WC13 2025: Hesperos CEO Presents “Utilization of human on a chip systems for rare disease regulatory submissions”

Track:  Accelerating rare disease therapeutics via adoption of human biology-based non-clinical drug development in global north and global south countries
Human Health – Replacement

Session Chair

Session Description
Rare diseases represent a significant disease burden and impact up to 10% of the world’s population. In particular, south Asian countries experience higher rates of rare genetic disorders which has been attributed to traditional marriage practices of endogamy and consanguinity. Despite this, there are no approved treatments for almost 95% of rare diseases. While the use of genetically engineered animal models is traditionally considered as one of the main strategies for rare disease therapeutics, these model systems have often proven unsuitable for providing human-relevant insights into the patho-mechanisms of rare diseases. Recently, efficacy data from a microphysiological system developed using human cells were used to support the authorisation of a clinical study for chronic inflammatory demyelinating polyneuropathy, a rare neuromuscular disease. This, and several other examples, highlight the potential of complex in vitro models (CIVMs), including induced pluripotent stem cell (iPSCs), organoids, and organs-on-chips, to provide personalized model systems for understanding the biological mechanisms and assessing drug efficacy for rare diseases.

This workshop will include two sessions. The first session will aim to provide a global perspective on the status quo of the use and development of complex in vitro models to support drug development of rare diseases. We will hear from experts from around the world who have successfully used CIVMs to demonstrate drug efficacy and learn more about the challenges faced across different global regions. This would include talks on successful case studies that have used CIVMs to test the efficacy of repurposed drugs and progress to clinical trials, general overview of how industry is currently using these systems in their internal drug development pipeline for rare diseases. The session would also include two talks from global south country-perspective where the higher cost of rare disease treatments also renders it inaccessible to most of the population, highlighting the need for his would be followed by a session where it would be discussed how these insights from rare diseases could be used to develop a generalised framework for efficacy testing for other disease categories. We hope to develop a white/status paper covering the recommendations that emerge from these sessions.

Session organization (90 minutes)

Session 1: Human preclinical models for rare disease therapeutics- Status quo and challenges (45 minutes)
1. Introduction– How CIVMs can provide a personalized system for efficacy testing, general statement of needs or challenges (Suggested Speaker: James Hickman, Hesperos Inc. – we have reached out to Dr Hickman and he has shown interest, yet to accept)
2. Case study – Developing efficacy models for cystic fibrosis using lung-on-a-chip (Suggested Speaker: Roberto Plebani, Emulate. Inc)
3. Accelerating the development of CIVMs for rare disease therapeutics in India: Not for profit speaker (Suggested speaker: Surat Parvatam, HSI/India)

Session 2: Breakout session on developing a generalized framework for incorporating CIVMs for efficacy testing based on insights from rare diseases (45 minutes)
This breakout session will focus on discussions on how strategies and insights from efficacy modelling for rare diseases can assist in creating a generalised framework for incorporating CIVMs into efficacy modelling of other disease models.