CONFERENCE DAY ONE
7.30 Check-In, Coffee & Light Breakfast
8.20 Chair’s Opening Remarks
AN EMERGING ERA OF CNS DRUG DELIVERY INNOVATION: HOW DO WE DETERMINE WHICH ROA IS BEST?
8.30 Selecting the Optimal Route of Administration Based on Delivery Vehicle Compatibility
Synopsis
- Reviewing the advantages and disadvantages of different RoAs
- Deciphering which RoAs are most suited to targeting different areas of the brain
- Evaluating the right RoA for different therapeutic modalities
9.00 Introducing a Minimally Invasive Precision Drug Delivery Platform That Enables Administration of Therapy to Intricate Brain Regions for a Diverse Range of Therapeutics in Neuro-Oncology & Neurology
Synopsis
- Presenting a novel precision drug delivery platform that outperforms conventional methods by maximizing on-target efficacy and minimizing systemic side effects
- Reviewing pre-clinical safety studies comparing Bionaut motion to standard ventricular catheter insertion in large animals
9.30 Panel Discussion | Assessing the Invasiveness of Local Administration Over Systemic Injection: Do the Risks Outweigh the Rewards?
Synopsis
- Making the case that a given CNS disorder requires local delivery: Is there sufficient evidence to support the requirement for a direct RoA?
- How does level of invasiveness differ depending on drug modality? Are genetic medicines safer to consider for local delivery due to one-time dosing?
- Which brain regions hold the most promise for targeting through device-mediated approaches?
10.00 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 Morning Break & Refreshments
Track 1: Systemic Delivery Chair: Yuan Yuan, Associate Scientific Director, Biogen
DEVELOPING NEXT GENERATION ANTIBODY CONJUGATES & EXPLORING BEYOND TRANSFERRIN
11.00 Translating Successful Learnings from J-Brain Cargo® to Innovate Second Generation Antibodies with Enhanced BBB Penetration in Other CNS Indications
Synopsis
- Outlining rationale for successful development of an antibody shuttle platform to increase brain uptake while managing risks of anaemia
- Applying transferrin-mediated transcytosis technology to optimize delivery of biologics to treat neurodegeneration, neuroinflammation, and more
11.30 Overcoming Translational Challenges of Cross Reactivity to Develop a Clinically Relevant BBB-Penetrant Antibody Platform
Synopsis
- Discussing the translational barriers of developing biologic-based drugs to cross the blood-brain barrier
- Navigating differences in BBB receptor profiles between in vivo models to ensure cross-reactivity
- Increasing confidence of in vivo toxicity studies to ensure safe translation to humans
12.00 Presenter Q+A & Audience Discussion
Synopsis
What do we mean by 'next generation' antibodies and how can we enhance their clinical relevance?
Track 2: Direct & Local Delivery Chair: Chris Kadamus, Executive Director of Engineering, DDCS, Eli Lilly
OVERCOMING SAFETY & TOXICITY CHALLENGES OF ICV & ICM TO TRANSFORM THEIR CLINICAL APPLICATIONS
11.00 Long-Term Chronic Delivery of VPA to the ICV: A Safety Summary
Synopsis
- A delivery system of including implanted drug pump and intracranial catheter was accomplished safely in 27 subjects
- The intracranial catheter could be implanted on target
- Long-term delivery of the drug to the ICV was safe with no adverse anatomical or toxicity signals
11.30 Addressing the Safety Concerns of Cisterna Magna Injections to Mitigate Brainstem Injury & Toxicity Risks
Synopsis
- Alleviating risks of localized toxicity associated with ICM administration in the clinic
- Identifying the optimal dosage window for therapeutics delivered by ICM to achieve desired expression levels in the target of interest
- Assessing the feasibility of ICM in clinical practice across different patient populations
12.00 Presenter Q+A & Audience Discussion
Synopsis
What must be done to transform the clinical scalability of these technologies through phase I & beyond?
12.30 Lunch & Networking
TRANSFORMING ABILITIES OF IN VITRO BBB MODELS TO ASSESS BRAIN DELIVERY
1.30 Advancing the Ability of In Vitro & In Vivo BBB Platforms to Test Receptor-Mediated Transcytosis of BBB Shuttles
Synopsis
- Replicating the complexity of receptor-mediated transcytosis in vitro
- Demonstrating correlation of the in vitro and in vivo results
- Enhancing confidence of in vitro studies for testing biologic transport across the blood-brain barrier
2.00 Leveraging Microfluidic Technology to Recapitulate & Characterize Blood-Brain Barrier Dysfunction
Synopsis
- Showcasing advancements in microfluidic capabilities
- Understanding blood-brain barrier leakage and permeability in diseased vs non-diseased states
- Applying microfluidic advancements to successfully study BBB transcytosis
2.30 Panel Discussion | Reviewing the Current Ecosystem of Available In Vitro Models for Studying Blood-Brain Barrier Permeability & Delivery
Synopsis
- How does the data you are looking to generate determine which model is most fit for purpose?
- How do you identify the most suitable model for testing each therapeutic modality?
- What key factors are absent in current in vitro models compared to the data obtained from mouse experiments?
BENCHMARKING EARLY CLINICAL ADVANCES IN DEVICE-MEDIATED INTRATISSUE DELIVERY
1.30 Exploring Engineering & Hardware Innovation in Device-Mediated Delivery Systems
Synopsis
- Outlining recent technological advancements in active implants and catheter mediated deliveries
- Navigating the engineering and product development issues that come with clinical scalability
- Overcoming challenges related to dosing, implantation and device refillability
2.00 Optimizing Dose Levels for Convection-Enhanced Delivery to Ensure Greater Transduction to Deep Brain Regions
Synopsis
- Exploring advances in gene therapy delivery through CED
- Achieving efficient delivery at a given dose level
- Navigating complex device architecture to achieve greater depth of penetration
Smrithi Padmakumar, Drug Product Development Senior Scientist, Spark Therapeutics
2.30 Roundtable Discussion: Scaling Up Convection-Enhanced Delivery for a Wider Scope of Clinical Applications: What Will it Take to Get There?
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas.
How can we scale this up & move away from real time monitoring to perform CED in a standard operating room? How can we ensure efficient quality control in the absence of real time imaging?
What else can be done to shorten the procedure time to allow CED to be scaled for more prevalent indications?
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 wider audience debate
3.30 Afternoon Break & Refreshments
REVIEWING NOVEL PK/PD MODELLING PROGRESS TO ASSESS BRAIN DELIVERY
4:00 Developing a Translational PBPK Model to Understand Species-Related Differences in TfR-Mediated Transcytosis of Large Molecules to the Brain
Synopsis
- Elevating understanding of the desired drug profile for efficient TfR-mediated delivery to specific brain targets Showcasing data from rats, monkeys, and human TfR knock-in mice
- Implications for applying the model to provide precise human dose projection of drugs leveraging TfR-mediated transcytosis
4:30 Determining BBB Penetration & CNS Biodistribution with Enhanced PKPD Modelling Capabilities
Synopsis
• Highlighting recent advancements in the development of PKPD efficacy models
• Studying delivery efficiency, target engagement and bioavailability of MDM2
inhibitors and radio-chemo sensitizers in the CNS
• Enhancing knowledge of their pharmacokinetic and pharmacodynamic
properties
5:00 Chair’s Closing Remarks
5:10 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.