PHAR2010: Drug Discovery- Portfolio Making Assignment

Internal Code: MAS1935

Portfolio Making Assignment:

1.The class is divided into groups of 2-5 students to prepare a portfolio (app. 10 pages per group member; max. 60 pages) and a final ppt presentation of max. 15 minutes (max. 16 slides) to be given in class in the last week of SP5. 2.Each group has been given at least one paper describing the role of a potential drug target for the treatment of a disease. This information will be used as starting point to develop a plan for the foundation of a start-up company. 3.Collect additional information from literature and the internet to address the following points in your portfolio: 4.Describe the disease (e.g. Alzheimers) and the therapeutic area (e.g. neurodegenerative diseases) -current treatment options; adverse effects -unmet medical needs -market consideration; financial burden; number of patients 5.Describe your target -molecular mechanism and link to disease (animal models; genetic defects) -protein structure of target -target class; closely related targets with potential for specificity problems -other potential targets -patent situation -novel, first in class, orphan disease, “me too” 6.Assays -design assay for primary screening strategy -compound selection (e.g. diverse library, focussed library, natural products etc.) 7.Verification of primary hits -design assay for follow-up (verification, dose response studies) -design assays for specificity testing if necessary 8.Animal model -identify suitable animal models to reach early proof of concept (ePOC) •Compound profiling for prediction of adverse effects -Drug-drug interaction -ADME / Tox -DMPK 9.Address potential problems for development -preclinical development -clinical trials / biomarkers Notes for Portfolio 10.You must not address every single point of this list. It is up to you to decide which topics are of central interest for your target and which parts you can keep short. 11.You also may add new themes which are specific for your project. 12.Your final document must have a table of contents and a list of correctly annotated references. Notes for Final Presentation 13.It is suggested that every opportunity be taken to observe others when they give oral presentations. Think critically about where they are standing, speaking speed, voice volume, eye contact, reading directly from slides. 14.Make sure your presentation is not too long. Cut down some of the material presented if necessary. 15.Slide formatting Font •Sizes between 20 and 28 are preferred. If there is insufficient space for at least 20 font, then split any specific slide into two, then increase the font size for half the information on each. 16.A non-serif font is easier to read (e.g. Arial, Calibri etc.), although a serif type font is probably better for large documents or books (e.g. Times New Roman). 17.Background format Choose a good background to fit with writing, figures, schemes and diagrams. -Practice There is no substitute for practice. Some may not have to practice as much as others. 18.It is always clear when much practice has been done, or not done. Even the most nervous person can overcome nervousness with sufficient effort. General notes -SI units and SI formatting SI units should be used almost all times. One obvious exception (the only exception?) in medicine is blood pressure, which continues to be expressed as millimetres of mercury (mm Hg). 19.Note that there should be consistency for use of standard units. If abbreviations are to be used (much preferred) then they should be introduced at first use and from there on always be used for all units. For the portfolio it might be useful to include a table of abbreviations. -English language Try to stay clear of jargon or common expressions that are used in some informal speech. For example, avoid the use of “stuff like that”, “and stuff”, “gunna”. Despite common usage, it is preferred to say “data are” rather than “data is” or “data were” rather than “data was”.