Discovery & Development Europe 2026

• AI in:
o Drug design
o High throughput screening
o Hit finding
• Productivity gains, limits, and evolution of data/models
• Industry case studies on generative AI: what worked, why, and data sources
• Measuring success and identifying next steps for improvement

• RNA is the fastest growing drug modality with huge unrealized potential
• SmartCoat is a complete delivery technology for genetic medicines
• SmartCoat introduces precise solutions for siRNA delivery
• Comparison with current lipid nanoparticle technology
• First indication plan: Targeting incurable cancers and heart disease
• Resolving the endosome escape problem
• SmartCoatTM: Development from SmartCoat prototype to v3.0
• Market entry strategy to maximize SmartCoat value & potential
• Plato Therapeutics Team
• Palto is a compelling investment opportunity

While the industry races to screen faster and bigger, at Iktos we’ve chosen a different path - we don’t screen, we design. Using generative AI, we create novel molecules tailored to the desired properties from the start. The true challenge, however, lies in making what we imagine at a reasonable cost and speed. By combining AI-driven design with synthetic feasibility prediction and automated chemistry, we’ve bridged the gap between virtual and real molecules. In this talk, we show how this approach redefines drug discovery - with tangible, made-in-the-lab examples.

• During lead optimization, PBPK models incorporating human in vitro parameters can predict the oral bioavailability of drugs, if the mechanisms limiting the oral exposure of a drug are known.
• This presentation describes how PBPK models can be used for hypothesis generation to identify mechanisms limiting drug exposure in preclinical species.

Emerging modalities and genetics driven target selection have reshaped our early discovery portfolio whilst business pressures insist on cycle time compression. AstraZeneca’s response is a synergistic toolbox of affinity based screening approaches whose complementary strengths accelerate internal hit discovery. This presentation introduces the toolkit, how it’s deployed, and the impact it’s having.

IAM1363 is a potential best-in-class type II HER2 inhibitor with broad activity across HER2 mutants, >5,000-fold selectivity over EGFR, preferential tumor enrichment, and robust brain penetrance. Enabled by Iambic’s AI/ML and high-throughput platform, IAM1363 is currently being evaluated in a Phase 1b clinical trial.

• Improving the efficiency and effectiveness by implementing the FAIR guiding principles for scientific data
• Tools enabling FAIR data
• Fair as a machine learning & AI enabler
• Benefits of FAIR in pharma

The size of the virtually explorable chemical space is expanding due to the application of novel digital workflows. Effective selection of quality monomers, scalable information encoding, and search engines for multi-modality knowledge extraction are keys to explore >billion-size collections at low cost while extracting hits with desirable profiles. This talk will focus on how we create and explore our chemical know-how with Nebula, AbbVie’s tailored chemical space platform.

• This presentation will outline four alternative future scenarios for synthetic origin molecule research.
• It will describe a breakthrough scenario enabling rapid druggability of all biological targets and accelerated development timelines.
• It will present a pragmatic scenario where progress continues incrementally without major technological disruption.
• It will examine a constrained future where geopolitical pressures limit innovation and redirect research priorities.
• It will explore a disruptive convergence of biology and digital technologies driven by AI and non-traditional players.
• It will conclude by highlighting key uncertainties and factors shaping the future of small molecule discovery.

• Multifactorial diseases: cancer, neurological disease (Parkinson's, Alzheimer’s), microbial illness
• Multiple Targeting Drugs/Ligands (MTD) approach: Hybrid and Chimeric Drugs
• Azaheterocyclic derivatives for multifactorial diseases: design, synthesis, and biological activity of hybrid and chimeric derivatives

• Integrating PAT, digital twins & AI for continuous workflows
• Real-world impact: productivity gains and decision-making power
• Balancing innovation with regulatory compliance

• Exploring what we really mean by neuroinflammation
• Highlighting key cellular and immune features involved in brain inflammation
• Considering how clearer definitions could guide future research and treatment

Most clinical ADCs rely on TOP1 or tubulin payloads, creating risks of overlapping resistance and shared toxicities. This talk presents Ryvu’s strategy for mechanistically distinct payloads designed to overcome resistance, expand the therapeutic window, enable combinability and support differentiated next-generation ADC programs across oncology indications and partnerships.

o An introduction into the tech stack of Ignota Labs
o Causal mechanistic understanding of Gefitinib drug-induced-liver-injury
o Deep assessment of how genotypes contribute towards individual patient toxicity and resistance.

Small molecule therapeutics continue to form the backbone of therapies for all types of life-threatening diseases. The last decade has seen considerable progress in delivering precision inhibitors which provide clinically meaningful benefit for patients suffering from cancer. In the desire to balance efficacy and tolerability candidate profiles become more sophisticated and resulting molecular design challenges more complex.
In my lecture I will discuss examples of impactful small molecule drugs with a focus on underlying design principles and enabling technologies. I will outline design challenges and emerging solutions for novel small molecule drug modalities including molecular degraders and antibody payloads.

High-throughput technologies provide a robust foundation for the discovery of novel lead structures. At Bayer, we have developed advanced platforms for high-throughput quantitative PCR (qPCR) and thermal shift assays (TSA). Case studies demonstrate how these technologies empowered successful hit identification. In addition, strategies for designing diverse and effective compound libraries are discussed, highlighting their critical role in successful screening campaigns.

Gal3 is a secreted protein know to induce inflammation and amyloid aggregation in the brain. It is expressed in microglia with the DAM phenotype, and secretion mediates neuronal cell death. We hypothesise that inhibiting or degrading Gal3 may be protective in Alzheimer’s disease, and could represent an effective way to maintain microglial homeostasis in the brain.

DNA-encoded Library (DEL) screening is widely recognized across academia and industry as a powerful hit-finding platform. This presentation will feature Amgen’s DEL screening platform and include examples of DEL applications within Amgen’s small molecule portfolio.

Functional genomics is exploding in drug discovery, making it hard to turn diverse, complex as-says into actionable insights. We addressed this at Novo Nordisk via an end-to-end data platform that guarantees compliance with a FAIR-by-design approach and unlocks immediate value through frictionless access, enabling confident decision-making from trusted data.

This talk introduces ready-to-use cardiac organoids as scalable human-relevant 3D models for drug discovery, highlighting cardiac safety assessment, functional evaluation, and toxicity testing for predictive preclinical workflows.

QUANTRO developed a Transcriptomic Discovery Platform that matches time-resolved transcriptional profiles triggered by small molecules with those from acute target degradation. This approach identifies direct inhibitors, modulators, and degraders of previously undruggable transcription factors and signaling nodes, with active discovery campaigns against c-MYC and YAP/TEAD.

• Introduction to Lundbeck External Research & Innovation
• Recent examples of building partnerships with Lundbeck

Molecular glues are attractive as potential targeted protein degrader therapeutics – but rational discovery has proven difficult. In this work, we will discuss multiple computational techniques implemented in MOE (Molecular Operating Environment) for modeling ternary complexes containing molecular glues.

• The future of partnering & what to prioritise
• Bringing together AI & data cross-functionally to drive drug discovery

Autonomous Mobile Robots (AMRs) transform research by automating routine tasks and extending operational hours to nights and weekends. We are implementing our first AMR, integrating liquid handlers, incubators, and readers for fully automating any microtiter plate assay in our Lab of the Future. This pioneering solution will boost productivity and enhance research outcome

Chemical biology connects biological insight to therapeutic opportunity. This talk highlights how chemical probes and targeted protein degradation, including PROTACs, interrogate protein function in cells to support target discovery and validation. Examples show how orthogonal perturbations reveal mechanisms, clarify causality, and uncover biology beyond inhibition, informing targets, leads, and biomarkers.

We present the first published DNA‑encoded library selection against an ion channel, enabled by detergent‑free Salipro® lipid nanoparticles. Using PANX1, we identify functional small‑molecule modulators validated by SPR, cryo‑EM, and electrophysiology, demonstrating a robust DEL workflow for challenging membrane proteins.

• Tailoring IP portfolio architecture to therapeutic modality (from small molecules to complex biologics)
• The IP paradox in AI-driven drug discovery (inventorship, confidentiality, ownership)

• Expanding target scope
• Resistance mechanisms
• Clinical pipeline readiness
• New effectors for TPD
• Safety & toxicity concerns

Real-world data work in pharma is bottlenecked by tasks that don't need
expert judgment — they need expert time. Evidence synthesis takes weeks.
TLF review takes days. Protocol drafting takes months.

We built the RWD AI Agent Platform: one workbench, many agents. Chat,
deep research, PubMed + web grounding, slide generation, code sandbox,
and 20+ specialist agents for ATTR-CM, RWE, pharmacovigilance, evidence
strategy, TLF review, and protocol writing — all behind a single UI with
human-in-the-loop review on every output.

The bet is breadth + speed: a shared platform so any new use case becomes
a new agent in days, not a new project in quarters. Results so far — 80%
less data wrangling, 10× faster literature review, evidence
consolidation from days to one hour.

Our principle: prototype first, perfect later. The tool isn't precious —
the researcher's time is.

While large strides have been made towards de novo design of molecular glue degraders (MGDs), library screening of cereblon ligands remains essential. We present here our MGD discovery platform that combines a hypothesis-driven second-generation molecular glue library with high-throughput deep proteomics screening. The screening data for >4,000 compounds will be discussed and exemplified with several degraders of novel neosubstrates with non-canonical ternary complex binding mode, as determined by cryo-EM.

We have cutting‑edge AI but ten‑year‑old data habits. This talk shares real lessons from a 3,000+ parameter tech transfer journey: why tools alone don't fix fragmented knowledge, how a process‑first mindset and data readiness are essential, and why connecting a knowledge platform to manufacturing systems is a trust and culture challenge, not a technical one.

Targeted protein degradation is an exciting and growing modality in drug discovery. This talk will explore the influence of natural protein half-life on the level of protein degradation observed with degraders, which can pose significant hurdles in screening for short-lived proteins.

Competitive molecular glues hold great promise to tackle challenging intracellular targets, but known examples were largely identified serendipitously. Here we present systematic approaches for the discovery of FKBP-assisted molecular glues and present the CellTrap effect as a general mechanism to enhance the cellular potency of these modalities.

• PK, safety & biomarker data
• Clinical relevance of models
• Leveraging literature vs datasets
• Personalised medicine
• Regulatory considerations

- Trifluoromethyl groups have historically been key functional groups to optimize compound series in drug discovery
- However, their persistence in the environment has raised concerns about their impact on sustainability
- We show how we can finetune the stability of trifluoromethyl groups to be stable enough for medicinal chemistry while not persisting prolonged time in the environment

• How investors evaluate emerging drug discovery technologies
o AI in drug discovery
• What drives funding decisions in a shifting biotech market
• Where capital is flowing next
• What drives founding decisions?

Microcrystal electron diffraction (MicroED) enables rapid structure determination of sub-µm crystals using an electron beam. Multipoint MicroED allows selective analysis of individual crystals within mixtures. This talk highlights its utility and high-throughput workflows for accurate solid-form identification.

The presentation covers why partnerships are important and gives an overview of the key steps to a partnership addressing some do's and don'ts.

Maximising AI’s impact in drug discovery requires practical tools that empower scientists. It also necessitates a focus on the implementation of proven algorithms and organisational change. Using case study examples from our StarDrop and Cerella platforms we show how AI’s value can be demonstrated.

CDD Vault is a secure, cloud-based platform that unifies chemistry and bioassay data across ELNs, registration systems, and analysis tools to drive reproducibility, collaboration, and AI/ML readiness.

• Challenges: Physical and Logical Data Silos in Development Sciences
• Solutions: Introduction of a Federated Infrastructure & Data Standardization through “Data Contracts” and a global Taxonomy

- Non-Degrading molecular glues inhibit protein targets through formation of a ternary complex. In this talk, our development of macrocyclic molecular glues that recruits FKBP12 will be discussed in context of inhibiting PPIs and other target classes.
- The structural basis for inhibiting PPIs will be shown through X-Ray structural data of ternary complexes with disruption of the TRADD-TRAF2 PPI being detailed.
- Learning from structural based drug design using ternary crystal structures will be presented.

• Bicycle Therapeutics is a clinical-stage company developing constrained bicyclic (Bicycle®) peptides.
• Properties of Bicycle® molecules suit precision therapies and could overcome challenges of targeting EphA2 – a valuable target in oncology characterized by Ph1 failures.
• Nuzefatide pevedotin, a Bicycle Drug Conjugate®, shows a differentiated preclinical profile and is currently in Ph2

Automation at AstraZeneca has evolved beyond its original High Throughput Screening purpose to support a broader range of scientific assays. By adopting an agile and collaborative approach, we have successfully repurposed existing automation platforms to accelerate projects, maximize laboratory footprint, and reduce the need for future capital investment.
This presentation will explore a practical case study demonstrating how internal automation expertise and cross-functional collaboration enabled the end-to-end automation of biocatalysis workflows, delivering enzyme-driven drug compound synthesis in a faster, more sustainable, and cost-effective way.

• Oncology leads the way in genome-based medicine with estimates of at least 90 FDA approvals requiring biomarkers for optimal patient selection – almost half of all drugs authorized since 1998
• In discovery, ‘Drugging the Cancer Genome’ is being further enabled by technologies such as CRISPR, chemical probes, structure/fragment-based design, degraders (PROTACs and molecular glues), immune-targeting and AI/machine learning approaches
• In clinical development, use of the ‘Pharmacological Audit Trail’ framework is being enhanced by cheaper next generation sequencing, liquid biopsy, and innovative PK/PD modelling

Targeted protein degradation presents a significant opportunity to tackle previously undruggable targets by utilizing the cell's proteasomal system to eliminate disease-causing proteins.
At Merck Healthcare KGaA, we have established specialized workstreams for bifunctional PROTACs and molecular glue degraders.
Examples of our hit identification from PPI, Del and hit characterization will be presented, highlighting our tailored assay suite to optimize these modalities in our drug discovery pipeline.

• How to make and de-risk decisions in drug discovery -target/indication prioritisation
• Accelerating discovery with AI-driven technologies
• Potential bottlenecks & future outlook

Protein kinases have been pursued as drug targets for over 20 years, but a significant part of the human “kinome” has not yet been addressed with specific inhibitors, despite its likely medical relevance. Our research group focuses on the identification of new covalent inhibitors for both validated and understudied protein kinases.
In our recent work, we and our collaborators have employed intact-protein mass spectrometry (MS) screens to explore kinase-inhibitor-like covalent libraries equipped with cysteine and tyrosine/lysine reactive “warheads”. These screening efforts have yielded a variety of promising hits, which were validated by complementary methods, including thermal shift assays, biochemical enzyme assays, cellular assays, and X-ray crystallography.
My presentation will detail our progress in extending the range of known covalent protein kinase inhibitors, especially focusing on new compounds targeting lesser-studied kinases. Additionally, I will discuss non-conventional binding modes we have identified, providing a basis for ongoing medicinal chemistry optimization.

• Antibody engineering to improve toxicity
• Balancing potency vs tolerability in payload design
• Will novel formats improve the toxicity profile?

Human translatable cellular models — iPSC-derived neurons, cardiomyocytes, and kidney organoids — are transforming pre-clinical research by bridging the gap between genomic target discovery and therapeutic development. Combined with CRISPR screening, automation, and ML-driven phenotypic profiling, these models enable scalable, disease-relevant target validation that improves translational predictivity across therapeutic areas.

Discovery of novel antibody fragments (scFvs), optimised for bioconjugation, design of hydrophilic linker-payloads, novel format drug conjugates for oncology

Our efforts to reimagine preclinical safety screening with humanized ACM, multiparametric data, and AI will be discussed

We present practical applications of AI-driven strategies for hit identification, including virtual screening, make-on-demand library mining, compound prioritization, and exploitation of data-rich screening decks, alongside iterative screening workflows and automated hit expansion.

• Bridging silos between devices, data platforms & teams
• Achieving interoperability: tech, teams, and standards
• Scaling automation from proof-of-concept to full lab adoption

Pharma executives and managers are racing to apply AI across drug discovery and development. But while the possibilities of AI advances can be very promising, many efforts falter before bringing home dependable, real‑world performance. This session outlines five essential elements for preparing your data, systems, and scientific workflows for AI success. You’ll learn why strong data governance, complete metadata, interoperable platforms, resilient model‑management processes, and scientific validation are critical for trustworthy, reproducible results. By aligning these elements early, you'll enable faster decisions, fewer surprises, and you'll develop AI tools that deliver more consistent value across the drug development lifecycle.

Aignostics and Bayer have co-developed a multimodal platform for AI-based patient stratification and target identification. By combining pathology foundation models with multimodal learning, it enables reverse translation from real-world tissue to novel, validated oncology targets — accelerating the discovery of therapies grounded in human biology. Here, we present the platform and findings in LUSC .

Cell Signaling Technology (CST) delivers rigorously validated research tools to enable reproducible discovery across basic and translational science. Our industry-leading antibody validation pipeline ensures high specificity and performance across key applications, including Western blotting, ELISA, immunofluorescence, and flow cytometry, supported by advanced proteomics and custom solutions.

Here, we highlight the importance of antibody specificity in enabling robust, high-content, and high-throughput assay workflows. Highly specific antibodies minimize background signal and improve contrast, which is essential for accurate automated image analysis. This directly enhances segmentation fidelity and quantitative readouts, enabling reliable detection of biologically relevant changes.

By integrating validated reagents with scalable assay platforms, these approaches improve data quality, reproducibility, and confidence in downstream analyses. Ultimately, this enables more effective target validation, phenotypic screening, and decision-making, accelerating drug discovery efforts.

Intractable targets (often termed “undruggable”) are proteins or biological molecules heavily implicated in diseases (particularly cancer) that cannot be easily modulated by conventional small molecule drugs. They are considered challenging because they lack defined, deep binding pockets required for traditional drugs to attach and inhibit function. They often involve protein-protein interactions (PPIs), transcription factors, and intrinsically disordered proteins.

Progressing “intractable targets” requires a hybrid approach combining novel chemistry with advanced DMPK to navigate non-Rule of 5 (bRo5) chemical space. Success hinges on employing targeted covalent modification to enhance selectivity and to transform shallow binding pockets into druggable sites, and using degradation technologies to shift from occupancy-based to event-driven pharmacology (degradation) allowing targeting undruggable proteins. While intractable targets require non-traditional chemistry, DMPK is not a downstream filter. Cross-functional integration between medicinal chemists and DMPK scientists is critical to address non-traditional physicochemical properties early.

In this presentation, we will illustrate that ADME strategies traditionally used for selecting reversible inhibitors may not be appropriate for the discovery and development of covalent inhibitors. Non-traditional assays in combination with “adequate” PK profiles are the critical considerations for progression of such compounds. Proteolysis-targeting chimeras (PROTACs) represent an emerging therapeutic modality. Their bifunctional design inherently leads to physicochemical properties that often lie beyond the limits for traditional oral small molecules. We will highlight the key challenges in optimizing oral PROTACs and discuss the need for alternative experimental approaches to accurately characterize their atypical ADME properties.

TBC

• Career journeys and lessons from senior women in pharma, biotech, and academia
• Navigating leadership challenges, mentoring, and fostering inclusive R&D teams
• Breaking silos: cross-functional collaboration to accelerate drug development
• Inspiring the next generation of women scientists and leaders

Affinity selection-mass spectrometry (AS-MS), combined with orthogonal biophysical methods (HTRF and TSA), provides a powerful platform for identifying binders against challenging targets such as membrane proteins and for protein-protein interactions. Integrated approaches accelerate hit identification and enable the generation of first tool compounds for previously intractable targets.

• Drug deposition determines efficacy, yet the local dose at the site of action is rarely quantified during development • Twinhale simulates aerosol transport and local deposition in patient-specific lungs using CT-derived anatomy, breathing patterns, and in vitro aerosol data • The platform enables virtual cohort studies and variability analysis prior to clinical trials • Applications include device selection, dose translation to humans, and supporting bioequivalence or bridging strategies using modelintegrated evidence

We will present a “patient in the lab” patient-derived organoid platform to evaluate preclinical efficacy and safety: oncology biobanks for biologics/ADCs and translational stratification as well as liver organoid profiling for early human-relevant toxicity risk.

PUREfrex® is a rebuilt, cell-free protein expression system that redefines how proteins are produced and explored. Its modular architecture offers researchers precise control at the molecular level, making it exceptionally adaptable to a wide range of applications—from expressing challenging biologics to enabling high-throughput workflows. Whether you're working on therapeutic proteins, novel modalities, or synthetic biology prototypes, PUREfrex® provides a flexible and efficient foundation. When paired with AI/ML platforms, it becomes a powerful accelerator for innovation in protein engineering and discovery.

DDIC, an international network of partners from industry and academia established in 2017, provides the framework for high-level application-oriented research and talent development for the pharmaceutical industry. Thereby the DDiC facilitates the translation of academic research into industrial application in a pre-competitive environment within a broad network of members from academia as well as pharmaceutical industry, equipment manufacturers and excipient providers.

Exploiting modern data science techniques in drug discovery requires platforms that generate data of sufficient scope and quality. DNA-encoded library (DEL) screening is one such data-intensive platform. In this talk, the hallmarks of effective utilization of DEL will be examined, and successful cases studies shared.

• Traditional targeted protein degradation approaches require high affinity binders to E3 ligases to generate PROTACs or molecular glues, which limits the scope of E3 ligases regularly used
• At Amphista we take a target first approach to degrader discovery, generating glue like molecules based on target binding ligands. This has enabled generation of Targeted Glue™ degraders against high value targets using three non CRBN/VHL ligases
• Learnings from our pipeline programs will be presented, demonstrating the advantages of Targeted Glues and our approach to compound optimization

What does it take to move from automated workflows to autonomous R&D cycles?
This talk explores the architectural requirements for lab-in-the-loop concepts and shows how interoperability standards like SiLA enable seamless integration, scalability, and real-time interaction between AI models and laboratory infrastructures.

1. Bayer Co.Lab is Bayer’s global incubator network supporting early-stage life science and biotech ventures
2. With fully equipped labs, shared infrastructure, a global community, and access to experts and partners, it helps founders bring their brilliant ideas to life - while reducing operational and financial overhead
3. With a global footprint of facilities in strategic innovation hotspots Cambridge (USA), Berlin (Germany), Beijing and Shanghai (China), and Kobe (Japan) combined with virtual members from around the globe, we have a portfolio of more than 30 highly innovative startups working in diverse disease and technology areas
4. A dynamic community, the expertise of pharma, and no strings attached

- Use of DEL-ML and eASMS for ligand identification
- Validation workflow of PROTACs

This talk introduces AcoustoFab’s contact-free acoustic levitation platform and its capabilities in multi-material handling and programmable manipulation. It highlights applications in drug discovery, focusing on omics sample preparation and 3D cell culture to improve biological relevance. The presentation also explores powder levitation for handling drug compounds and raw materials, and concludes with perspectives on enhancing efficiency, reproducibility, and innovation across bioanalytical and pharmaceutical workflows.

As AI models become increasingly integrated into drug discovery, predicted protein structures are now used to support target assessment, hypothesis generation, and molecular design. In this talk, we will discuss where experimental protein structures remain critical as reference data.
Drawing on examples from cryo-EM, X-ray crystallography, and structural analysis, we will focus on how experimental data can validate models, resolve uncertainty, and inform structure-guided discovery workflows in the age of AI.

Current in vitro models ignore the mechanical forces that shape every biological process, from tumor growth to neurodegeneration, leading to poor clinical translation and costly drug failures. NeoMag is the first commercial technology enabling non-invasive, programmable mechanical stimulation of cells and tissues. Backed by an EIC Transition grant and validated at UCSF, Imperial College, and UCL, we are building the standard tool for mechanobiology-driven drug discovery, opening a new category in preclinical research.

AI + robotics-powered formulation platform that accelerates development time and reduces cost.
Closed-loop learning from real experiments improves the prediction of solubility, stability, bioavailability, and release.
Multiplexed screening across injectables, orals, and RTU forms reduces trial-and-error and material waste.

• DendroPharm develops innovative nanocarriers for targeted drug delivery and offers GMP manufacturing and formulation development.
• Fields of focus are transdermal therapeutics, veterinary medicine, and innovative excipient technologies.
• Dendropharm´s platform technology enables selective accumulation of drugs in inflamed or tumor tissue, enhancing bioavailability, reducing systemic side effects, improving skin penetration, and preventing opioids from crossing the blood–brain barrier, supporting safer non-addictive pain therapies and precision oncology applications.
• Dendropharm is looking for investors, customers, licensing partners

Pulsed Dipolar Spectroscopy (PDS), a branch of Electron Paramagnetic Resonance (EPR) spectroscopy that focuses on the measurement of interspin dipolar interactions, holds significant value for the investigation of protein-protein interactions (PPIs), capable of elucidating structural constraints, determining dynamic information, and counting coupled spins. Protein oligomerization is a specific type of PPI of great interest to pharmaceutical and drug-discovery industries. Because oligomerization influences and regulates many key protein categories such as enzymes, ion channels, and transcription factors, it plays a vital role in many biological and physiological processes that make appealing targets for therapeutic development. Therefore, understanding the mechanism of interaction is of significant interest to the scientific community.

Here, we apply Double Electron Electron Resonance (DEER), a robust and popular PDS technique, to investigate the dimerization of a homodimeric protein system. The distance distributions extracted from the DEER data are used to gain insight into the structure and dynamics of the dimeric complex, while careful analysis of the modulation depth of the dipolar traces reveals the populations of monomer and dimer states. Furthermore, we investigate the changes in these aspects as a function of buffer conditions and against the introduction of compounds that promote dimerization of the protein. These findings exemplify the capacity of EPR to contribute to questions across the PPI space, and the principles demonstrated here can be easily extrapolated toward other hot-topics within the biopharmaceutical industry, such as induced proximity and targeted protein degradation.

• Drug development is slow, costly, and inefficient, with a 93% clinical failure rate and 59% toxicity in Phase I oncology trials .
• Animal testing has poor predictive power; >200 million animals are used yearly, and the US/UK/EU plan to phase it out.
• Existing in vitro models lack vasculature, throughput, or reproducibility.
• Living Networks introduces the first high throughput vascularized microtissue (<1 mm) enabling 3D tissues with perfused vasculature.
• Platform supports drug screening, automated image analysis, and cancer specific vascular phenotyping.
• Demonstrated PoC screens show distinct drug mechanism dependent vascular responses.
• Team combines expertise in microfluidics, tissue engineering, and software.

Patient-derived microtumours (PDMs), isolated from surgical tumour resections, preserve the native tumour microenvironment, including structural heterogeneity and immune interactions. This platform allows for physiologically relevant, in vitro evaluation of lead candidates, such as advanced therapy medicinal products (ATMPs) and immunotherapies, while supporting the identification of personalised, patient-specific cancer treatment strategies.

- Network architecture for Automation in labs is generally an afterthought
- We did not learn from production, but inherited IT-practices that are not fit for automation and labs
- For AI we need good data quality
- This means: reliable data, full audit trails, cyber security
- For that we need to mitigate against data poisoning and unintentional modifications by staff
- Tools: Expertise in digital infrastructure, networking and software development; signed data; audit trails for methods, scripts, etc.
- For automation: Availability is the main (security) goal. This means no cloud, no internet dependencies, etc.

Closed Door Steering Group Meeting for Drug Discovery

ImmunXnano, a deep biotech spin-off project from the German Center for Neurodegenerative Diseases (DZNE), revolutionizes immunosafety profiling for drugs, vaccines, and nanomedicines.
Our cutting-edge technology uses high-dimensional phenotypic data from primary human cells to provide unprecedented insights into immune system interactions. We enable precise functional profiling of immune activation, cytokine signaling, and toxicity—key factors in predicting therapeutic efficacy and safety.
We empower pharmaceutical and biotech partners to accelerate R&D, reduce risk, and ensure product safety.

This talk explores how protein engineering and optimizing the secretory pathway with signal peptides can improve protein yield, quality, and scalability in mammalian expression systems, enabling more efficient manufacturing of antibodies, vaccines, and emerging biologic modalities

Generative AI streamlines early-phase drug discovery by unifying molecular design and synthetic planning to navigate chemical space more efficiently than traditional methods. Integrated with high-speed experimentation, these frameworks accelerate design-make-test-analyze cycles. To address data limitations, active learning strategically identifies the most impactful experiments, while deep interactome learning ensures the design of novel bioactive molecules with desired properties from scratch. Collectively, these technologies provide a predictive filter that optimizes the path from initial discovery to viable leads by prioritizing the most promising molecular designs.

SARM1 is a key cellular mediator of axonal degeneration and a promising therapeutic target for nervous system disorders such as chemotherapy-induced peripheral neuropathy. We discovered and optimized a novel class of base-exchange inhibitors (BEXi) against SARM1 and evaluated their effects in primary neuronal cell assays. While BEXi effectively suppressed SARM1 activity in vitro and in cells, sub-inhibitory concentrations unexpectedly activated SARM1 and accelerated neurite degeneration. Even at high concentrations, BEXi delayed but did not prevent neurite loss in neuronal cultures. These findings highlight the complexity of SARM1 modulation, emphasizing the necessity for robust, mechanism-based cellular screening in the discovery of neuroprotective therapies.

- An overview of iPSC-based disease modelling at DZNE
- A showcase of combining the genetics of clinical cohorts with iPSC-derived neural models to develop genetic therapies for spinocerebellar ataxias using antisense oligonucleotides
- The establishment of axon-omics for biomarker discovery in motor neuron diseases.

Hypometabolism is a hallmark of neurodegenerative disease, but is it merely a downstream consequence of accumulated pathology, or an active driver of disease progression? This talk explores emerging evidence that metabolic dysfunction may play a causal, and potentially targetable, role in neurodegeneration.

• NIDB-3101 binds two distinct Tau epitopes and has Fc mutations for enhanced half-life
• NIDB-3101 binds a larger variety of Tau entities from AD brain tissue than benchmark Abs and robustly inhibits AD brain-derived seeds induced intracellular Tau aggregation
• The combination of sub-nanomolar affinity and cellular activity together with enhanced half-life leads to a modelled lower human efficacious dose than benchmark

• Pharma-biotech-academia collaboration models that actually work
• When to partner vs acquire vs build internally
• Data-sharing in a competitive landscape
• Public-private partnerships in Europe: success stories & failures
• Regional differences – US vs EU
o Scaling in Europe

In my talk I will give an overview of Servier and highlight our Strategy for rare Neurology. The talk will also dive deeper into two of our Neuroscience Assets that are in clinical development

• Theoretical position for intracellular kinase modulation
• Experimental modelling to validate target/drug probe
• Initial data in patients

Rare pediatric neurological diseases remain one of the most challenging and underserved areas in medicine. While advances in genetic and clinical diagnostics have significantly improved our ability to identify these conditions early and precisely, therapeutic progress has unfortunately not kept pace. For many children and families, treatment options are still largely limited to supportive care rather than disease-modifying interventions. There is a critical need to bridge this gap by developing disease-modifying and meaningful therapies that can alter the clinical course of these disorders. I look forward to sharing perspectives on the current landscape, the opportunities ahead, and the research we are conducting at S. M. Discovery Group (SMDG) on several rare pediatric diseases and our potential First-in-Class genetic medicines.

• Key drivers behind renewed pharma and biotech interest (e.g., Alzheimer’s approvals, novel mechanisms, better biomarkers)
• Understanding the underlying biology of neurological indications
• How can we get better in our understanding the full picture of the diseases?

Brain health conditions affect 1 in 3 Europeans, costing €430bn annually — yet many are preventable and underfunded.
Innovative therapies and cross-sector partnerships are accelerating progress across epilepsy, mental health, and rare neurological disorders.
Policymakers, industry, and society must commit now — investing in brain health saves both lives and public expenditure.

• Integration of iPSC-derived cell models to explore neuroimmune mechanisms and validate emerging targets
• Translational value and challenges of modulating neuroinflammatory targets like TREM2 and RIPK1
• Sex specific differences

The Landmark Project is a large public-private partnership supported by multiple global Pharma to identify disease-modifying drug targets and predictive biomarkers for Parkinson's disease risk and progression. The study is profiling hundreds of post-mortem human brains each with subject-matched CSF across digitized advanced neuropathology and multiple omics (WGS, scRNA-seq, proteomics, lipidomics etc) for genetically-anchored causal inference.

Using iPSC-derived microglia models, this presentation explores neuroinflammatory mechanisms in Alzheimer’s disease. We examine how risk SNPs, including APOE-dependent and independent variants, alter microglial responses, and how female-specific X chromosome aging and escape genes shape disease phenotypes, neuronal survival, and sporadic Alzheimer’s disease risk in females.

Generation of single cell RNA seq altas of iPSC microglia from 20 prodromal AD donors
Microglial states and their transition in response to different challenges
Molecular pathways induced by challenge

Advanced cellular and high-content screening platforms to investigate TDP-43 pathology and splicing dysfunction in ALS/FTD, enabling discovery and validation of novel therapeutic targets and modulators for neurodegenerative disorders

•Introducing a high-capacity microfluidic platform specifically designed for neuroscience drug discovery
•Enabling the study of neurological processes in vitro, such as uptake and propagation of misfolded proteins, synaptic transmission and cellular interactions, or axonal degeneration and regeneration

Rare pediatric neurological diseases remain one of the most challenging and underserved areas in medicine. While advances in genetic and clinical diagnostics have significantly improved our ability to identify these conditions early and precisely, therapeutic progress has unfortunately not kept pace. For many children and families, treatment options are still largely limited to supportive care rather than disease-modifying interventions. There is a critical need to bridge this gap by developing disease-modifying and meaningful therapies that can alter the clinical course of these disorders. I look forward to sharing perspectives on the current landscape, the opportunities ahead, and the research we are conducting at S. M. Discovery Group (SMDG) on several rare pediatric diseases and our potential First-in-Class genetic medicines.

Since the COVID-19 pandemic, ionizable lipids have gained significant attention for gene delivery in gene editing and enzyme replacement therapy. However, most available ionizable lipids were developed for vaccines and optimized for immunogenicity, which limits their suitability for repeated systemic dosing. We present the rational design of biodegradable ionizable lipids that offer improved tolerability and reduced organ/immune toxicity while maintaining robust mRNA expression. Our proprietary lipids exhibit high circulatory stability and degrade selectively upon intracellular stimulation, enabling favorable safety and clearance profiles. This work demonstrates hepatic- and spleen-targeted LNP formulations with potential applications in infectious disease vaccines and repeated-dose mRNA therapeutics. These formulations address key challenges in non-viral RNA therapeutic delivery.

- Can we use NOF’s lipids without a license agreement?

- Can you provide CDMO service for LNP development by using NOF’s lipids?

- What is the competitor lipids in this presentation?

• Novel Oncolytic Peptide therapies for hard to treat cancers
• Membrane-Lytic Peptides and immunogenic cell death
• Reprogramming the local tumour microenvironment for systemic response

• Why intranasal delivery is compelling for small molecules
• Formulation and device considerations unique to intranasal products
• Development, safety, and translational challenges

The phrase “Lab-in-the-loop” is an often-used term. But what does it really mean? How far along are we on our journey towards autonomous science?

This presentation will focus on how Recursion is operating at the interface between the physical world and the virtual world. In particular, the infrastructure required to enable agentic orchestration of the AI-driven, iterative, design-make-test and learn testing that refines small molecule therapeutics.

• Considerations for Patient-Centric Product Development
• Acceleration Strategies for Small Molecule Development
• Trade-off considerations: “Right first time” vs “Fast to patients”

Nova Biomedical collaborated with a leading human‑milk nutrition manufacturer (anonymized for confidentiality).  The Company’s products have touched the lives of more than 125,000 critically ill infants.  Attention to quality is a major area of focus when developing these products that are derived from human donors, with the aim of concentrating the nutritional composition consistently before release.  

To ensure this, the donor and their milk undergo extensive screening processes before being cleared to processing.  A vital step is the testing of the raw milk product.  During this testing phase, the donor milk undergoes a critical osmolality test for product adulteration before being cleared to processing.  With many incoming samples that require this test, an automated approach is needed to ensure workflow efficiency.
The OsmoTECH HT Micro‑osmometer from Nova Biomedical’s Advanced Instruments portfolio enables high‑throughput osmolality testing of up to 192 samples per run, allowing more donor samples to be screened simultaneously while significantly reducing hands‑on operator time. This poster highlights the workflow efficiencies and time savings achieved using the OsmoTECH HT

The utility of ion-pair reversed-phase (IP-RP), anion exchange (AEX), and hydrophilic interaction liquid chromatography (HILIC) methods is assessed for phosphorothioate (PS) diastereomer-pair separation.
The determination of diastereomeric composition in the antisense strand of an siRNA is demonstrated.
The deliberate change of the diastereomeric composition could be made detectable.
In manufactured batches of the same sequence comparable diastereomer is demonstrated.

• Expanding theranostic applications
• Complex manufacturing, isotope supply constraints, and short half-life logistics
• Regulatory hurdles, GMP compliance, and specialised infrastructure requirements

Recent technological advancements & platforms
Non-viral platforms
Brain-shuttles
Predicting Future Successes: What Is Next for Modalities in CNS Diseases?
Clinically relevant biomarkers and new in-vitro assays

This panel will explore the scientific and technological challenges of formulating oral GLP-1 therapies, including absorption enhancement, stability, bioavailability, and patient-centric design
• Formulation strategies
• Emerging delivery platforms
• Translational hurdles
• Future directions shaping next-generation oral peptide therapeutics

The presentation will discuss the challenges and opportunities in formulating biologics. In particular, it will focus on the use of ionic liquids to address bioavailability and solubility challenges in high-concentration subcutaneous biologics. It will also cover how effective ex vivo assessment models can accelerate formulation discovery and development.

- Oral peptide delivery is constrained by GI proteolysis and unfavourable physicochemical properties
- Unnatural amino acids enable systematic expansion of peptide design space
- A pancreatin assay quantifies susceptibility to intestinal degradation
- Integrated physicochemical data guide rational design for oral stability and bioavailability

• Studying the effect of different co-surfactants on stabilising the niosomal membrane
• Investigating the influence of niosomes' compositions on formation of a pH gradient
• Visualisation of pH gradients via pH indicator bromocresol green (BCG) as a novel encapsulated model molecule to visually investigate the ability of niosomes to entrap drugs through active loading
• Application of the most optimised BCG niosomal formulation to encapsulate a therapeutic anticancer drug molecule via pH gradient active loading

• Non-animal based in vitro models for efficacy
• Digital twin approaches for in silico clinical screening

• Stabilising complex modalities
• Evaluating non-traditional formulation strategies — including lyophilisation, co-formulation, microencapsulation, or amorphous systems

Retention and delivery of biologics to the anterior and posterior of the eye remains challenging due to ocular anatomy. Topical application of a hydrocolloidal formulation achieves therapeutic delivery of anti-VEGF to the eye posterior, potentially overcoming the need for needle-based delivery. Additional applications and possible mechanisms will be discussed.

• Key scientific breakthroughs enabling oral GLP‑1 (permeation enhancer, targeting the stomach)
• Translational challenges and clinical proof-of-concept
• Next-generation strategies for improving efficacy and scalability

High concentration monoclonal antibody (mAb) formulations present major development challenges, including high viscosity, aggregation, reduced solubility, and liquid–liquid phase separation. Arginine Glutamate (Arg·Glu, A 172) has emerged as a multifunctional excipient capable of mitigating these issues by modulating protein–protein interactions. In this session, we highlight data demonstrating Arg·Glu’s effectiveness in lowering viscosity and improving colloidal stability across diverse mAb formats. Comparative studies show that Arg·Glu provides superior anti aggregation performance and a more favorable safety profile than Arginine Hydrochloride. A focused case study further illustrates its ability to enhance stability under stressed conditions. Overall, Arg·Glu represents a promising excipient for enabling robust, patient friendly high concentration mAb formulations.

Modern drug development continues to face significant challenges related to solubility, stability, and bioavailability of emerging therapeutic compounds. Emerging formulation strategies designed to address these limitations are highlighted, including recently FDA-approved products enabled by Captisol® technology. The FAST (Feasibility Assessment/Solubility Testing) Program is also introduced as a streamlined approach for early-stage screening, enabling rapid evaluation of drug/cyclodextrin interaction and more informed formulation decision-making.

A central focus is the TOPAZ™ formulation platform, a novel low-water system that enables expansive compositional flexibility and the creation of diverse formulation architectures. This platform facilitates improved solubility and stability while supporting multiple routes of administration, including oral and topical delivery. A case study illustrates its effectiveness in addressing challenging molecules, demonstrating enhanced solubility, improved taste-masking, and increased bioavailability observed in preclinical evaluations.

Collectively, these approaches represent an integrated framework for accelerating formulation development, mitigating technical risk, and broadening the range of drug candidates that can be successfully advanced toward commercialization.

This presentation explores conformational binding between different types of biomolecules using Microfluidic Modulation Spectroscopy, highlighting how molecular-level binding insights drive modern biopharma and accelerate protein, peptide, and RNA drug development at scale now.

The pharmaceutical development pipeline recently surpassed 50% biologic APIs in active programs. Innovative delivery methodologies for biologic APIs are therefore increasingly valuable to improve patient compliance, efficacy, and reduce off-target effects. This presentation will focus on applications of biologic APIs delivered via non-invasive dry powder inhalers to the nose and lung.

A wide range of indications can be addressed via respiratory delivery: Local treatment of lung diseases such as COPD, lung cancer and pulmonary fibrosis is of great interest. For small biologics, such as peptides or short RNA, non-invasive systemic delivery can also be achieved via the highly-vascularized lung and nasal tissues. Both pulmonary and nasal mucosal vaccines offer the potential to confer immunity at the primary site of infection in respiratory diseases. Finally, an emerging area of interest involves nasal delivery of biologic APIs through the olfactory nerve to bypass the blood-brain barrier.

Spray drying enables the production of engineered particles suitable for respiratory delivery, including delicate APIs such as peptides, proteins, and nucleic acids. When formulated with appropriate stabilizers, buffers, and excipients, these powders can achieve long-term stability at 25 °C, reducing reliance on cold-chain distribution. Case studies will illustrate formulation and manufacturing approaches for proteins and nucleic acids delivered as dry powders to the respiratory tract.

• Democratisation of M&S: From expert tools to self-service simulation app
• Empowering formulation scientists with validated first-principle models
• Enabling virtual experiments before lab work to accelerate development

Our mission is to develop high-performing lipid nanoparticles for delivering therapeutics and vaccines. I will discuss our digital-first approach to designing novel ionizable lipids, optimizing formulations, and planning experiments to meet customer needs, with a focus on molecular modeling and integrated high-throughput experimentation in our labs.

• Challenges of GLP-1 formulations ensuring stability, permeability, solubility enhancement and overall effectiveness of the final product
• Key formulation strategy to enhance oral administration of peptides

ImmTAX biotherapeutics redirect T cells to target disease-specific antigens with high potency and specificity. This presentation outlines their mechanism of action and our formulation development strategy, emphasising early optimisation of physical, colloidal and chemical stability. We discuss trade-offs and demonstrate how ultra-low dose requirements are enabled through IV dilution workflows.

• Advanced biophysical characterisation to understand degradation pathways and stability risks
• Integration of data science and predictive modelling to forecast long-term stability
• Accelerating developability assessment and formulation decision-making through in-silico insights

• Crystal defects have been shown as the primary cause of nitrosamine formation in ranitidine hydrochloride
• Recrystallisation of ranitidine hydrochloride can essentially stop the formation of nitrosamines on storage by eliminating crystal defects
• This technology can be used in conjunction with traditional nitrosamine-reducing approaches to further mitigate nitrosamine formation for all crystalline drugs

• Direct precipitation was selected to allow production of manageable Drug Substance (DS) amorphous
• Continuous DS processing implemented although scale-up and materials challenges resulted in the need for a mitigation plan
• Aiming to strengthened DS–Drug Product interface by generating consistent physical attributes

• Challenges and opportunities in developing high-dose antibody formulations suitable for subcutaneous administration
• Overview of innovative formulation and delivery approaches for high-concentration biologics
• Key considerations related to manufacturability, scalability, and regulatory expectations for next-generation antibody therapeutics

- Tailoring formulation strategies for complex biologics, RNA therapeutics & emerging modalities
- Overcoming formulation and stability challenges in established and next-generation therapies
- Advancing cross-functional development across formulation, process & analytical development

Formulating sensitive or complex molecules often requires mitigating oxidation risks, ingredient incompatibilities, poor solubility, or achieving targeted delivery. In this presentation, we will share real life examples of complex formulations and demonstrate how selecting the right capsule technologies provided practical and robust solutions to these challenges.

We will cover:
• a case study using lipid based systems and permeation enhancers to enable oral delivery of a therapeutic peptide
• strategies to handle ingredient incompatibility through Cap in Cap technology
• achieving targeted enteric delivery without post filling coating using Capsugel® Enprotect® capsules
• customization pathways offered through our Innovaform® Accelerator center

Together, these approaches represent scalable and flexible strategies to unlock challenging formulations across modalities.

Drug development can feel like detective work – especially when a molecule changes character unexpectedly. Through the lens of Dr Jekyll and Mr Hyde, this session uncovers the clues hidden in solubility, stability, and solid state behaviour that only strong preformulation practice can reveal. Come for the literary twist; stay for the science that saves programs time, cost, and complexity.

Achieving effective absorption and therapeutic efficacy is critical for the success of oral small molecule drugs. To optimize performance, a deep understanding of key biopharmaceutic properties—such as solubility, dissolution, and membrane permeability—is essential, as these parameters directly impact bioavailability and clinical outcomes.
This session will present innovative in-vitro methodologies designed to enhance developability assessments of small molecules and emphasis will be placed on dynamic dissolution and absorption models, which, when integrated with mechanistic modeling, provide valuable insights into rate-limiting steps in oral absorption. Selected case studies will illustrate the impact of food on oral drug absorption, the possible benefits of using nanoparticle formulations, and the impact of dose level on altering the mechanism for oral absorption when comparing pre-clinical to clinical outcome.

• Introduction to enhancer based delivery of macromolecules
• Our hypothesis around PDE inhibitors for improving bioavailability of oral peptides
• In vitro selection of formulations
• Preclinical in vivo testing and selection of lead formulation
• Clinical testing of the lead formulations

- Pre formulation is the Compass
- Establishing the QTPP: Pre-Formulation as the Knowledge Foundation
- Mapping Molecular Behaviour: Early-Stage Definition of Baseline CMAs
- Initial Risk Assessment (RA)
- Intrinsic Chemo-metrics vs. Solid-State Phases [CRAMSN Crystal Engineering Case Studies]
- Constructing the Design Space
- The Ultimate Control Strategy

Next Gen Drug Delivery at MERCK KGaA developed a unique system for activation and subsequent immobilization of binders at LNP surface for cell specific oligonucleotide delivery. This Site-specific Protein Optimization for Targeting (SPOT^TM) technology allows for the generation of LNPs with outstanding targeting and transfection efficiency in vitro and in vivo.

• Designing for delivery early: integrating molecule, modality, and delivery strategy from preclinical stages
• Route of administration as a strategic lever: choosing and optimising IV, SC, oral, and emerging routes for real-world use
• Breaking delivery barriers: tackling stability, bioavailability, targeting, and manufacturability challenges
• From innovation to implementation: which delivery technologies (AI, automation, digital tools) are truly improving clinical and commercial outcomes

-xPhore is a peptide based platform amenable to deliver most nucleic acid modalities, including siRNA, mRNA, circRNA and DNA
-xPhore delivers RNA/DNA to sites of inflammation without liver sequestration
-Modularity allows application in different indications

In this work we share new insights into how siRNA off-targets can be identified and assessed.

• Definition of oral biologics and delivery challenges
• Clinical and market landscape: a peptide-dominated reality
• Key biopharmaceutical barriers: stability and permeability
• Enabling technologies for oral peptide delivery
• Clinical case studies and lessons learned
• Strategic implications and future outlook

Integrating high-throughput screening with advanced kinetics modeling enables efficient, robust predictive stability modeling for glycoconjugate vaccines and this can be applied to other modalities as well. This approach uses minimal (analytical) resources, with high correlation between rapid assays and traditional methods, supporting accelerated drug product development when long-term data are scarce.

This presentation explores how modern analytical techniques move beyond traditional bulk measurements to reveal individual particle properties. Through real case studies, it is shown how morphology, wall thickness, and particle level distributions provide deeper insight into spray dried and tabletted materials, enabling better formulation decisions, process understanding, and performance prediction.

1. In your experience, where have bulk powder properties been sufficient for decision‑making—and where have they clearly failed to predict clinical or manufacturability performance?
2. Which individual particle attribute do you believe most strongly impacts downstream pharmaceutical performance: morphology, internal structure, or particle‑to‑particle variability—and why?

It is particularly important for biopharmaceutical companies developing and distributing fragile biomolecules to ensure the stability and activity of their products during long-term storage and shipment.
In accordance with quality by design (QbD) principles, advanced kinetic modelling (AKM) has been successfully used to predict long-term product shelf-life, relying on Arrhenius-based kinetic models built from data generated in short-term accelerated stability studies. In the case presented, AKM was used to support formulation selection. AKM predicted 3-year HMWs formation using only 6-month stability data on several prototypes. This predictive approach allowed to make the best-informed decision on the maximal concentration for commercial formulation lock in a context of significant aggregation compound.

Combination Products in the drug delivery has been evolving over time with traditional and more advanced delivery modalities adapting to treating patient’s needs. We will explore this evolution, highlight some advancements in drug delivery modalities and look at the changing landscape.

• Exploring the foundation of combination products
• Reviewing the evolving landscape and advancements in drug delivery
• Highlighting non-traditional routes of administration

• Multiphysics-based models
• AI/ML-based models
• Synchronisation with the physical counterpart

Multiphysics simulations are highly effective for developing pharmaceutical processes, but their computational cost limits scalability. Physics-Informed Neural Networks (PINNs), trained on multiphysics simulation data, can retain comparable detail and complexity while being far less computationally intensive, enabling faster development and highly scalable process modeling.

For large emerging modalities such as antibody–oligonucleotide conjugates with intracellular targets, efficacy is constrained not by tissue distribution or cellular uptake, but by intracellular availability at the site of action. We present an approach enabling productive intracellular delivery, linking exposure, cellular uptake, and endosomal escape to on-target delivery and functional target engagement, reframing delivery as the key determinant of pharmacological activity.

Closed Door Steering Group Meeting for Formulation

Innovative mRNA formulations and delivery approaches
Improving selectivity and efficiency of delivery
Utilising data driven approaches to cover come delivery challenges

For large emerging modalities such as antibody–oligonucleotide conjugates with intracellular targets, efficacy is constrained not by tissue distribution or cellular uptake, but by intracellular availability at the site of action. We present an approach enabling productive intracellular delivery, linking exposure, cellular uptake, and endosomal escape to on-target delivery and functional target engagement, reframing delivery as the key determinant of pharmacological activity.