8:00 am Check-In, Coffee & Light Breakfast
8:45 am Chair’s Opening Remarks
INNOVATIONS IN CNS DRUG DELIVERY: ENGINEERING, TARGETING, & FORMULATION STRATEGIES TO ENHANCE EXPOSURE TO THE BRAIN
9:00 am Advancing a Brain-Targeted AAV Gene Therapy for Alzheimer’s Disease
Synopsis
- Evaluating functional RNA silencing in the brain
- Advancing brain-targeted viral vector design
- Exploring the path from preclinical success to clinical impact
9:30 am Formulation Strategies for Long-Acting Injectable Prodrugs in Schizophrenia: Reducing Dosing Burden
Synopsis
- Designing prodrugs for extended CNS exposure: How modulation of physicochemical properties supports monthly or longer dosing for conditions like schizophrenia
- Overcoming high-load suspension issues, selecting excipients, and optimizing vehicles for improved injectability and stability
- Trends toward patient-centric administration (e.g., subcutaneous), potential for at-home use, and innovations in drug-device combinations
- Approaches to modulating physicochemical properties (e.g., solubility, lipophilicity) and overcoming challenges in formulating high-concentration suspensions for extended release
- Lessons learned from preclinical candidate selection, vehicle optimization, and preparing for clinic – highlighting the formulation’s path from bench to bedside
10:00 am Speed Networking
Synopsis
A prime chance to make the most of in-person networking and forge new connections with an expanding community of experts adopting innovative technologies to target therapeutic candidates to the CNS. Designed to maximize your introduction to numerous new individuals and serve as a catalyst for ongoing discussions during the summit, connect with peers across translation, medicinal chemistry, device development, protein engineering, DMPK, biology, and more.
10:30 am Morning Break & Refreshments
Track 1: Systemic Delivery
Nasal Pathways to the Brain: Precision Delivery for Maximum Patient Benefit
11:00 am Nose to Neuron Delivery of Creatine: Unlocking A Novel Platform for Brain Delivery
Synopsis
- Explore evidence of improved memory performance and brain energy modulation
- Discover innovations in formulation – development of a creatine prodrug capable of passive olfactory nerve entry
- Designing a phase 2-ready package targeting neurodegenerative diseases and creatine transporter deficiency
11:30 am Session Reserved for Aptar Pharma
12:00 pm Pioneering Non-Invasive CNS Drug Delivery: The Promise & Challenges of Nasal Routes to Enable Direct CNS Targeting While Minimizing Systemic Toxicities
Synopsis
- Share insights on leveraging natural anatomical pathways for CNS access
- Address gaps in technology and knowledge that early-stage developers need to overcome
- Showcase how nasal delivery can complement or disrupt traditional systemic and invasive CNS delivery methods
Track 2: Direct & Local Delivery
Enhancing CNS Impact through Optimized Direct Delivery Strategies & Careful Formulation
11:00 am Optimizing Technical Workflow to Safely & Effectively Deliver Gene Therapies via ICM Administration
Synopsis
- Leveraging intra-cisterna magna (ICM) delivery to maximize vector distribution throughout the CNS
- Key technical and clinical considerations for precise and reproducible administration to the cisterna magna
- Evaluating neuroimaging strategies to guide and confirm targeted delivery
- Exploring scalability, safety, and dose optimization for broader clinical application in genetic CNS disorders
11:30 am Session Reserved for Realeve
11:40 am Roundtable Discussion: Not All Modalities Are Created Equal: Overcoming Formulation Challenges in Intrathecal Delivery to Speed CMC & Improve Patient Outcomes
Synopsis
- How do different modalities uniquely challenge formulation for safe and effective intrathecal delivery?
- What formulation strategies – buffers, excipients, conjugates – are proving most effective or problematic for IT delivery of ASOs and siRNAs?
- What are regulators looking for in safe and scalable IT formulations, and how can we align preclinical testing with clinical expectations?
- What are the risks of bolus dosing on CSF composition, and how can formulation mitigate neurotoxicity across species?
12:30 pm Lunch & Networking
Track 1: Systemic Delivery
Showcasing Innovations in CNS Delivery Vehicles Using Viral, Non-Viral, & Chemistry- Driven Strategies to Overcome Biological Barriers
1:30 pm Novel AAV Capsids Enable Modular Tropism for Brain, Liver & Muscle with Minimized Hepatotoxicity
Synopsis
- Engineering capsids with surface-displayed mini antibodies to facilitate receptor-mediated transcytosis across the blood-brain barrier
- Introducing targeted mutations in capsid loop regions to abrogate liver receptor binding, achieving a reduction in hepatic tropism
- Incorporating muscle-specific targeting peptides to create a modular vector platform with tuneable multi-organ tropism profiles
2:00 pm Leveraging AI-Powered Natural Nanoparticles: A Novel Dual-Mechanism Shuttle for Enhanced BBB Penetration & Parenchymal Delivery
Synopsis
- Introducing the dtBBB-NNP platform, engineered using a proprietary AI-driven engine (NNP Design AI™) to ensure predictable and robust Blood-Brain Barrier penetrating capabilities for next-generation CNS therapeutics
- Elucidating the novel dual-mechanism of action that facilitates highly efficient transcytosis across the BBB, enabling targeted delivery of a wide range of therapeutic payloads including oligonucleotides (ASO, siRNA) and enzymes
- Presenting compelling preclinical in vivo data demonstrating significant payload accumulation in the brain parenchyma and specific uptake by key CNS cell types, and discussing the platform’s cGMP-readiness for clinical translation
2:30 pm Redefining BBB Penetration: Engineering Small Molecules for Active CNS Delivery via Uptake Transporters
Synopsis
- Harnessing endogenous uptake transporters for targeted brain delivery
- Designing integrated, linker-free small molecules for improved specificity and stability
- Using animal studies, tissue distribution analysis, and transporter expression atlases to predict and minimize off-target effects in non-CNS tissues such as the heart and liver
Track 2: Direct & Local Delivery
Unlocking the Power of Direct CNS Delivery Using Smarter Devices, Safer Routes & Scalable Solutions
1:30 pm Approaches for Intra-CSF Delivery: Enabling Precision & Access
Synopsis
- Showcase the evolution of device-enabled CNS drug delivery, including programmable infusion systems
- Discuss applications to genetic medicine
- Address translational considerations to support longitudinal use in chronic dosing scenarios
2:00 pm Round-table Discussion: Straight to the Targett: Exploring the Role of Direct CNS Access in Modern Therapeutics to Solidify Local Administration in the Clinic
Synopsis
Highly interactive breakout discussions designed to crowdsource solutions, share cross-functional insights, and explore the practical barriers to clinical adoption.
- What safety, procedural, or monitoring strategies are most critical to de-risk intra- CSF and other direct CNS delivery methods in clinical trials?
- How can integration between therapeutic formulation and delivery systems be optimized to accelerate approval of combination products?
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open a wider audience debate.
3:00 pm Afternoon Break & Refreshments
3:30 pm Scientific Poster Session
Synopsis
This is an informal session to help you connect with your peers in a relaxed atmosphere to continue forging new and beneficial relationships. With an audience of CNS experts eager to hear the latest drug delivery innovations, you will have the opportunity to display a poster presenting your own research. Don’t miss out on the chance to connect, learn, and present.
TAILORING CNS DRUG DELIVERY: ALIGNING DISEASE, DELIVERY ROUTE & DEVICE FOR IMPROVED PATIENT EXPERIENCES
4:00 pm Through the Patient’s Eyes: Designing CNS Delivery for Trust, Tolerability & Long-Term Use
Synopsis
- How patients weigh delivery burden (e.g., implants vs. systemic) against expected benefit and adherence
- How communication, risk awareness, and control shape comfort with new delivery technologies
- Strategies to involve patients early through advisory boards, feedback, and co-design
4:30 pm Panel Discussion: Matching Mechanism to Disease – Optimizing CNS Delivery Strategies Across Indications to Enhance Treatment Efficacy & Safety
Synopsis
- Where systemic vs. direct (e.g., IT, ICV, convection-enhanced) routes are used
- How targeting location, BBB access, and regional specificity guide decision-making
- The impact of disease stage and progression on delivery strategy