7:50 am Registration & Breakfast

8:50 am Chair’s Opening Remarks

  • Bryn Martin Vice President of Research, Drug Delivery, and CSF Sciences, Alcyone Therapeutics


9:00 am Maximizing Drug Uptake into the CNS Via Intrathecal Injection to Spearhead Clinical Efficacy

  • Haiyan Peng Associate Director, Research, Alnylam Pharmaceuticals


  • 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


  • 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


  • 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


  • 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

  • Darin Falk Chief Scientific Officer, Lacerta Therapeutics
  • Jeff Kurz Associate Director, Alnylam Pharmaceuticals


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


1:45 pm Breaking the Blood-Tumor Barrier to Revolutionize Primary Brain Tumor Treatment Development


  • 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

  • Cheng-Chia Wu Assistant Professor, Columbia University Irving Medical Center


  • 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


3:15 pm Highlighting Success in Antibody Shuttle-Mediated Transportation for Amyloid-Targeted Therapeutics


  • 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


  • 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

  • Anu Bhattacharyya Executive Director Biology/Nucleotide Repeat Disorders, PTC Therapeutics


  • 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

4:45 pm Chair’s Closing Remarks & End of Conference

  • Bryn Martin Vice President of Research, Drug Delivery, and CSF Sciences, Alcyone Therapeutics