Discovery & Development Europe 2026

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

• 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

Advanced manufacturing technologies, such as 3-dimensional printing (3DP), for pharmaceutical dosage form development and manufacturing are gaining increasing attention and interest from the pharmaceutical industry, academia, governments, and regulatory authorities around the world. This is because of the potential of these innovative approaches to uniquely solve drug delivery challenges, decrease development time, improve quality through the use of process analytical technologies (PAT), develop personalized medicines, automate compounding, and operate in non-traditional supply chain models (e.g., distributed manufacturing).

Aprecia pioneered the use of 3D printing in pharma by obtaining FDA approval of the first and only 3DP drug product. Since then, new innovations have advanced the technology, and other companies are using different 3DP modalities to support clinical programs and drug product manufacturing in hospital settings.

This presentation will provide an overview of the current 3DP landscape for pharmaceutical drug product development and highlight the different modalities being used today. The principle of operation and advancement of binder-jetting 3DP will be discussed in more detail, including application of PAT to monitor product quality, product and data traceability, and process control. Selected case studies that highlight unique applications of 3D printing will be discussed, and the presentation will conclude with a consideration of future opportunities and challenges.

- 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

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

• What makes advanced therapeutic modalities inherently difficult to formulate and deliver?
• Overcoming key biological barriers: stability, targeting, immunogenicity, and intracellular delivery
• Case studies across diverse modalities:
o Cell & gene therapies; ADCs; biologics and emerging complex modalities
• Lessons learned from clinical successes and setbacks across modalities
• Where current formulation and delivery strategies are still falling short—and what’s needed next

• 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.

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.

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.

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.

• 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

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.

-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

Closed Door Steering Group Meeting for Drug Discovery