Explore the Agenda

8:00 am Morning Coffee and Check-in

8:55 am Chair’s Opening Remarks

Ensuring Safe, Compliant & Measurable Nose-to-Brain Delivery for CNS Therapeutics

9:00 am Regulatory Perspectives on Nose-to-Brain Delivery Pathways to Accelerate Safe and Compliant CNS Drug Development

Lead Scientist, Oragenics Health
  • Clarify regulatory expectations for intranasal drugs targeting the CNS
  • Explore how FDA and EMA evaluate device/formulation combination products for safety and efficacy
  • Provide guidance for designing preclinical and clinical packages to support regulatory approval

9:30 am Session Reserved for Medpharm

10:00 am Reimagining Rare Disease Treatment Through Intranasal Delivery to Transform Patient Experience

Chief Science Officer, KIF1A.ORG
  • Human Impact of Rare Disease: Framing unmet needs through the lived experience of patients and families
  • Why Nasal Delivery Changes the Paradigm: Lessons from neurology showing how intranasal routes can replace invasive administration
  • ASOs and the Nose-to-Brain Opportunity: Exploring why intranasal delivery may unlock safer, more practical CNS access

10:30 am Morning Break

Translating Preclinical Nasal Models to Predict Human Clinical Outcomes for Safer and More Effective Therapeutics

11:30 am Predictive Animal Models for Nasal Drug Absorption and Distribution for CNS Applications

Engineering Foundation Professor, Virginia Commonwealth University
  • Examine the value and limitations of current models for nasal drug delivery
  • Explore anatomical and physiological differences that impact translation from animal studies to humans
  • Understand strategies to increase predictive power, through novel technology including nasal casts

12:00 pm Session Reserved for Intertek

12:10 pm Leveraging Organoids and Innovative Models to Predict Clinical Outcomes

Senior Scientist, Translational Chemistry, AltPep Corporation
  • Explore the use of organoid systems to study local toxicity, deposition, and absorption patterns
  • Examine how these models informed dosing, formulation, and delivery strategies for clinical trials
  • Highlight lessons learned and best practices for integrating preclinical models into development programs

12:40 pm Experimental Models to Quantify, Predict & De-Risk Brain Delivery

Assistant Professor, Harvard Medical School
  • Reviewing current experimental evidence and biological mechanisms governing nose-to-brain transport and brain parenchyma exposure
  • Benchmarking in vitro brain tissue models against animal and emerging human in vivo PK/PD data to strengthen preclinical translation
  • Exploring how advanced brain models and experimental datasets can inform predictive tools, formulation strategy, and future clinical study design

1:10 pm Lunch Break

Transforming Therapeutics Through Intranasal Delivery of Advanced Modalities: Overcoming Formulation Barriers to Maximise Efficacy and Patient Impact

2:00 pm Optimizing Carriers for Nasal Nucleic Acid Delivery and Lipid Nanoparticles to Improve Delivery Efficiency and Maximize Therapeutic Effect

Director - Formulation & Delivery Sciences, Ionis Pharmaceuticals, Inc.
  • Deep dive into lipid nanoparticle (LNP) technologies for RNA and oligonucleotide delivery
  • Exploring novel mechanisms of action and key design considerations influencing delivery performance
  • Landscape overview of current and emerging approaches in intranasal nucleic acid delivery

2:30 pm Exploring Powder-Based Intranasal Delivery as a Platform for Systemic Therapeutics

Chief Executive Officer, Nasus Pharma
  • Compare powder versus liquid intranasal delivery, highlighting how deeper nasal deposition, increased surface area, and richer vascular access can drive faster and higher systemic absorption
  • Explore the critical challenges of particle size distribution, particle shape, and limited large-scale manufacturing capacity, and how proprietary formulation strategies can address them
  • Advancing powder-based naloxone and epinephrine programs, including device considerations, scalability planning, and where innovation is still needed across delivery technologies and manufacturing infrastructure

3:00 pm Intranasal Delivery of a Novel Synthetic Small-Molecule Antibiotic Using Nanocarriers

President & CEO, Logix Biosciences
  • Trace the journey from honey-derived antimicrobial activity to a scalable, water-soluble synthetic small molecule designed for intranasal delivery
  • Explore how intranasal administration enables early intervention against viral infections that trigger secondary bacterial disease, with implications for resistance management
  • Learn how nanocarrier delivery supports sustained local release, minimizes systemic exposure, and enables safe degradation into non-cytotoxic components after therapeutic action

3:30 pm Chair’s Closing Remarks

3:35 pm End of Conference Day Two