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Discovery & Development US 2027
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Hilton San Diego Bayfront, CA
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
How To Address Biomarker Needs For Novel Targets
Industry Presentation Conference Room 3- Identifying biomarker needs to support the development of novel therapeutic targets
- Approaches and challenges in biomarker discovery, validation and translation
CNS 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 3- Approaches to discovering and developing novel therapeutics for neurodegenerative diseases
- Key challenges in translating promising drug candidates into clinical development
Delivering 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
Applications Of High-Throughput Data Collection In AI/ML
Industry Presentation Conference Room 1Quantitative 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.
Encapsulated 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 Presentation Conference Room 3- Exploring plant-based manufacturing approaches for gene therapies targeting rare diseases
- Opportunities and challenges in translating novel manufacturing platforms into therapeutic development
Novel Models For ADME-Tox Research
Workshop Conference Room 3- Exploring novel models for assessing absorption, distribution, metabolism, excretion and toxicity
- Opportunities and challenges in improving the predictive value of ADME-Tox studies
Where Smart Capital Meets Science: An Entrepreneurial Perspective On The Next Wave Of Drug Discovery Innovation
Fireside Chat Conference Room 4• How can a founder navigate funding and launching drug discovery technologies?
• What drives funding decisions in a shifting biotech market and where capital is flowing next?
• How can entrepreneurs remain informed on, and advance the impact of AI on society and in healthcare, in a rapidly growing market?
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
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
Innovating 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.
Deeper 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.
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
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 3• 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
Novel Organoids For Modelling Virus-induced Neuroinflammation
Industry Presentation Conference Room 3This 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.
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
Catching Oxidative Stress Early: Smarter Biomarkers And Toxicity Screening
Solution Provider Presentation Conference Room 3This talk shows how QN technology measures reactive oxygen species (ROS) to tackle two major drug discovery bottlenecks: finding biomarkers that predict which patients will respond to treatment, and spotting toxicity risks before costly development.
Two case studies cover ROS as an early neuroscience biomarker and preclinical toxicity screening, with data showing advantages over current tools. Attendees will see how earlier, quantitative oxidative stress readouts enable faster go/no-go decisions and lower pipeline attrition and cost.
RNA-Driven Neurotherapeutics By Small Molecules And PROTEIMERS
Industry Presentation Conference Room 3We reported that the broad-acting Alzheimer’s disease (AD) clinical drug Posiphen (PS) inhibits translation of the amyloid precursor protein (APP) as well as the mRNA encoding alpha-synuclein (asyn), the major culprit protein of Parkinson’s disease (PD) and the Huntingtin protein. However, our new pipeline of translation blockers seeks to prevent neurodegeneration. These are potent drugs that interfere at higher selectivity with these 5’untranslated regions (5’UTRs) of the neurodegenerative disease-associated transcripts, i.e. the APP/ amyloid of AD and PD specific asyn. These new therapeutic agents operate without off-target inhibition of other markers of neurodegenerative diseases and were shown to limit both environmental and viral-associated neurodegeneration. The amyloid precursors APP and asyn were recently shown to be active in cellular iron transport. The translation of their mRNAs in neural cells are each controlled by a modified Iron-responsive Elements (IRE) RNA stem-loops. Notably, our new 5’UTR inhibitors operated to limit APP-amyloid and asyn in the nanomolar range without off-target perturbations of iron homeostasis. These were screened/selected from ~200,000-component compound molecular libraries and were validated to treat the pathologies of neurodegeneration using neuroblastoma cell models and iPSC derived dopaminergic (DA) and cholinergic neurons. These translation inhibitors should limit brain amyloid and the formation of toxic fibrils of asyn in the brains of well-known mouse models of AD and PD. We can present data on their pharmacological and toxicology properties. Recently, we discovered a line of therapeutic benzimidazoles that inhibit APP translation linked to providing RNA-directed inhibitory actions to limit COVID by lowering the translation rates of the replicase in SARS-CoV-2.
Dealing With High Manufacturing And Product Costs In The Gene Therapy Space
Roundtable Discussion Conference Room 4• Scalability challenges in manufacturing
• Supply chain and raw material constraints
• Process optimization and automation opportunities
• Regulatory and quality compliance costs
• Innovative business and partnership models
From Oral to Inhaled: Lifecycle Delivery Strategies For Pulmonary Disease Therapeutics
Roundtable Discussion Conference Room 4When 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
Small Molecules To Mitigate Cisplatin Toxicities Including Hearing Loss
Industry Presentation Conference Room 3• Cisplatin toxicities affect 1 million 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
AI & In Silico Tools In Formulation Development
Roundtable Discussion Conference Room 4• Formulation challenges of novel modalities (e.g., ADCs and bispecific antibodies) relative to conventional mAbs
• Analytical and biophysical tools for formulation development of novel modalities
• Opportunities and challenges of AI/ML in formulation development for next-generation biologics
Workshop: Integrating NAMs Into Translational Drug Development - Presentation 1: Validation & Regulations For NAMs
Workshop Conference Room 3• Approaches to validating New Approach Methodologies for drug discovery and development.
• Regulatory considerations and challenges in gaining acceptance for NAMs
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.
Workshop: Integrating NAMs Into Translational Drug Development - Panel Discussion: How To Use NAMs
Panel Discussion Conference Room 3• 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
Critical 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?
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
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 1: Own Your Biological Intelligence: How To Use Biological Foundation Models And Your Proprietary Data To Find Better Targets And Biomarkers
Solution Provider Presentation Conference Room 4Pharma’s proprietary data and scientific expertise should become intelligence it owns. This talk introduces how Helical adapts biological foundation models to a company’s own biology and puts them to work in target identification and biomarker discovery.
We’ll explore what it takes to make these models useful in research: evaluations that reflect the scientific question, complex inference workflows, and continuous improvement from experimental results. We’ll also explain why ownership of data, evaluations and model weights matters, and how these capabilities fit into the tools and AI agents scientists already use.
Start Up Presentation 2: Synfini – The Agile Chemistry Foundry
Solution Provider Presentation Conference Room 4Synfini’s Cloud accessible Agile Chemistry Foundry integrates open AI dry labs with AI-driven robotic chemistry to accelerate the discovery of optimal small molecule drugs. Automation of complex medicinal chemistry has demonstrated success against the majority of molecular targets.
The 90% of Biology Nobody's Measuring
Solution Provider Presentation Conference Room 4Genomics reshaped how we study disease. But between genotype and phenotype, most of biology remains invisible. The small-molecule chemistry that drives disease progression, drug response, and toxicity sits in a layer that conventional tools largely miss. This talk introduces biochemical omics: the ability to quantify metabolites, lipids, and peptides from existing instruments at a fraction of historical cost. Drawing on case studies in oncology disease characterization, novel target discovery, and clinical trial stratification, Jim Flanigon shows what changes when researchers can finally see the other 90%.
Predicting Drug Response: Explainable AI For Precision Clinical Development
Solution Provider Presentation Conference Room 4Zephyr’s LDT-agnostic AI models predict drug response with biological explainability, enabling precise patient stratification to de-risk and accelerate therapeutic development
Brilliant AI. Disconnected Data. Now What?
Solution Provider Presentation Conference Room 4What happens when powerful AI meets lab data that cannot move beyond the instrument? This session challenges the model-first mindset and puts a less glamorous but critical bottleneck in the spotlight: common architecture. Join Intel’s Beenish Zia to explore how connected lab workflows, shared data context and AI closer to the instruments could help biopharma move beyond isolated pilots. Discover the architecture questions worth asking today to prepare for tomorrow’s automated labs, wet-lab robotics and physical AI.
Small Pellets, Big Possibilities: Unlocking the Power of MUPS
Solution Provider Presentation Conference Room 4MUPS (Multiple Unit Pellet System) technology offers significant formulation flexibility, enabling immediate-release, delayed-release, extended-release, or combination release profiles through adjustment of pellet coating characteristics. MUPS is particularly valuable for products requiring robust release performance, improved patient compliance, and lifecycle management opportunities.
Key Advantages:
• Reduced dose dumping risk
• More uniform GI distribution
• Flexible release profile design
• Suitable for IR, DR, and ER products
• Enhanced formulation robustness
• Patient-friendly dosage form
Forma Life Sciences is a leader in MUPS technology and utilization of this technology in formulation development of modified release formulations and achieving specific target product profiles.