background and Problem Solved

The original patent disclosed methods for treating Friedreich ataxia by increasing TfR1 palmitoylation, but had limitations in terms of patient compliance, treatment efficacy, and iron homeostasis. This inventive concept addresses these limitations by introducing novel formulations, computational models, and synergistic co-administration, providing a more comprehensive and effective approach to treating Friedreich ataxia.

Detailed Description of the Inventive Concept

The inventive concept comprises four main aspects: (1) sustained-release formulations of TfR1 palmitoylation-increasing drugs to improve patient compliance, (2) a computational model integrating genomic data, iron content measurements, and TfR1 palmitoylation levels to provide personalized treatment plans, (3) synergistic co-administration of TfR1 expression-increasing and TfR1 palmitoylation-increasing agents to enhance iron homeostasis, and (4) personalized monitoring of treatment efficacy through TfR1 palmitoylation level measurements. These aspects work in concert to provide a more effective and targeted approach to treating Friedreich ataxia.

Novelty and Inventive Step

The new claims introduce sustained-release formulations, computational models, and synergistic co-administration, which are novel and non-obvious improvements over the original patent. These innovations provide a significant enhancement to the treatment and prediction of therapeutic responses in Friedreich ataxia patients.

Alternative Embodiments and Variations

Alternative embodiments of the inventive concept could include different sustained-release formulations, computational models incorporating additional data types, and synergistic co-administration of different agents. Variations could also include the use of different TfR1 palmitoylation-increasing drugs or the integration of additional biomarkers for personalized monitoring.

Potential Commercial Applications and Market

The inventive concept has significant commercial potential in the pharmaceutical and biotechnology industries, particularly in the development of treatments for rare genetic disorders like Friedreich ataxia. The market for Friedreich ataxia treatments is growing, and this inventive concept could provide a competitive advantage in terms of treatment efficacy and patient compliance.

Original Patent Information

Patent NumberUS 11,857,532
TitleTreatment and prediction of therapeutic responses in patients suffering from Friedreich ataxia
Assignee(s)INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE), The Assistance Publique—Hôpitaux de Paris (APHP), FONDATION IMAGINE