Plasmid DNA Constructs for Sustained Therapeutic Protein Expression
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Solution Overview
Problem
Conventional protein drugs require frequent administrations due to short half-life in circulation, leading to inconvenient dosing schedules and adverse side effects.
Innovation Solution
DNA constructs encoding therapeutic proteins for sustained expression in mammalian hosts, allowing production of therapeutic proteins within patient cells, such as skeletal muscle cells, which act as in vivo bioreactors, reducing administration frequency and maintaining therapeutic protein levels for extended durations.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional protein drugs are administered, then therapeutic effect is achieved, but administration frequency must be increased due to short half-life
Solution Approach 1:
The patent introduces a DNA construct that encodes the therapeutic protein, which is delivered to and expressed within host cells before the actual therapeutic effect is needed. This preliminary action of introducing the genetic code allows the host cell to continuously produce the therapeutic protein, eliminating the need for frequent external administrations.
Solution Approach 2:
The host cell takes over the production of the therapeutic protein by expressing the DNA construct locally within its own cellular environment. This self-service mechanism allows the cell to autonomously produce and release the therapeutic protein continuously, replacing the need for external frequent administrations.
2Reliability
If conventional protein drugs are administered frequently, then therapeutic window is maintained, but convenience and patient compliance deteriorate
Solution Approach 1:
The DNA construct is introduced into host cells as a preliminary action, establishing a self-sustaining production system that maintains therapeutic levels continuously without requiring frequent dosing interventions, thereby improving convenience while maintaining therapeutic window.
Solution Approach 2:
The patent enables continuous production of the therapeutic protein within host cells through the expressed DNA construct, replacing discontinuous frequent administrations with a continuous generation process that maintains therapeutic levels consistently over extended periods.
3Speed
If conventional protein drugs are used, then immediate therapeutic effect is achieved, but duration of action is limited
Solution Approach 1:
The host cell serves itself by producing the therapeutic protein continuously through the expressed DNA construct, extending the duration of action from the limited half-life of externally administered proteins to prolonged continuous production within the cellular environment.
Solution Approach 2:
The DNA construct acts as an intermediary that translates genetic information into continuous therapeutic protein production within host cells, bridging the gap between initial rapid effect and sustained long-term action by establishing a persistent production mechanism.
Data Source
AI summary
The disclosure is directed to compositions that comprise a plasmid DNA construct having a DNA sequencing encoding a therapeutic protein, or a fragment thereof, in vivo, along with methods of generating and manufacturing the antibody or therapeutic protein, as well as methods for preventing and/or treating a disease in a patient.