7:50 am Registration & Breakfast
8:50 am Chair’s Opening Remarks
BYPASSING THE BLOOD-BRAIN BARRIER WITH TRANSLATABLE DIRECT DELIVERY SYSTEMS
9:00 am Maximizing Drug Uptake into the CNS Via Intrathecal Injection to Spearhead Clinical Efficacy
Synopsis
- Optimizing efficiency of chronic delivery via ICV injection
- Enhancing therapeutic diffusion from the cerebral ventricular system to the target tissue
9:30 am Optimizing Technical Workflow to Safely & Specifically Deliver Gene Therapies Directly to the Brain Target Region
Synopsis
- Maximizing therapeutic distribution in the CNS with convection-enhanced delivery
- Outlining key considerations to be made when planning a safe and effective trajectory to the brain target
- Exploring scale-up potential to allow for widespread clinical utility of convection-enhanced delivery
10:00 am Circumventing the Blood-Brain Barrier Via Intra-CSF & Convection Enhanced Delivery of Glioblastoma Therapeutics
Synopsis
- Assessing therapeutic biodistribution in the CNS via ICV and CED
- Achieving maximal and localized brain tissue penetration by circumventing the BBB
- Exploring potential applications of these delivery systems for neuro-oncology and neurological indications in the clinic
10:30 am Morning Break & Refreshments
11:30 am Elevating Safety & Efficiency of Gene Therapy Programs with Direct Intra-CSF Administration Routes
Synopsis
- Tailoring capsids for specific intra-CSF delivery routes depending on the indication to be targeted
- Identifying the optimal dosage window for therapeutics delivered by ICM to achieve desired expression levels in the target of interest
- Mitigating risks of brainstem injury during ICM delivery
12:00 pm Roundtable Discussion: Evaluating Utility & Clinical Acceptance of Promising Direct CNS Delivery Modalities
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas:
- What are the greatest safety risks to be mitigated to advance clinical utility of intra-CSF delivery methods?
- How can synergy between the drug and the administration device be improved to streamline development and approval of drug-device combination products?
12:45 pm Lunch & Networking Break
BENCHMARKING BLOOD-BRAIN BARRIER BIOLOGY IN NEURO-ONCOLOGY
1:45 pm Breaking the Blood-Tumor Barrier to Revolutionize Primary Brain Tumor Treatment Development
Synopsis
- Overcoming the treatment blockade presented by primary brain tumors surrounded by an intact blood-brain barrier
- Reviewing lessons learned from brain tumor studies evaluating ability of drugs to cross the BBB
- Exploring the promise of focused ultrasound to augment exposure of primary brain tumors to non-BBB penetrant anti-cancer compounds
2:15 pm Novel Ultrasound Technology: Accelerating Access to Glioblastoma & Pediatric Gliomas While Minimizing Toxicity for Improved Treatment Outcomes
Synopsis
- Showcasing successful clinical procedure for safely and efficaciously carrying out microbubble-based BBB opening
- Maintaining selectivity for chemotherapy while avoiding peripheral exposure through careful timing of BBB opening
- Exploring possibilities for further clinical applications of focused ultrasound-mediated delivery to the CNS
2:45 pm Afternoon Break & Refreshments
BREAKING BARRIERS: TRANSLATIONAL & CLINICAL PROGRESS WITH BBB-CROSSING TECHNOLOGIES
3:15 pm Highlighting Success in Antibody Shuttle-Mediated Transportation for Amyloid-Targeted Therapeutics
Synopsis
- Identifying the optimal bispecific antibody format for maximizing CNS exposure
- AT-07, the conjugation of AT-04 to a VNAR-derived brain shuttle, enhanced brain penetration in a mouse model and has the potential to increase effectiveness in clearing neuropathic fibrillar deposits
- AT-07 demonstrated a 10-fold increase in brain penetration versus AT-04 in a PK study in wild-type mice
3:45 pm Development of Next Generation CNS Drugs: Trontinemab, an Anti-Amyloid Beta Brainshuttle Antibody
Synopsis
- Developing “Brainshuttle”, a versatile platform to enhance brain delivery of therapeutic proteins and other cargos across the blood brain barrier via receptor mediated transcytosis
- Showcasing translational strategies and learnings from the Brainshuttle antibody fusion trontinemab: The journey from mice to patients, towards a new generation of Amyloid beta lowering therapies
4:15 pm Optimizing Small Molecule Therapies for Maximal Blood-Brain Barrier Penetration
Synopsis
- Optimizing BBB-crossing capabilities of small molecule candidates by enhancing influx and minimizing efflux
- Maintaining sufficient molecular transportation to and within the CNS in nonclinical through to clinical species
- Demonstrating successful target engagement in the brain region of interest to advance clinical studies