CONSORTIUM CALL TOPICS 2025

• The role of neuroinflammation and cell stress/survival as a mechanism for therapeutic intervention in neurological diseases (neurodegeneration, multiple sclerosis, cerebral infarction) (Pfizer, MSD, Shionogi, Takeda, Astex, J&J Innovation, Eisai, Eli Lilly and Company)
• Novel diagnostic approaches for early and cost-effective identification of neurodegenerative disease (Pfizer, MSD, Shionogi, Takeda, Astex, J&J Innovation, Eisai, Eli Lilly and Company)
• Technology challenge that highlights the Neuroscience theme: ‘Beyond antibody’ approaches to directly target proteinopathies in neurodegenerative disease, MASH, IBD, SLE, LN, Type 1 diabetes and celiac disease (Eisai, Takeda, Astex, J&J Innovation, Pfizer, Astellas, MSD)
• Innovative validation of neurodegeneration or oncology targets using novel technologies such as machine learning and multiomics and/or complex model systems (Astex, J&J Innovation, Pfizer, MSD, Eli Lilly and Company)
• Loss of integrity in the aging blood-brain barrier – peripheral immune signalling to CNS glia (Takeda, MSD)
• 3-D culture systems to analyse role of target modulation for neurodegenerative disease (Takeda, MSD)

• Chronic inflammatory diseases especially IBD, Asthma, COPD and IPF (immune regulation, Treg biology, myeloid cells, structural cell – immune cell interactions, complex human in vitro models and/or a strong translational focus in these areas) (Sanofi, Astellas, J&J Innovation, Pfizer, MSD)
• In vitro/in vivo models of dermal inflammation. Emphasis on the role of sebocyte/fibroblast interactions and interactions with the human skin microbiome (Sanofi, J&J Innovation, Pfizer, MSD)
• The role of the small intestinal microbiota in triggering and maintaining inflammatory diseases, e.g. in Crohn’s disease and Celiac disease (Ferring, Sanofi, Astellas, J&J Innovation)
• Innate Immunity (Astellas, AstraZeneca), Innate pathways relevant to chronic inflammatory disease (Sanofi, J&J Innovation)
• Clonal hematopoiesis-related diseases (Astellas, J&J Innovation)
• Technology challenge that highlights the Immunology and Inflammation theme: ‘Beyond antibody’ approaches to directly target proteinopathies in neurodegenerative disease, MASH, IBD, SLE, LN, Type 1 diabetes and celiac disease (Eisai, Takeda, Astex, J&J Innovation, Pfizer, Astellas, MSD)
• Technology challenge that highlights the Immunology and Inflammation theme: Platform to identify biomarkers, or identified biomarkers of early detection, patient segmentation and prognosis prediction for target diseases (MASH, IBD, SLE, LN, T1D, celiac disease) utilizing patients’ samples and data (Astellas, MSD)

• Host response to infectious diseases (Shionogi, Astellas)

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• Innovative validation of neurodegeneration or oncology targets using novel technologies such as machine learning and multiomics and/or complex model systems (Astex, J&J Innovation, Pfizer)
• Clonal hematopoiesis-related diseases (Astellas, J&J Innovation)

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• The role of the small intestinal microbiota in triggering and maintaining inflammatory diseases, e.g. in Crohn’s disease and Celiac disease (Ferring, Sanofi, Astellas, J&J Innovation)

• New route of vaccine administration (on IgA enhanced prophylaxis basis) (Shionogi)

• Cellular reprogramming of cells, e.g. immune cells, other cell types (Pfizer, Sanofi, Astellas, J&J Innovation, AstraZeneca)

• Protein homeostasis: Improved and novel targeted approaches for protein degradation, Molecular Glues, New E3 ligases (cell and tissue specific), Degrader Antibody Conjugates (Pfizer, Eli Lilly and Company, Astex, Astellas, J&J Innovation, AstraZeneca, Eisai, MSD)

• Platform technologies: Editing: (a) Epigenetic editing, (b) Gene editing, (c) RNA editing and application in novel therapeutics, (d) In vivo base editing approaches for cell / gene therapies (J&J Innovation, Astellas, Pfizer)

• Enabling systemic exposure/long-term bioavailability of macromolecules following oral exposure (J&J Innovation, Shionogi, Sanofi, AstraZeneca)

• Improving oral availability of macrocyclic peptides via mechanisms that do not rely on absorption enhancers (Eli Lilly and Company, J&J Innovation)

• Induced proximity pharmacology (‘TAC’ mechanisms such as PROTAC and related modalities): assays, reagents, tools, modelling, chemistry and/or pharmacology (technology) (Astellas, Astex, J&J Innovation, Pfizer, AstraZeneca, Eisai)

• AI/ML in Discovery chemistry and biology (technology) (Astellas, Sanofi, Astex, J&J Innovation, Pfizer, AstraZeneca)

• AI technology that can accelerate structure-activity relationship research (Shionogi, Sanofi, Astex, Astellas, J&J Innovation, Pfizer)

• Novel linker strategies for ADCs (antibody-drug conjugates, including antibody-RNA) to improve delivery of cytotoxic or therapeutic molecules (Eli Lilly and Company, Astellas, J&J Innovation)

• RNA therapeutic platform (small RNAs and mRNA): (Eli Lilly and Company, Sanofi, Astellas, J&J Innovation, AstraZeneca):

a) Ways to reduce RNA uptake into the liver
b) Understanding fundamental drivers of transcytosis and exocytosis to increase delivery across and not into barrier cells
c) Identifying receptors to enable RNA delivery into specific cell types
d) Where does the RNA go once inside delivered into the cell?
e) How to optimize release from endosome
f) Understand RNA Structure and Function & Interactions with Small Molecules: use of Machine Learning & Artificial Intelligence
g) Replicating RNA-small molecule binding seen in vitro, in cellular conditions

– Downstream effects of RNA binding, such as inhibition of protein expression or increase of protein expression

– Understanding novel technologies used to determine small molecule binding RNA targets in the cell 

– Leveraging current understanding of RNA structure and cellular environment to improve selectivity 

h) Protein upregulation via small molecules RNA binding effect

• Ways to innovate beyond first generation mRNAs and non-viral nanoparticles: (Eli Lilly and Company, Astellas, Sanofi, AstraZeneca)

a) Is it possible to make chemically-modified mRNAs to improve stability vs serum nucleases
b) Polymeric nanoparticle (PNP) engineering for precise size, shape, charge, and deformability control. Making PNPs as physically distinct from lipid nanoparticles (LNPs) as possible could open up targeting tissues beyond the liver
c) Demonstrate efficient (& safe) delivery/(transfection) of mRNA to extra hepatic tissues (could be extension of 2b or with polymers or via different non viral delivery system)

• Platform to identify disease-causing somatic mutation, or identified somatic mutation as new therapeutic targets utilizing patients’ samples and data (non-oncology diseases) (Astellas)

• Platform to identify biomarkers, or identified biomarkers of early detection, patient segmentation and prognosis prediction for target diseases (MASH, IBD, SLE, LN, T1D, celiac disease) utilizing patients’ samples and data (Astellas, MSD)