Recombinant Invertase Composition for Room-Temperature CSID Therapy
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Solution Overview
Problem
Current treatments for congenital sucrase-isomaltase deficiency (CSID), such as the use of sacrosidase, are thermally unstable and require refrigeration, limiting their usability and patient compliance, and do not fully replace the enzymatic activity of the endogenous sucrase-isomaltase enzyme complex.
Innovation Solution
Development of recombinant mutant invertase enzymes with improved stability and activity, including enhanced pH, thermal, and proteolytic stability, suitable for oral administration, and formulation into stable pharmaceutical compositions that can be stored at room temperature.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If sacrosidase solution is used to treat CSID, then enzymatic activity is provided, but thermal instability causes degradation and requires refrigeration
Solution Approach 1:
The patent applies parameter changes by modifying the enzyme's physical and chemical properties through protein engineering. Specifically, the sacrosidase enzyme undergoes structural modifications (such as mutagenesis) to alter its thermal stability parameters, allowing it to maintain enzymatic activity at higher temperatures without degradation, thus eliminating the need for refrigeration while preserving therapeutic effectiveness
Solution Approach 2:
The patent employs composite materials by formulating the enzyme therapy in a stabilized complex that combines the modified sacrosidase enzyme with protective agents or carrier molecules. This composite formulation enhances the enzyme's resistance to thermal degradation and proteolytic digestion, maintaining stability and activity under varied storage and administration conditions
2Reliability
If sacrosidase solution is used to treat CSID, then enzymatic activity is provided, but acid instability causes degradation and limits usability
Solution Approach 1:
The patent applies parameter changes by modifying the enzyme's structural parameters to resist acid-induced degradation. Through protein engineering techniques, the sacrosidase enzyme is modified to maintain its three-dimensional structure and catalytic activity in acidic environments, enabling safe administration with food and beverages without premature degradation
3Reliability
If recombinant mutant enzymes are developed, then stability and activity are improved, but manufacturing complexity increases
Solution Approach 1:
The patent employs copying by using recombinant DNA technology to create synthetic copies of the modified enzyme gene in host cells. This allows for standardized, scalable production of the enzyme therapy through microbial fermentation, reducing manufacturing complexity compared to extracting and modifying enzymes from natural sources while maintaining improved stability and activity characteristics
4Ease of operation
If liquid formulation is used, then ease of administration is improved, but shelf life is reduced due to instability
Solution Approach 1:
The patent applies parameter changes by modifying the formulation's physical state and chemical composition to enhance stability. The enzyme therapy is formulated as a stabilized liquid or lyophilized powder that maintains stability over extended periods at room temperature, extending shelf life while preserving ease of administration through simple reconstitution or direct oral delivery
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
The recombinant mutant enzymes provide increased stability and activity, allowing for effective treatment of CSID with improved patient compliance and ease of use, as they can be administered orally and stored without refrigeration.
Implementation Method 1
catalyzing the hydrolysis of dietary sucrose and starch
Implementation Method 2
recombinant mutant invertase (sucrase) enzymes that are active in humans and have greater stability and/or activity than naturally occurring enzymes
Data Source
AI summary
Disclosed are recombinant mutant invertase enzymes. Also disclosed are pharmaceutical compositions comprising an invertase enzyme, e.g., a recombinant mutant invertase enzyme, and an optional isomaltase enzyme. The enzymes and compositions can be used, among other things, to treat congenital sucrase-isomaltase deficiency (CSID).