Best compound characterization protocol
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The document outlines a comprehensive protocol for compound characterization and testing, emphasizing the importance of understanding workflows to enhance efficiency and safety in compound development. Invivo Biosystems specializes in in-vivo testing with small animal models, particularly focusing on nutraceuticals for health and longevity, amid a growing market projected to be worth over $302 billion by 2022. It details various stages of compound analysis, case studies, and the application of findings in real-world scenarios, ultimately aiming for quick decision-making and reliable data for patent applications.
![The IVB Process: Simple. Effective. Fast.
13
INTRODUCTION
Understand
Your Goal
DESIGN
Create A
Personalized Catalog
LOGISTICS
Finalize Your Project
Design
REPORT OUT
Maintain Clarity and
Transparency
[ 3 MONTHS ]](https://image.slidesharecdn.com/bestcompoundcharacterizationprotocol-200624235810/85/Best-compound-characterization-protocol-13-320.jpg)
![INTRODUCTION
Understand
Your Goal
DESIGN
Create A
Personalized Catalog
LOGISTICS
Finalize Your Project
Design
REPORT OUT
Maintain Clarity and
Transparency
[ 3 MONTHS ]
Your Data Package, Your Next Move
14
MARKETING MATERIALS
PRESS RELEASE
INVESTOR MEETINGS
FUNDING ROUNDS
GRANT PROPOSALS
PATENT
APPLICATIONS
DEVELOPMENT
PIPELINE
WHITE PAPERS
SCIENTIFIC
PUBLICATIONS](https://image.slidesharecdn.com/bestcompoundcharacterizationprotocol-200624235810/85/Best-compound-characterization-protocol-14-320.jpg)
![IVB Longevity Compound Assessment: Stage I
17
Stage I: DOSAGE OPTIMIZATION
- Rule out possible toxicity.
- Test for most suitable downstream dosage.
- Determine… a) whether full longevity experiment suitable;
b) if so, at what compound dosage/under what conditions.
[Drug]
GFP
constitutive
RFP
induced
EC50](https://image.slidesharecdn.com/bestcompoundcharacterizationprotocol-200624235810/85/Best-compound-characterization-protocol-17-320.jpg)
![The IVB Process: Simple. Effective. Fast.
25
INTRO
Understand
Goal
DESIGN
Personalize
Catalog
LOGISTICS
Finalize
Design
REPORT
Maintain
Transparency
[ 3 MONTHS ]
Project or
Pipeline Goal
YOUR:
Complete
Data Package
YOUR:](https://image.slidesharecdn.com/bestcompoundcharacterizationprotocol-200624235810/85/Best-compound-characterization-protocol-25-320.jpg)
Best compound characterization protocol
- 2. Compound Characterization The compound characterization market is growing increasingly profitable and competitive at the same time. In order to develop a new compound product, the testing step is indispensable. Unlike drug discovery, compound testing is not as restrictive, but understanding the main workflow is still necessary to excel in the market. In order to help you improve both the efficiency and safety of compound testing, we developed the protocol to assist you in your findings.
- 3. Table of Contents 4-7 8-16 17-26 26-34 35-36 IVB Overview Market Overview Longevity Compound Assessment Case Use: Compound Analysis IVB: About Us
- 4. InVivo Biosystems 4 InVivo Biosystems is the market leader for early in-vivo testing using small animal models to gain a better understanding of the efficacy, mode of action, toxicity and potential targets of novel compounds. Right Model. Right Test. Right Insights.
- 5. In-Vivo Testing: Obtain the Data You Need 5 The Nematode Caenorhabditis elegans The Zebrafish Danio rerio COMPOUND TESTING TOXICITY ANALYSIS LIFESPAN HEALTHSPAN AGING STUDIES Research Idea Experimental Hypothesis Project / Pipeline Goal YOUR: Complete Data Package Fully Analyzed Results Data-Backed Conclusions YOUR: InVivo Biosystems: IN-VIVO WHOLE-ORGANISM TESTING
- 6. InVivo Biosystems: Your On-Call Lab 6 Nutraceutical Biopharma Biotech Pharmaceutical COMPANY NEED PROJECT SCOPE TACTICAL Select Experiments or Models STRATEGIC Early-Mid Project Phases MAXIMAL Full Project Completion
- 7. Nutraceuticals + Healthy Aging 7 Nutraceutical Biopharma Biotech Pharmaceutical COMPANY NEED PROJECT SCOPE TACTICAL Select Experiments or Models STRATEGIC Early-Mid Project Phases MAXIMAL Full Project Completion “Nutraceuticals may be used to improve health, delay the aging process, prevent chronic diseases, increase life expectancy, or support the structure or function of the body.”2 US nutraceutical market expected to reach > $138 billion USD by 2027.3 Global nutraceutical market expected to reach > $302 billion USD by 2022.4 Nutraceutical
- 8. Nutraceutical Market Drivers and Pain Points CHALLENGES DRIVERS RESTRAINTS Strong Consumer Interest: - anti-aging, general - longevity and health - skin care - heart disease - aging-related disease Resources and Time Cost: - high cost of raw material production - limited supply of quality manufacturing materials - long development pipeline increases production cost - lengthy lab-to-market timeline if animal testing involved Increasing Competition and Awareness: - growing competition as new players continuously enter market - increasing awareness among consumer base demands validation of claims - results must be backed by scientific evidence of efficacy AND safety 8
- 9. EARLY go/no-go decision making FAST experiment turn-around ECONOMICAL pipeline Nutraceutical Market Needs 9 limited resources – limited budget – urgent deadlines
- 10. Common Model Systems, Known Pitfalls HTS of 1000’s of compounds Fast data turnaround X No organ-system complexity X No whole-organism effects No HT possibilities X Long timelines X Full organ system complexity Human-relevant dosages Regulation Resources / Cost Experimental Timeline Model Complexity Cell Culture Mice Relevance to Human 10
- 11. The InVivo Biosystems Approach HIGH QUALITY DATA – FAST TURNAROUND – LOW COST MEDIUM THROUGHPUT – LOW REGULATION model systems capable of providing the detailed answers necessary to pursue your research aims Regulation Resources / Cost Experimental Timeline Model Complexity Cell Culture MiceFishWorms Relevance to Human 11
- 12. EARLY go/no-go decision making FAST experiment turn-around ECONOMICAL pipeline Nutraceutical Market Need: IVB Delivers 12 We focus on proof-of-principle experiments that provide the preliminary data necessary to quickly and confidently make go/no-go decisions during early-stage investigations.
- 13. The IVB Process: Simple. Effective. Fast. 13 INTRODUCTION Understand Your Goal DESIGN Create A Personalized Catalog LOGISTICS Finalize Your Project Design REPORT OUT Maintain Clarity and Transparency [ 3 MONTHS ]
- 14. INTRODUCTION Understand Your Goal DESIGN Create A Personalized Catalog LOGISTICS Finalize Your Project Design REPORT OUT Maintain Clarity and Transparency [ 3 MONTHS ] Your Data Package, Your Next Move 14 MARKETING MATERIALS PRESS RELEASE INVESTOR MEETINGS FUNDING ROUNDS GRANT PROPOSALS PATENT APPLICATIONS DEVELOPMENT PIPELINE WHITE PAPERS SCIENTIFIC PUBLICATIONS
- 15. InVivo Biosystems Platform Solutions 15 The Nematode Caenorhabditis elegans LIFESPAN + HEALTHSPAN The Zebrafish Danio rerio In Vivo TOXICITY TESTING CUSTOM DESIGN SOLUTIONS
- 16. The Longevity Platform – 3 Stages 16 LIFESPAN + HEALTHSPAN QUESTION What are the effects of a compound on lifespan, healthspan, and transcriptional changes related to aging? What is the mechanism of action? STAGE I Dosage Optimization STAGE II Data Collection STAGE III Molecular Analysis
- 17. IVB Longevity Compound Assessment: Stage I 17 Stage I: DOSAGE OPTIMIZATION - Rule out possible toxicity. - Test for most suitable downstream dosage. - Determine… a) whether full longevity experiment suitable; b) if so, at what compound dosage/under what conditions. [Drug] GFP constitutive RFP induced EC50
- 18. IVB Longevity Compound Assessment: Stage I 18 Fig. 1.1: EC50 determined for compound Lu0128. Fig. 1.2: Viability, growth, and development toxicity analysis for compound Lu0128. Xenobiotic Stress Oxidative Stress C. elegans Length C. elegans Development
- 19. 19 IVB Longevity Compound Assessment: Stage II Stage II: DATA COLLECTION - Specific metrics as pertinent to experiment. - Monitor animals for survival, morphology, specific movement patterns, etc. - Generate a full, high-resolution and multi-dimensional data set.
- 20. 20 IVB Longevity Compound Assessment: Stage II Stage II: DATA COLLECTION - Specific metrics as pertinent to experiment. - Monitor animals for survival, morphology, specific movement patterns, etc. - Generate a full, high-resolution and multi-dimensional data set.
- 21. 21 IVB Longevity Compound Assessment: Stage II Stage II: DATA COLLECTION - Specific metrics as pertinent to experiment. - Monitor animals for survival, morphology, specific movement patterns, etc. - Generate a full, high-resolution and multi-dimensional data set. N = 75 ΔT = 3 days P = 0.15 N = 150 ΔT = 1 day P = 0.014 N = 300 ΔT = 1 hour P = 0.0000054 High Resolution Analysis
- 22. IVB Longevity Compound Assessment: Stage II 22 Fig. 2.1: Survival and hazard rate curves from lifespan assay. Survival Rate Hazard Rate Fig. 2.2: (L) Morphological features analyzed, and (R) Principal Component Analysis (PCA) of multi- dimensional data set. Principal Component Analysis
- 23. 23 IVB Longevity Compound Assessment: Stage III Stage III: MOLECULAR ANALYSIS - Perform whole transcriptome analysis (WTA). - Delve into mechanism of action. - Identify pathways and players responsible for observed effects. Fig. 3.1: Volcano plot depicting gene expression change in compound- treated v. untreated Day 10 adult worms. C.e. Day 10 Adults
- 24. 24 IVB Longevity Compound Assessment: Stage III Longevity Pathway Analysis Fig. 3.2: Gene expression analysis comparing compound-treated v. untreated Day 10 adult worms, grouped by pathway.
- 25. The IVB Process: Simple. Effective. Fast. 25 INTRO Understand Goal DESIGN Personalize Catalog LOGISTICS Finalize Design REPORT Maintain Transparency [ 3 MONTHS ] Project or Pipeline Goal YOUR: Complete Data Package YOUR:
- 26. Case Use: Compound Analysis for ENCo* 26 *Company name and other identifiers have been altered to maintain confidentiality. Company: European Nutraceutical Company (ENCo) A small European-based nutraceutical company focused on developing… - natural extracts and compounds that have the ability to promote healthy aging; - food supplement formulations for the improvement of human vitality and longevity. Company’s Need:Move very RAPIDLY from HYPOTHESIS to early PROOF-OF-CONCEPT
- 27. Case Use: Compound Analysis for ENCo* 27 ENCo: “While mammalian models are currently the gold standard for drug development, the enforced 3Rs and animal welfare guidelines have required us to re-evaluate our development pipeline to include early stage in-vivo hypothesis testing. Understanding the biology of our compounds necessitates the use of an alternative model prior to going to more regulated, more resource and time-intensive models.” 3Rs of Animal Use in Research REPLACE REDUCE REFINE
- 28. Case Use: Compound Analysis for ENCo* 28 ENCo’s Hypothesis / Question: A. Do ENCo’s selected compounds (Compound A and Compound B) have any whole-organism effect on animal longevity? B. If so, what is the mechanism driving Compound A and Compound B efficacy? C. How does ENCo characterize Compound A and Compound B to advance progress towards clinical applications?
- 29. Case Use: Compound Analysis for ENCo* 29 ENCo’s Goal: Identify a single compound or synergistic compound mixture with the most positive and reproducible effect on longevity. ENCO’s Framework: - Data that is directly relevant to human health and the whole-body system. - Short 6-month timeline to encompass experimental design, preparation, execution and analysis of results. - Data to be formatted and available for use in patent application(s). ENCO’s Requirements: - Seeking whole-organism, in-vivo data rather in-vitro experiments. - Seeking fast, reliable and biologically relevant data in a whole-animal model. - Avoiding associated cost, timeline and regulatory paperwork of rodent models.
- 30. Case Use: Compound Analysis for ENCo* 30 ENCo’s Challenges: - In-vitro data alone not suitable or sufficient for downstream patent application needs. - Rodent models are gold standard for in-vivo work, but too time consuming and too costly at this stage. The In-Vivo Biosystems Solution Using the small animal model C. elegans, we proposed and executed a specific set of experiments designed by our expert team to address ENCo’s specific research goals, and to provide rapid and reliable data based upon the company’s short-term priorities and long-term aims. Population Locomotion Individual Locomotion Molecular Phenotyping Lifespan Assessment Healthspan Assessment
- 31. Case Use: Compound Analysis for ENCo* 31 Kaplan-Meier survival curve depicting results of compound impact on C. elegans lifespan. ENCo – Sample Data Acquired: Lifespan Assessment Lifespan assay tracking individual worm lifespan within a population; percent of worm population surviving (y-axis) versus time in days (x-axis).
- 32. Case Use: Compound Analysis for ENCo* 32 Electropharyngeogram (EPG) results depicting contraction of the pharyngeal muscle in C. elegans adult worms. ENCo – Sample Data Acquired: Individual Locomotion Frequency of contraction of the pharyngeal muscle (left) and amplitude of contraction of the pharyngeal muscle (proxy for strength of muscle contraction, right).
- 33. Case Use: Compound Analysis for ENCo* 33 Achieved Outcome: Using the data that we generated, ENCo was able to… - obtain conclusive and reproducible data within their allotted timeframe; - apply for multiple patents in a timely manner; - ultimately moved forward with further development of lead compounds revealed during the project. Animal Model Conditions Tested Assays Utilized Figures/Table s Generated Project Time Project Cost Patent Applications 1 15 $45,500 12 4 13 1 + ENCo ANALYSIS: BY THE NUMBERS C. elegans weeks USD
- 34. INTRODUCTION Understand Your Goal DESIGN Create A Personalized Catalog LOGISTICS Finalize Your Project Design REPORT OUT Maintain Clarity and Transparency Case Use: Compound Analysis for ENCo* 34 MARKETING MATERIALS PRESS RELEASE INVESTOR MEETINGS FUNDING ROUNDS GRANT PROPOSALS PATENT APPLICATIONS DEVELOPMENT PIPELINE WHITE PAPERS SCIENTIFIC PUBLICATIONS
- 35. InVivo Biosystems 35 In-Vivo Biosystems is the market leader for early in-vivo testing using small animal models to gain a better understanding of the efficacy, mode of action, toxicity and potential targets of novel compounds. Right Model. Right Test. Right Insights.
- 36. InVivo Biosystems 36 LEARN MORE www.invivobiosystems.com 1.844.663.8749 Right Model. Right Test. Right Insights. anna.malinkevich@invivobiosystems.com yoanne.clovis@invivobiosystems.com support@invivobiosystems.com