Discovery & Development US 2026
Hilton San Diego Bayfront, CA
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Agenda
Programme
Day
Discovery Of First-In-Class Selective ARID1B Degraders For The Treatment Of ARID1A Mutant Cancers
Industry PresentationLeveraging Foghorn's discovery platform and structure-based design, our team identified several selective ARID1B binder series, which were then used to develop VHL- and CRBN-based molecules that induce robust ARID1B degradation via the ubiquitin-proteasome system. This presentation will provide an overview of the assay cascade that drove the program from binder optimization through the advancement of heterobifunctional degraders, delivering compounds with on-mechanism activity, high selectivity, and downstream transcriptional modulation. This work provides proof-of-concept for ARID1B degradation as a promising therapeutic strategy and offers a blueprint for targeting previously intractable chromatin remodelers.
The Power Of Partnerships: Aligning Pharma, Biotech, And Academia To Accelerate Development & Innovation
Panel Discussion Conference Room 4• Bridging cultures and goals
• Shared resources and expertise
• Accelerating translation
• How partnerships can streamline moving discoveries from the lab to clinical development and market
Automation & AI: Examples From Recursion
Industry Presentation Conference Room 2Multi-Agentic AI For Drug Discovery and Development
Industry Presentation Conference Room 6AI for drug discovery has largely focused on individual tasks such as target identification and molecular design, using machine learning, generative models, and, more recently, generative AI. A major limitation of many AI approaches, however, is that they remain point solutions addressing only one part of the drug discovery and development process. This presentation will explore how multi-agent AI systems can integrate multiple specialized tasks into a coordinated, end-to-end approach to drug discovery and development.
Breaking The First In Class Barrier In Small Molecule Drug Discovery
Keynote Conference Room 1Preclinical model validation of a first-in-class small molecule at a novel target can be treacherous: old disease models may not be suitable for new drugs. Orphagen’s experience in drug development with orphan nuclear receptors in rare oncology and immunology sheds some light on the pitfalls.
The Target Validation Frontier: Multi-Omics And AI In Drug Discovery
Panel Discussion Conference Room 1• Standardization and integration of omics datasets across platforms and labs
• Balancing computational predictions with experimental validation
• Challenges in scaling multi-omics for high-throughput target ID
• Regulatory considerations for omics-informed drug development
• Where AI fits in multi-omics pipelines
Foundation Models For Chemistry: A New Paradigm In Hit Discovery And Lead Optimization
Industry PresentationSolving The Right Problems – Finding AI’s Value In Drug Discovery
Panel Discussion Conference Room 2• How to use AI/ML to solve drug discovery problems
• Success metrics
• Overcoming validation hurdles
CNS Drug Repurposing Using AI/ML Frameworks
Industry PresentationDealing With High Manufacturing And Product Costs In The Gene Therapy Space
Roundtable Discussion Conference Room 2Delivering Therapies Across The Blood-Brain Barrier
Panel Discussion Conference Room 4• Developing optimized BBB models for drug discovery
• Predicting future
• Recent technological advancements & platforms
• Challenges and unities for the delivery of biopharmaceuticals
• Using nasal to brain route
Novel Hit Identification Techniques
Industry Presentation Conference Room 1Technological Advancements For The Discovery Of Complex Therapeutic Targets
Industry Presentation Conference Room 1New therapeutic modalities are transforming what constitutes a drug target. Targets may now encompass protein combinations, molecular complexes, treatment-induced cell states, and spatial relationships between tumor and immune cells. This talk will illustrate how integrated omics, proteomics, spatial biology, real-world clinical data, AI, and functional screening enable scientists to identify, measure, and engineer these increasingly complex therapeutic opportunities.
Challenges In Lead Discovery For Viral Macrodomains: When Standard Cell Models Fail
Industry PresentationIn this talk, a successful effort to discover leads targeting a viral macrodomain will be detailed. In particular, the inability of standard cellular models to fully capture relevant interactions of virus and host cell presents a challenge for this class of targets. The approaches used to overcome this limitation will be discussed.
Astellas's TPD Strategy and Updates from the KRAS Degrader Pipeline
Industry Presentation Conference Room 1An overview of Astellas’ targeted protein degradation strategy and portfolio, highlighting recent progress from the KRAS degrader ASP3082 and the development of our pan-KRAS degrader program ASP5834.
Pushing The Boundaries Of Target Discovery In 2026 & Beyond – New Technologies, Approaches, Industry Directions
Panel Discussion Conference Room 1• The evolution of modalities
• When to pursue small molecules, ADCs, protein degraders, RNA modalities, or multi-modal approaches
Applications Of High-Throughput Data Collection In AI/ML
Keynote Conference Room 2Quantitative Adaptive Libraries, or 2nd Gen DELs, enable training of high-resolution binding models. ML-design, synthesis, and screening of these DELs give models the sampling density, and diversity needed to help drive medicinal chemistry campaigns.
Multicellular Perturbation Models For Drug Discovery
Industry Presentation Conference Room 2• AI architecture for virtual clinical trials and phenotypic in silico drug discovery
• Building TRAILBLAZER, the first AI foundation model built specifically for multicellular biology, capable of simulating patient-level treatment outcomes
• Case studies of phenotypic in silico drug discovery and modeling multicellular patient responses to treatments
Generative AI-Guided Selections for GPCR Antibody Discovery
Industry Presentation Conference Room 1What if GPCRs could be engineered to behave like soluble proteins? Using generative AI, we design soluble GPCR surrogates that express at high yield in human cells, retain native ligand binding, and are experimentally validated for structural fidelity. This approach transforms historically challenging membrane targets into tractable reagents for drug discovery.
Integrating Human-Centric Biology, AI, & New Approach Methodologies For Phase 1 Clinical Trials
Industry Presentation Conference Room 1The Degrader & Glue Landscape In 2026
Industry Presentation Conference Room 1Novel Models For ADME-Tox Research
Industry PresentationAdvancing Multimodal Omics Data In Drug Discovery
Industry Presentation Conference Room 1AI In Drug Discovery
Industry Presentation Conference Room 1Accelerating The DMTA Cycle Through AI Adoption And Automation
Industry Presentation Conference Room 2- Drug discovery follows the design–make–test–analyze cycle.
- AI/ML is transforming data analysis and molecular design.
- Automation can accelerate compound synthesis and testing.
- Together, they can close the DMTA loop and achieve efficiencies at an unprecedented scale.
From Screening to Pharmacology: Lead Optimization of CDK2 Molecular Glue Degraders
Industry Presentation Conference Room 1CCNE1 amplification/overexpression, and activation of CDK2, is associated with poor prognosis in multiple cancers. CDK2 inhibitors have advanced into the clinic;however, poor selectivity against other CDKs have limited their benefit. CDK2 molecular glues provide improved selectivity and potentially clinical benefit. Using Plexium’s TPD platform, we identified selective CRBN-based CDK2 degraders.
Where Smart Capital Meets Science: Investing In The Next Wave Of Drug Discovery Innovation
Panel Discussion Conference Room 4• 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
• How investor are looking at non-dilutive founding
AI-Driven Autonomous Labs - What Is Feasible Today?
Industry Presentation Conference Room 1AI-driven drug discovery labs have automated individual wet-lab workflows, but fragmentation remains: rigid scripts, siloed data, and manual coordination across functional islands. We present LabClaw, an agentic operating system built on OpenClaw that shifts lab automation from isolated robotic tasks to autonomous, end-to-end orchestration. Five domain-expert agents turn natural-language hypotheses into executed protocols, spanning target generation, LIMS checks, hardware control, and closed-loop analysis. In an anti-aging target-validation case study, one query became a fully executed 11-step SOP with only three human checkpoints, letting a single scientist supervise 10+ parallel, self-correcting workflows running 24/7.
Beyond The Hype: AI, Data & The Industry Shift In Drug Discovery R&D
Panel Discussion Conference Room 4• How industry dynamics are reshaping pharma R&D strategy & decision-making
• Digital transformation, AI & data-driven labs of the future
• What leaders need to prioritize to turn innovation into real scientific & business impact
Challanges Of Adopting NAMs: Regulatory & Technical Bottlenecks
Roundtable Discussion Conference Room 4• Navigating regulatory uncertainty, validation standards, and industry confidence when integrating NAMs into development pipelines
• Overcoming technical hurdles including model reproducibility, data translation, scalability, and cross-platform comparability
Hit Finding And Characterization In Covalent Drug Discovery
Industry Presentation Conference Room 1- Strategic application of complementary approaches for covalent hit finding in vitro and in cells
- Chemoproteomics applications for hit characterization and determination of novel ligandable sites on targets of interest
- Library considerations for covalent hit finding at Novartis
What If DNA No Longer Set the Timeline?
Panel Discussion Conference Room 4DNA is foundational to advanced therapies, yet traditional plasmid production can introduce delays before development has truly begun. Drawing on more than two decades developing nucleic acid medicines, Kate Broderick, PhD, will explore how cell-free synthetic DNA can remove that early constraint—enabling faster iteration, greater design flexibility, and a more direct path from promising science to scalable manufacturing and, ultimately, to patients.
From Innovation to Impact: Market Strategies Driving the Next Wave of Drug Delivery Technologies
Panel Discussion Conference Room 4• What actually defines a “successful” delivery innovation in the market?
• From lab breakthrough to commercial product: where do most strategies fail?
• Pharma vs biotech vs academia: who owns the pathway to market?
• Market access and reimbursement as the real gatekeepers of innovation
Zetasizer Core: The New Standard In High-Throughput Protein Stability Screening
Solution Provider Presentation Conference Room 1Rapid biotherapeutic candidate selection demands comprehensive stability profiling from minimal sample volumes. Introducing the Zetasizer Core, a high-throughput microplate platform designed to transform protein characterization.
By unifying Dynamic Light Scattering (DLS), Static Light Scattering (SLS), and Differential Scanning Fluorimetry (DSF) into a single automated workflow, it simultaneously measures hydrodynamic size, aggregation state, colloidal and thermal stability directly in standard microwell plates.
This integrated approach dramatically increases throughput, reduces consumable costs, eliminates analytical bottlenecks, and delivers high-density biophysical datasets. Discover how the Zetasizer Core empowers scientists to accelerate candidate ranking, streamline formulation development, and make critical development decisions with confidence.
Solution Provider Presentation
Solution Provider Presentation Conference Room 1How To Accelerate Research With AI Scientists And Multi-Agent Orchestration
Solution Provider Presentation Conference Room 2Submission required
The Use Of Quantum Mechanics In The Future Of Drug Discovery
Roundtable Discussion Conference Room 1• Implementing AI to aid in generative design
• Looking to the future
Validation & Regulations For NAMs
Industry PresentationFrom Genetic Association To Allosteric Tractability: A Connected-Evidence Framework For TYK2 Target Validation
Solution Provider Presentation Conference Room 1Most drug candidates fail in the clinic, and much of that attrition traces back to a target selected on incomplete or poorly connected evidence. This talk argues that the constraint is no longer data volume but data connection. Validation confidence depends on how rigorously independent lines of evidence are linked and stress-tested, and on treating drug ability and developability as validation criteria applied before commitment rather than downstream surprises. Using CAS BioFinder®, we present target identification and validation as one connected workflow, grounded in a real example: the validation of TYK2 in immune-mediated disease. We trace a path from a naturally occurring, partial loss-of-function protective variant (P1104A) that shields carriers from lupus and multiple sclerosis, through well-mapped JAK/STAT pathway biology, to the insight that allosteric inhibition of the regulatory pseudo kinase (JH2) domain confers selectivity over related JAK kinases. This is a convergence that no single data type could establish alone. Together, these threads illustrate how systematically integrating genetic, pathway, and structural evidence strengthens target rationale, exposes weak hypotheses earlier, and reframes tractability and developability as first-class criteria in the validation decision itself.
Advancing Drug Discovery With Human iPSC Neuroscience Models
Solution Provider Presentation Conference Room 3This presentation focuses on how human iPSC-derived CNS cells are improving the modelling of neurological disease and CNS drug discovery. A central theme is the development and functional relevance of NAM enabling assays and custom assay development.
How To Use NAMs
Panel Discussion• Organoids and organ-on-chip models
• In vitro disease modelling
• Toxicity and safety screening
• Disease biology
• PK/PD and toxicity modelling
• In silico models
• Implementation and application of NAMS
Solution Provider Presentation
Solution Provider Presentation Conference Room 1Deeper Biophysical Characterizations Through Electron Paramagnetic Resonance: A Complementary Tool For Biopharma’s Toughest Questions
Solution Provider Presentation Conference Room 1Electron Paramagnetic Resonance (EPR) is a powerful biophysical tool complementary to many of biopharma’s best assets, probing biomolecular structure and dynamics, supramolecular complex arrangements, equilibria and kinetics, and endogenous metal binding sites. Through High Q’s FATHOM® platform for EPR spectroscopy, we present key case studies demonstrating the power of the methodology, the information it can acquire, and the answers it can help inform.
Cell Free Antibody Engineering: Accelerating Discovery And Screening with PUREfrex®
Solution Provider Presentation Conference Room 2PUREfrex® is a fully reconstituted, cell free protein expression system enabling rapid, high throughput antibody engineering. It efficiently produces scFv, Fab, and IgG, and supports robust ribosome display (PUREfrexRD) for screening highly diverse libraries. Combined with AI/ML workflows, PUREfrex® accelerates discovery of next generation antibodies and cyclic peptide modalities with enhanced precision, flexibility, and scalability for biologics R&D.
Partnerships, Platforms Or Acquisitions: What Is The Winning Model For Brain Health Innovation?
Panel Discussion Conference Room 4collaboration models that actually work
• When to partner vs acquire vs build internally
• Data-sharing in a competitive landscape
• Public-private partnerships in USA: success stories & failures
Submission required
Solution Provider Presentation Conference Room 1Submission required
Integrating AI-ML And Automation In Discovery Chemistry at J&J
Industry Presentation Conference Room 2This talk will discuss the automation capabilities in Discovery Chemistry at J&J, and our efforts on integrating AI/ML and automation to accelerate small molecule drug discovery.
Novel Organoids for Modelling Virus-induced Neuroinflammation
Industry PresentationThis presentation will discuss the development of new microphysiological models of sensory-innervated lung epithelium to investigate respiratory viral neurotropism and its implications for managing and treating the most common human respiratory diseases such as asthma, COPD, and pneumonia.
AI/ML, Specializing In Omics & Systems Biology
Industry Presentation Conference Room 1Learning State Representations And Interpretable Multi-Omics Models For High-Resolution Cancer Modeling And Prediction
Industry Presentation Conference Room 1* Multi-Omic Integration: Models must synthesize diverse data types (e.g., genomics, transcriptomics) rather than relying on isolated datasets.
* High-Level Biological Focus: Instead of focusing on low-level data like individual gene mutations, models should use high-level representations (e.g., signaling pathways, mutational signatures) to improve interpretability and allow for incremental improvements.
* Elimination of "Black-Box" Algorithms: The field needs explicit unsupervised and self-supervised models that clearly map out higher-order biological structures and cellular/tumor "states."
* Transparent Predictive Modeling: The insights from these unsupervised models should serve as evidence-based inputs for simpler, highly transparent supervised models to accurately predict patient responses and improve stratification.
* Next Generation Information-Based Frameworks: To build trust among researchers and clinicians, models should base their internal metrics on Bayesian or Information Theoretic frameworks, ensuring that predictions are quantifiable and easily interpretable.
Small-Angle X-ray Scattering for Early Detection of High-Concentration Antibody Viscosity
Industry Presentation Conference Room 5HT-SAXS predicts viscosity early: Low-q structure factor changes at ≤10 mg/mL reliably flag high-viscosity mAbs long before high-concentration testing is needed
More accurate than kD: SAXS structure factor upturn/downturn directly captures attractive vs repulsive interactions and outperforms kD in viscosity classification (100% vs 82% accuracy)
High-throughput, low sample demand: Automated SAXS enables rapid, scalable developability screening using minimal material, suitable for early discovery workflows
Design And Formulation Of Long-Acting Entecavir Prodrugs For Improved Hepatitis B Treatment Adherence
Industry Presentation Conference Room 6HBV chronic infection requires lifelong daily therapy, risking non-adherence and relapse. We developed long-acting injectable (LAI) prodrugs of Entecavir (ETV) via our platform, screening >30 candidates. The lead prodrug shows sustained IM release >1 month, lower Cmax, low injection volume (<500 µL), and plasma levels above therapeutic threshold—offering a promising adherence-improving LAI approach for HBV.
Overview Of The Challenges Of CMC
Industry Presentation Conference Room 6Formulating Next-Gen Biotherapeutics: Strategies To Enhance Stability, Quality, And Scalability
Panel Discussion• Stabilising complex modalities
• Evaluating non-traditional formulation strategies — including lyophilisation, coformulation, microencapsulation, or amorphous systems
Machinability Of Spray Freeze-Dried Engineered Microparticles
Industry PresentationSpray freeze drying enables the production of highly porous engineered powders ideal for thermosensitive molecules, including biologic drugs. The process generates distinctive microstructures that strongly influence flowability, mechanical behavior, and downstream processability. This talk examines how particle architecture governs machinability, highlighting implications for pharmaceutical manufacturing.
RNAi Delivery, LPN, & Conjugate Approaches
Industry PresentationCan Oral Peptides Finally Replace Injections? From Breakthroughs To Biological Limits
Panel Discussion Conference Room 5• Is oral delivery of peptides a solved engineering problem or a biological limit?
• What level of oral bioavailability is actually “good enough” for clinical success?
• Will oral GLP-1s redefine the market or remain niche alternatives?
Breaking Out of Trial-and-Error: ML and Robotics for Predictive Drug Delivery Formulation
Industry Presentation Conference Room 6Drug delivery formulation still relies on manual, iterative experimentation. This talk shows how combining robotic formulation and characterization with ML models trained on continuous testing data creates a closed loop: simulate, formulate, validate. Real-world case studies demonstrate faster development timelines and predictable performance before entering preclinical studies.
From Formulation To Market: What Makes A Nasal Product Commercially Viable?
Panel Discussion Conference Room 6Recognize how unmet clinical needs and therapeutic advantages determine the commercial success of nasal drug products.
Evaluate the critical interplay between formulation, device design, patient usability, manufacturability, and cost in determining market adoption.
Apply regulatory, scalability, and differentiation considerations early in development to assess the commercial viability of nasal drug products.
Bio-inspired Fused-Core Ionizable Lipids Enable Efficient Endosomal Escape & mRNA Delivery
Industry Presentation• Novel fused bicyclic core-based lipids from renewable biomass outperform industry benchmarks in endosomal escape, protein expression, and antibody generation when used for delivering mRNA vaccines.
• Rational design and evaluation of 60+ lipids uncovered key design principles governing mRNA delivery efficiency and LNP thermostability
• With good manufacturability, superior thermostability, and robust protein expression, these lipids demonstrated great potential as next generation LNP components for the delivery of stable and scalable mRNA vaccines and therapeutics
Developing BBT-101 - A First-In-Class, Intranasal Formulation
Industry Presentation Conference Room 6This discussion will focus on the challenges of repurposing a drug and delivering it intranasally. Topics to be discussed will include the recent acceptance of intranasally delivered drugs, IP, the 505(b)(2) pathway, fundraising, non-dilutive funding, partnering, and more. Discussion will be based on our experience developing an intranasally delivered therapeutic for disorders of the CNS, with a focus on concussions.
Balancing Biopharmaceutics, Stability & Manufacturability In LAI Suspension Formulation Design
Industry Presentation Conference Room 6First Line Of Defense: Nasal Delivery Of Low-Cost Biologics For Pan-Viral Defense
Industry PresentationDelivering spirulina-based biologics for respiratory viral defense demands tight integration across production, processing, and formulation. This talk covers how Lumen's cGMP-scale spirulina cultivation, downstream extraction, intranasal formulation and device selection work together as a single pipeline—transforming a low-cost, photosynthetically-grown protein source into a stable nasal therapeutic capable of protecting against a broad spectrum of upper respiratory viruses.
From Oral to Inhaled: Lifecycle Delivery Strategies for Pulmonary Disease Therapeutics
Roundtable Discussion Conference Room 6When and why to consider alternative delivery routes as lifecycle extensions for oral drugs
Practical formulation, device, and regulatory considerations when repurposing a candidate for inhalation
Lessons learned and open questions across industry and academia
Unlocking AI/ML In Drug Delivery — Overcoming Data Quality & Access Barriers
Panel Discussion Conference Room 5• What types of data are most valuable for formulation and delivery research?
• Current barriers: access, standardization, quality, and reproducibility
• Strategies for data sharing across pharma, biotech, and academia
• Case examples of successful AI/ML applications in drug delivery
• Building collaborations to create usable, high-quality datasets
Small Molecules To Mitigate Cisplatin Toxicities Including Hearing Loss
Industry Presentation Conference Room 6Cisplatin toxicities affect 1million cancer patients globally each year
Repurposing small molecule drugs to mitigate these toxicities is a powerful strategy
We have identified >100 candidates among which the top candidate Ting4 is effective and safe in preclinical models
We have reformulated Ting4 and will start clinical phase Ib trials in cancer patients soon.
Overcoming Formulation Challenges For Inhaled Biologics
Industry Presentation Conference Room 6The Next Frontier In RNA Delivery: Antibody-Oligonucleotide Conjugates, Clinical Validation, CMC Challenges And Future Directions
Industry Presentation Conference Room 4• AOCs are emerging as the next generation of RNA therapeutics, combining antibody targeting with oligonucleotide payloads to enable delivery beyond the liver. Clinical proof-of-concept is growing, with industry momentum.
• CMC remains a key hurdle. AOCs combine the challenges of biologics and oligonucleotides, requiring control of stability, conjugation, heterogeneity, manufacturability, and analytical complexity.
• Future success depends on solving delivery and CMC challenges, particularly extrahepatic targeting, scalable manufacturing, and next-generation RNA payloads.
Advancing Oral Peptide Delivery Through Integrated Peptide Engineering And Formulation Design
Industry Presentation Conference Room 5Oral delivery of peptides remains a significant challenge. Despite extensive research, formulation-based approaches alone have made only incremental progress in overcoming absorption barriers. Emerging evidence suggests that a more integrated strategy, combining formulation development with rational peptide engineering, may provide a more effective path toward improving oral absorption and achieving clinically meaningful bioavailability.
Keeping It Together: Understanding And Mitigating Liquid-Liquid Phase Separation In Biologics Formulations
Industry Presentation Conference Room 6o Liquid-liquid phase separation (LLPS) is a critical development challenge in high-concentration protein formulations, driven by weak, reversible protein-protein interactions that can manifest as opalescence, viscosity changes, or visible phase separation.
o Strategies to identify and characterize LLPS will be covered. Formulation composition, the primary means to control LLPS, can serve as both a diagnostic and mechanistic probe: strategic modulation of excipients can isolate the dominant interaction forces driving LLPS, allowing mechanism-specific characterization and targeted intervention.
CMC Considerations for Combination Products And Emerging Solutions
Industry Presentation Conference Room 6• Strategic FDC value: move beyond convenience by proving therapeutic contribution, patient benefit, and lifecycle quality.
• Innovation-led differentiation: use multilayer tablets, microencapsulation, hot-melt extrusion, and 3D printing to enable complex combinations while reducing incompatibility risk.
Breaking The Bottleneck For Biologics & Small Molecules Drug Delivery
Panel Discussion Conference Room 4• What makes oral delivery so attractive—and so difficult—for biologics/oligos?
• Novel approaches: nanoparticle carriers, permeation enhancers, and formulation tricks
• Case studies of progress (in oral biologics
• Balancing patient convenience vs. technical feasibility
• How AI/ML can help accelerate innovation
ABI-171: Exploring Inhaled Delivery As A Lifecycle Strategy For An Oral Anti-Fibrotic Drug In IPF
Industry Presentation• Overview of ABI-171 as a novel oral anti-fibrotic therapeutic for IPF with orphan designation & preclinical proof of concept
• Rationale for exploring inhaled/intranasal delivery as a lifecycle management strategy: direct pulmonary targeting, potential for reduced systemic exposure, and expanded therapeutic utility
• Key formulation and delivery challenges in transitioning an oral candidate to an inhaled route and early learnings
Oral Inhalation Through Nebulizers
Industry PresentationNext-Gen DNA Medicine And Delivery: The Potential For In Vivo Therapeutic Protein Production In Rare Disease
Industry Presentation Conference Room 5• Promising clinical proof-of-concept of long-term in vivo protein expression via non-viral local gene delivery to the skeletal muscle
• Absence of host immune response against the vector or encoded proteins enabled drug redosing
• Data support potential in the development of durable protein replacement therapies in rare genetic disease, including Fabry, Hypophosphatasia (HPP) and Hemophilia A diseases
Translational Nasal Drug Delivery Platforms for Targeted CNS Therapeutics
Industry PresentationDemystifying Fogging In Lyophilized Products
Industry Presentation Conference Room 5The talk would cover recent work aimed at establishing a mechanistic understanding of factors affecting fogging in freeze-dried products. While fogging is a routinely encountered cosmetic defect in freeze-dried formulations, it can potentially impact container closure integrity. Hence, vials with fogging above the shoulder and neck are often rejected during visual inspection. This, in-turn, can result in high batch reject rates during clinical and commercial manufacturing. The severity of fogging in lyophilized products is impacted by a varied range of factors. These can be broadly classified as: formulation composition, pharmaceutical processing conditions and primary packaging. Hence, a systematic study to investigate the influence of each of these factors on fogging enables a mechanistic understanding and consequently, the design of suitable mitigation strategies.
Utilizing Nanotechnology Approaches
Industry Presentation Conference Room 4Formulation Strategies to Overcome Peptide Gelation During Lyophilization
Industry Presentation Conference Room 6Peptide gelation complicates lyophilization scale-up by affecting primary drying rates and heat transfer. This case study examines gelation behavior of an investigational peptide and its impact during freeze-drying, showing how understanding this interplay enables robust, transferable lyophilization cycles. The findings offer practical solutions for advancing peptide therapeutics from lab to market.
AI & In Silico Tools In Formulation Development
Roundtable Discussion Conference Room 4Durable DNA-Based Leptin Gene Therapy for Treatment of Lipodystrophy
Industry Presentation Conference Room 4Congenital generalized lipodystrophy is driven by severe leptin deficiency, with daily metreleptin injections the only approved therapy. Using the non-viral Fusogenix Proteolipid Vehicle, a single intravenous or intrathecal injection of DNA-encoded leptin restored leptin for over six months in ob/ob and CLA-fed mouse models, normalizing weight, food intake, and metabolic function.
Design Considerations For IV In-Use Studies To Support Clinical Administration
Industry Presentation Conference Room 6A thoughtful design of in-use studies ensures drug stability and compatibility during dose preparation and clinical administration. An overview of study design considerations such as the regions and number of clinical sites and anticipated dose preparation and administration procedure is presented. A case study walks through an example of in-use studies to support an early phase program with clinical sites in multiple regions, weight-based dosing, and a switch from undiluted to diluted administration.
Novel Tumour Targeting Peptide Therapies
Industry Presentation Conference Room 5Oncoloytic peptides as next generation cancer therapies
Abscopal vaccination - amplifying local cell celling through systemic immune engagement
Insights Into Successful Industry Clinical Collaborations
Industry Presentation Conference Room 5• The session will analyze the landscape of clinical collaborations, focusing on how these external partnerships act as a key driver for innovation in combination therapies.
• You will explore the "give-and-receive" drug model, detailing how to bridge internal assets with external innovation to effectively mitigate risk and improve evidence generation.
• Discussions will cover the challenges and successes of managing these partnerships to accelerate development and redefine approaches to complex treatment pathways.
Formulation & Delivery Challenges For Oral Peptides
Industry Presentation Conference Room 5Reimagining Abuse Deterrence Through Controlled-Release Engineering
Industry PresentationThe talk highlights innovation-oriented evaluation strategies, including stress-based dissolution testing, extraction resistance profiling, and kinetic descriptors such as partial area-under-the-curve (pAUC) to characterize early exposure behavior. These tools provide mechanistic insight into how formulation design choices translate into exposure profiles relevant to abuse liability assessment.
Inhaled Delivery Of Biologics
Industry Presentation Conference Room 5High Dose, High Volume Biologics
Industry PresentationHigh concentration protein therapeutics have become more popular as they favor subcutaneous (SC) administration. Successful development of high dose biologics requires adopting certain formulation approaches to overcome technical challenges such as viscosity, solubility, stability, process issues and delivery limitations. There is no one approach fits for all. This talk will outline some key aspects while designing high concentration protein therapeutics from the formulation and process standpoint.
Title TBC
Industry PresentationProtein Stability: A Drug Delivery Formulation Challenge
Industry Presentation Conference Room 5Heat and organic solvents are commonly used in preparing sustained delivery formulations for small molecule drug delivery. The applications of these formulations are not extended to sustained release formulations of proteins because of the protein stability concern. We have performed case study on surrogate protein to study impact of the high temperature stress and organic solvent to understand the stability of the protein. The results of these case study will be shared and guidance for developing sustained release formulations for proteins will be provided.
mRNA Importance
Industry PresentationProgramming Skeletal Muscle As A Bioreactor: A Platform For Durable In Vivo Delivery Of Therapeutic Proteins
Industry Presentation Conference Room 5• Application of MYO Technology™ and DNA electroporation to support sustained GLP-1 expression
• Broader platform potential for delivery of antibodies and other therapeutic proteins, with reduced dosing burden, manufacturing complexity, and cost
• DNA electroporation as a flexible, non-viral delivery approach offering scalability, durable expression, and the potential for repeat dosing
Arg.Glu* (Arginine-Glutamate) As Ideal Excipient For High Concentration Monoclonal Antibody And Complex Biologics Formulations
Solution Provider Presentation Conference Room 5High 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 literature and data demonstrating Arg·Glu’s effectiveness in solving some of the challenges associated with high concentration and complex biologics formulations
Sponsor Presentation
Solution Provider Presentation Conference Room 5Submission required
AI-Powered mPredict™ Co-Crystal Prediction Tool: Recent Advances & Experimental Validation
Solution Provider Presentation Conference Room 5Up to 90% of APIs in development suffer from poor solubility, making co-crystallization a key solid-formulation strategy. We present an AI-based co-crystal prediction tool combining quantum chemical calculations and machine learning to rank co-former candidates for a given API.
Solution Provider Presentation
Solution Provider Presentation Conference Room 6Preformulation And Solid Form Technologies Utilizing MicroED As A Game Changer For API Characterization
Solution Provider Presentation Conference Room 5Preformulation study is a key for the successful IND of drug candidates. API solid form is one of its critical elements for which 3DED/MicroED analysis is often required because only micrograms of powders and submicron crystals are available.
Solution Provider Presentation
Solution Provider Presentation Conference Room 6Critical Attributes For Successful mRNA Based Drug Products
Roundtable Discussion Conference Room 4• What are critical elements for drug substance?
• What makes a good delivery system?
• How sustainable can mRNA-based drug products be?
Solution Provider Presentation
Solution Provider Presentation Conference Room 6Making Smart CMC Decisions In Early Drug Development: Strategies To Accelerate IND Readiness
Industry Presentation Conference Room 6Early CMC decisions on dosage form selection and what to lock versus defer have a lasting impact on development timelines and Phase 3 success. Establishing a robust design space before Phase 3 enables future manufacturing flexibility. With new FDA/EMA AI principles, AI-assisted development must meet rigorous traceability, validation, and risk-based expectations.
The Next Frontier In Inhalation Therapy: How Capsule Engineering Directs Fine Particle Delivery And Aerosolization Performance
Solution Provider Presentation Conference Room 6Pulmonary delivery is expanding from respiratory to systemic therapies. This presentation explores how capsule, API, excipient and device interactions influence DPI aerosolization and support robust next-generation inhalation development.
Delivery-Driven Innovation: Unlocking The Potential Of Next-Generation Therapeutics
Keynote Conference Room 5• When delivery—not the molecule—is the bottleneck
• Overcoming barriers across peptides, CNS therapies & infectious diseases, and emerging modalities
• From AI to clinical use
Start Up Zone Presentations x2
Industry Presentation Conference Room 4Why Formulations Fail — Lessons Learned Across Modalities
Panel Discussion Conference Room 4• Common reasons for failure (e.g., stability, delivery barriers, patient compliance)
• Case studies: when a formulation didn’t meet expectations
• The role of biological barriers (e.g., blood-brain barrier, tissue penetration) in failure
• How early-stage decisions can set projects up for success or failure
• Creating a culture that shares failures constructively
Start-Up Presentation - QT Sense
Industry Presentation Conference Room 4Start of Track: Nasal & Inhalation Drug Formulation & Delivery
Industry Presentation Conference Room 6Start of Track: Drug Product Development, CMC & Developability
Industry Presentation Conference Room 6BIONDD® Oral Capsule Technology – Delivery Of Biomolecules Achieving Consistent Similar PK Profiles To Subcutaneous Injection In Dogs
Solution Provider Presentation Conference Room 5Oral delivery of biologics remains challenging due to their degradation in the GI tract and poor absorption, thereby being commonly administered by subcutaneous injections. The BIONDD® technology, with a convenient small-size 00 oral capsule, overcomes these challenges by administering the active substance directly into the stomach wall. Pre-clinical in vivo studies with BIONDD® have shown successful oral delivery of various biomolecules in dogs with a bioavailability comparable to SC injection.
Start-Up Presentation - Helical AI
Industry PresentationStart-Up Presentation - Synfini
Industry PresentationHow To Address Biomarker Needs For Novel Targets
Industry PresentationCNS Drug Discovery Case Study
Industry Presentation Conference Room 3Development Of Small Molecule Huntington’s Disease Therapeutics With A Novel Mechanism Of Action
Industry Presentation Conference Room 3• Hit identification in a phenotypic screen
• Demonstration of selective degradation of mutant huntingtin and cellular disease phenotype reversal
• PK/PD relationship in preclinical mouse models
Development Of Novel Therapeutic Drugs For Neurodegenerative Diseases
Industry Presentation Conference Room 3Encapsulated Cell Therapy: Safe, Durable, And Broadly Applicable Ophthalmic Drug Delivery
Industry Presentation Conference Room 3Encapsulated Cell Therapy (ECT) is a novel drug delivery mechanism, made up of device and biologic components into a single implantable product. This implant has a robust safety profile, is highly durable, and may be applicable to a broad range of disease states.
Reinventing CNS Drug Discovery With AI And Human Brain Organoids
Industry Presentation Conference Room 3CNS drug development faces a fundamental productivity problem: more than 90% of neuroscience drug candidates fail in clinical development, with failure rates for disease-modifying therapies in major neurodegenerative disorders historically approaching 100%. A major contributor is the limited ability of conventional preclinical models to predict therapeutic efficacy in patients. This talk will present a human-first discovery platform that combines patient-derived brain organoids with biomarker-based screening endpoints and AI disease gene network analysis to generate human-relevant evidence of therapeutic efficacy before dosing patients in the clinic. Using programs in Parkinson’s disease and rare genetic neurological disorders, I will show how this approach can identify disease-modifying mechanisms and targets, discover and prioritize therapeutic candidates, and build differentiated drug pipelines directly from human disease biology. By shifting critical efficacy decisions to human disease models before clinical testing, this strategy has the potential to reduce clinical attrition, improve capital efficiency, accelerate candidate selection, and increase the probability of clinical success in neuroscience.
Understanding The Role Of AI & Data In Neuroscience Research
Industry PresentationGenomics Technologies For Neurological Indications
Industry Presentation Conference Room 3Transformative Gene Therapies For Treating Rare Diseases Using A Novel Plant-Based Manufacturing
Industry PresentationInnovating Clinical Trial Design
Industry Presentation Conference Room 3Advancing clinical trial innovation from planning to outcomes.
Utilizing digital endpoints in clinical trials to shorten timelines, reduce costs, and enhance statistical power.
Employing artificial intelligence (AI) and machine learning (ML) to facilitate early detection of efficacy.
Utilizing AI/ML for the synthesis of clinical data to foster biological and mechanistic comprehension.
Regulatory Acceptance Of Efficacy And Safety Data For Neurological Diseases Utilizing Human-On-A-Chip Systems
Industry Presentation Conference Room 3Multiple drugs have been repurposed into INDs for neurological diseases using only efficacy data from these models. Data will be prepared for ALS, Alzheimer's, Myasthenia Gravis, Charcot-Marie-Tooth disease as well as others. Digital twins have been used to extrapolate in vitro data to in vivo outcomes.
Bridging The Translational Gap In Neurodegenerative Drug Development
Roundtable Discussion Conference Room 4• Building more predictive preclinical programs using human-relevant models and biomarkers
• Leveraging AI to improve target prioritization, patient stratification, and clinical trial design
• Overcoming the biggest barriers to translating promising discoveries into clinical success
• What recent neuroscience programs have taught us about reducing late-stage attrition
Single-Cell And Spatial Omics Approaches For Functional Genomics
Industry Presentation Conference Room 3While pooled CRISPR screens nominate causal genes, single-cell and spatial omics reveal the cell states and tissue niches through which they act. We present a modular platform integrating CRISPR-KO, CRISPRi and CRISPRa screens with guide capture, multiomic Perturb-seq, and spatial CRISPR guide detection in tissue and in cell culture.
Precision Nanotherapeutic Targeting of Pathological NMDA Receptor Signaling in Alzheimer’s Disease
Industry Presentation Conference Room 3Alzheimer’s disease drug development has been limited by the difficulty of suppressing pathological neuronal signaling without disrupting normal synaptic function. We developed a nanoscale therapeutic strategy that uses steric selectivity to preferentially target extrasynaptic NMDA receptor signaling while preserving synaptic activity. In cellular and 5xFAD mouse models, this approach produced long-lasting functional and pathological benefits, including improvements in cognition, neuronal health, neuroinflammation, and amyloid-associated pathology. This presentation will discuss the scientific rationale, preclinical validation, translational challenges, and development path for this precision nanotherapeutic platform as a potential disease-modifying approach for Alzheimer’s disease
AI Rewiring The Future Of Drug Discovery
Keynote Conference Room 1• Taking a deep dive into generative AI for molecular design to illustrate individual methods that have transformative implications
• Putting it all together – active learning and multi-property optimisation for multiple drug properties
• Using AI to integrate early and late-stage considerations – agentic AI approaches
• How do we collaborate across the industry to develop these frameworks?
Afternoon Break & Refreshments
Lunch Break
Morning Break & Refreshments
Close of Day One & Networking Drinks Reception
Lunch Break
End of Event
Morning Break & Refreshments
How Early Should Developability And Drug Delivery Considerations Influence Target Selection And Lead Optimization Decisions?
Roundtable Discussion Conference Room 4• What risks emerge when delivery and developability are addressed too late in the discovery process?
• Should delivery feasibility be a formal criterion when prioritizing targets?
• How can discovery and development teams collaborate earlier to improve program success rates?
From Discovery to Delivery: Turning Scientific Innovation into Commercial Success
Roundtable Discussion Conference Room 4• What separates promising science from commercially viable innovation?
• Common pitfalls in translating research into therapeutics
• Building collaborations between academia, biotech, and pharma
• How startups can position themselves for investment and strategic partnerships
Target Discovery, Validation, And Therapeutic Innovation In Autoimmune Diseases
Industry Presentation• Target identification using in silico and multi-omic approaches
• CRISPR-based high-throughput functional validation in human primary immune cells
• Development of innovative therapeutic modalities for the nominated target
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