NCATS i3D-Rare Workshop
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About the Workshop
BACKGROUND: Most rare diseases still lack approved treatments despite major advances in research and technologies. Recently, the FDA Modernization Act 2.0 was signed that allows for alternative to animal testing, including cell-based, 3D tissue and in silico models, for purposes of drug and biological product applications. The development and use of 3D tissue models provide a huge opportunity to accelerate the therapeutic development for rare diseases. 3D tissue models, also called microphysiological systems, are enabling the creation of patient-derived assay platforms for drug testing and the generation of physiologically relevant functional and genomics data to help make in silico models more predictive. To help deliver on this opportunity, the National Cancer for Advancing Translational Sciences (NCATS) is looking to work with the biomedical community to accelerate the creation of a platform of 3D tissue models generated from iPSCs (i3D-RARE) to use as testing assays for discovery and for the faster development of new treatments for rare diseases.
i3D-RARE PROJECT GOAL: To develop and operationalize a platform of validated rare disease 3D cellular models (spheroids, organoids, bioprinted, tissues chip) using iPSCs to accelerate the development of personalized therapeutics for rare diseases.
OBJECTIVES OF i3D-RARE:
- Create a portfolio of validated rare disease iPSCs (patient derived and CRISPR edited), corresponding tissue relevant iPSC-differentiated cells, and iPSC-derived 3D organoids and tissue models.
- Establish comprehensive OMICS profiles of the rare disease iPSC-derived cellular models.
- Operationalize the use of the iPSC-derived 3D cellular models with functional assays for predictive efficacy and toxicity testing of therapeutic candidates, including gene therapies, gene targeted therapies such as antisense oligonucleotides, and small molecules.
- Develop and validate AI-based computational predictive models of therapeutics efficacy and toxicity from multi-omics and functional assays data generated.
- Disseminate acquired knowledge and material to publicly accessible databases, repositories, and publications.
OBJECTIVES OF THE WORKSHOP:
The workshop is a landscaping exercise to inform on gaps, bottlenecks and challenges in the space of iPSC-derived 3D cellular models to accelerate the development of therapies for rare diseases, determine where NCATS could play a catalytic role in advancing this field, identify the type of investment NCATS should make, engage and coordinate other stakeholders, and receive feedback on the original objectives of the initiative from the community.
Agenda
WORKSHOP PRODUCTS:
Primary: Workshop report outlining major needs and areas of synergy among various stakeholders.
Secondary: Position paper on the best path forward when it comes to the use of 3D tissue models to advance drug discovery/development for rare diseases
ACCOMODATION REQUEST:
Interpretation services can be requested if needed, by September 7
ORGANIZING COMMITEE: Elizabeth Ottinger, Marc Ferrer, Danilo Tagle, Dobrila D. Rudnicki, Geetha Senthil, Wei Zheng, Catherine Chen, Min Jae Song, Carlos Tristan, Ewy Mathe, Bryan Traynor, Tiina Urv, Samuel G. Michaels, & Anton Simeonov.
