General Information

Workshop Mission Statement

Target Validation for Non-Addictive Therapeutics Development for Pain


Despite advances in the understanding of pain physiology there has been a paucity of novel analgesic medications developed to address the still high unmet need of pain patients especially those who rely on addictive opioid medicines for pain relief. The successful development of novel non-opioid, non-addictive therapeutics requires advances in preclinical target validation that demonstrate sufficient human disease relevance to trigger investment in non-clinical development and subsequent clinical trials to obtain proof of biology and early proof of efficacy before registrational studies.  Translating novel target discoveries to the clinic depends on the quality of target validation and the rigorous implementation of research operating plans to provide compelling data supporting a causative link between the modulation of molecular target(s) and a disease phenotype associated with the disease state.

Overall goal

Publish guidelines and best practices that provide a roadmap for target validation that can guide teams in the discovery of novel therapeutics for pain. The framework emerging from this meeting will cover important stages in target identification and validation. The sessions will discuss the opportunities presented by cutting edge tools, technologies and modalities that can now be used in vitro and in vivo to address gaps and challenges in the discovery of novel analgesics.

Overall scope

The full target validation process covers target identification to proof-of concept efficacy in human subjects in clinical trials. This workshop will focus on the key preclinical experimental validation package components that de-risk and provide rigorous rationale for the pursuit of novel targets with minimal side effects and a reduced potential abuse/addiction liability into drug discovery campaigns.

The focus will be on the stage appropriate data required to justify further preclinical and clinical therapeutics development activities across the translational spectrum.  Several perspectives will be presented including: industry, academia, and venture capital.

Non-clinical development strategies and early clinical trial designs will not be part of this workshop.


        Breakout Session A - Human Evidence for Target Validation

  • Evidence from the human genome. Genotype:phenotype studies to identify and validate gene-dose effects for targets associated with pain conditions in specific patient populations.
  • New methodologies (as for ex. computational system biology and omics approaches) to link target(s) to pain phenotype(s).
  • Hypothesis of the pathophysiology of a disease, protein structure-function insights and assessment of target druggability potential.
  • Selection of a target validation strategy to provide early assessments of target engagement and modulation coupled to pain circuitry.

        Breakout Session B - Tools and Models to Test Hypotheses in vitro

  • In vitro models (human preferred) that mimic the disease phenotype and/or the molecular mechanism hypothesis.
  • Known analgesics or bio/chemical probes to validate and differentiate molecular hypothesis and the mechanisms.
  • Development of predictive, efficient, and reliable modulatory and delivery toolboxes to modify the desired and unwanted expression or activity in cellular physiologic relevant models.
  • State-of-the art technologies to monitor and characterize the downstream physiologic read outs with robust sensitivity, reproducibility, and specificity.

        Breakout Session C - Tools and Models to Test Hypotheses in vivo

  • Development of predictive, efficient, and reliable modulatory and delivery toolboxes to modify the expression or activity of a target in vivo.
  • State-of-the art technologies to monitor and characterize the downstream phenotypic or mechanistically relevant in vivo read outs with robust sensitivity and reproducibility.
  • Test effectiveness in assays of disease physiology and potential safety concerns.

Version: 2.1.0