Enhanced Treatment and Prediction of Therapeutic Responses in Friedreich Ataxia Patients
Legal Citation
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 Number | US 11,857,532 |
|---|---|
| Title | Treatment 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 |