The lecture content from Weeks 1 to 8 will provide you with knowledge regarding how potential therapeutics are developed and translated into clinical trials in the biotechnology industry. The lectures are integrated with specific case studies to support your learning with real-world examples. In this assessment, you will be tasked to use this knowledge, and survey the existing literature on similar case studies, to develop your own potential new therapeutic. Ideally, this will initially involve the selection of a specific disease which has a key "driver" that is targetable by pharmaceuticals. This may be a disease without current treatments available, or an existing disease where treatments are available, however there is scope for further therapeutic improvement. You will select this disease by Friday of Week 4 and confirm this selection with the Unit Coordinator. Upon approval to proceed, you will develop a written proposal that addresses the following sections:

Part A: Who is your target patient population? What are the key characteristics of the disease that this therapeutic is designed to target?

Part B: What pre-clinical models of the disease would you propose to use to test your therapeutic?

Part C: Describe the pre-clinical toxicity study that you may propose for your therapeutic?

Please note the following as a guide:

• The entire written proposal should be from 1,500 to 2,000 words in length.
• Accurately describe the disease that you are proposing, including the specific characteristics of the disease that your proposed drug is targeting. This may be a genetic abnormality (e.g. gene translocation or mutation), abnormality in the blood serum biochemistry (e.g. excessive protein or lipid in the blood) etc.
  
• The text should be prepared using a standard word processing software (e.g. Microsoft Word) with the appropriate use of figures, diagrams or tables as required and saved as a PDF.
• Specific referencing to scientific literature is essential. APA or Harvard style is acceptable, and the use of Endnote is highly encouraged.
  

The assessment of this written report will be based on the demonstrated knowledge of disease mechanisms and therapeutic targets, rationalisation and justification of your arguments and ideas, support of these arguments and ideas using appropriate robust scientific literature and clarity of the proposal with accurate referencing.

A detailed marking rubric will be available on the Moodle site for this unit.

Laboratory-based (preclinical) testing of pharmaceuticals is essential to demonstrate the desired efficacy and biological activity in the treatment of various diseases. Employers within the biotechnology and medical research industries commonly use cell-based models of human disease, and will be looking for graduates with a sound knowledge and practical training in such techniques. Hence, you will be assessed in this unit regarding your performance in undertaking these cell-based techniques, as described below.

During the Residential School, you will be provided with a pharmaceutical agent that has been recently discovered to have activity against cervical cancers. The hypothetical biotechnology company you are employed in has recently identified this agent from a plant that is indigenous to Australia, and currently has little knowledge of how this drug works as an anti-cancer agent. Your task during the Residential School will be to undertake a series of experiments (as outlined in a Laboratory Manual provided on the Moodle site) to explore the anti-cancer properties and mechanism of action of this new potential treatment for cervical cancer. The below techniques will be used during the Residential School:

• Culturing of human cervical cancer cells
• Flow cytometry
  
• Immunofluroescence microscopy
• Quantitative real-time PCR

Sufficient training on both the practical and theoretical components of these procedures will be provided during the Residential School. You will complete each of these cell-based techniques (as outlined in the Laboratory Manual on the Moodle site for this unit) and will be assessed on the quality of the outcomes of these procedures. The assessment will be conducted as follows:

• The unit coordinator will undertake the cell-based techniques in parallel with you as a benchmark of quality of the experimental output.
• If your first attempt at the technique does not meet the minimum passing criteria, you will be allowed a subsequent maximum of (two) attempts at this technique.

You will be assessed on the following criteria:

• Aseptic culture techniques
• Quality and interpretation of scatterplots from flow cytometer
  
• Quality of microscopy images
• Quality of quantitative real-time PCR results
• Analysis and interpretation of real-time PCR data
  
• Adequate labeling of experimental samples

Detailed marking rubrics will be available on the Moodle site for this unit. Each technique will be scored as per the rubric, and minimum of 70% of the total marks are required to pass this assessment.

Biotechnology companies are required to provide adequate documentation of their laboratory activities. Laboratory records are the primary source of evidence provided when ownership of biotechnology company's intellectual property (IP) is contested in a court of law. Hence, you will be assessed on your competency to accurately record your experimental findings during the Residential School. You will undertake the cell-based techniques as outlined in the Laboratory Manual (available on the Moodle site for this unit). You will be tasked to record your daily activities, results, observations and conclusions in this manual. This will also include information relevant to preparation for the techniques, including calculations involved in the preparation of solutions or reagents. There will also be daily worksheets provided to you regarding compliance with Standard Laboratory Practices in a PC2 Laboratory (Physical Containment Level 2 Laboratory). 

You will be required to complete the Laboratory Manual in conjunction with the discussions held during Residential School and the associated reading available on the Moodle site for this unit. Submission of the completed Laboratory Manual can be performed as hard copy at the completion of Residential School, or online through the Moodle Assessment Submission Site.

You will be assessed on the following criteria:

• Appropriate use of an introduction for each experiment
  
• Accuracy in laboratory recording, including the methodology used and documentation of results
• Quality and appropriate use of labeling and images or tables for presentation of results
  
• Correct spelling and appropriate use of technical terms
  
• Demonstrated knowledge of the regulatory laws, government guidelines or institutional committees that govern biosafety, animal and human ethics

Marks will be allocated within the Laboratory Manual for each section, and a detailed marking rubric will be available on the Moodle site for this unit.

Presentation of your research findings at scientific meetings or conferences will ensure dissemination of your discoveries to the broader scientific community. In this assessment, your task is to present research findings on the specific anti-cancer drug that you were using during residential school. The oral presentation of your research findings will begin with your experimental results that you achieved during the residential school. You will then present a series of logical subsequent experiments and anticipated results to conclusively determine the mechanism of action of this relatively unknown anti-cancer drug. You will refer to recent scientific literature in this field to design these subsequent experiments in an effort to draw robust conclusions on the mechanism of action of this drug. It is expected that the volume of experimental findings presented (and proposed) during this presentation will be sufficient for publication in a scientific journal (approximately 6 to 8 figures).

Please note that the presentation can be between 10-15 minutes in duration. It can be recorded and uploaded into the Moodle Assessment Submission Site. It should follow the below sections:

• Introduction regarding the discovery of the drug and target patient population.
• Presentation of methods used at Residential School
• Experimental results achieved during Residential School
• Presentation of proposed subsequent experiments and anticipated results
• Conclusion drawn from existing results (from Residential School) and from proposed experiments

The presentation can be prepared using any standard presentation software (for example, Powerpoint or Keynote). Links to recording software and instructions for how to record the presentation will be available on the Moodle site.

You will be assessed on the following criteria:

• Clearly introduce the background information regarding the drug and accurately identify and describe the characteristics of the target patient population.
  
• Accurate and clear presentation of methodology (both from existing protocols used at Residential School and subsequent proposed experiments).
  
• Coherent and logical presentation of results in tabular or graphical format. 
• Clear presentation of microscopy images with accurate labeling.
• Logical discussion of results with accurate and definitive conclusion outlined.
• Appropriate use of referencing of scientific literature

A detailed marking rubric will be available on the Moodle site for this unit.