Small Molecules Translation Roadmap
This roadmap provides a simplified workflow summarising key activities and considerations for development, evaluation and commercialisation of small molecules, to facilitate their effective translation into the clinic.
The Therapeutic Design & Development phase of small molecules translation should involve an understanding of the patients needs in order to identify opportunities for clinical benefit. Additional considerations include the Target Product Profile (TPP) and Intellectual Property (IP).
Target Identification involves selecting biological targets that interact with your small molecule. Once identified, the target and appropriate assay must be validated.
Once a target is known and an assay for activity has been developed, it is necessary to search for lead compounds that can potentially interact with the target.
In lead optimisation, the aim is to refine the lead properties to enhance required biological activity and reduce the potential for unwanted off-target side effects. This includes physicochemical, solubility, ADME, toxicity potential and pharmacokinetics properties.
Final candidate molecules needs to possess a well-define set of properties before they are considered suitable for testing in humans, including chemical, physiochemical, pharmacological, pharmacokinetic and safety and toxicity potential properties.
Once the preclinical small molecules compound is selected, the data is collated to support an approval of a Investigational New Drug (IND) application. This application is given to a regulatory body, so that the compound can move forward into human clinical trials.
This section involves some capabilities beyond UCL (such as manufacturing and post-release monitoring), however we do have a number of teams that can get you to this position.