Small Molecules Translation Roadmap

Where are you up to with your Small Molecules project? Use the icons below to learn about the steps needed for successful translation of small molecules and what UCL support is available at each stage of the process.

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.

Therapeutic Design & Development

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 & Validation

Target Identification involves selecting biological targets that interact with your small molecule. Once identified, the target and appropriate assay must be validated.

Lead Discovery

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.

Lead Optimisation

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.

Preclinical Development

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.

Clinical Development

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.

Commercial Adoption

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.