6th Gene Therapy for CNS Summit – What’s it About?

2024 is poised to be a breakthrough year for neurological treatments. Gene therapy is at the forefront, offering a one-time, safe, and potentially curative solution for these devastating diseases. The 6th Gene Therapy for CNS Summit reflects this progress, featuring the latest advancements in AAV capsids, administration routes, and preclinical models. With more companies submitting INDs for neurological diseases than ever before, the summit brings together leading experts to translate these early successes into treatments for complex conditions like Alzheimer's and Parkinson's Disease.

Join us to hear the latest and greatest on:

  • Exploring breakthroughs in CNS-tropic capsid design for superior neuron targeting and improved therapeutic outcomes
  • Delving into real-world clinical data on gene therapy for neurodegenerative diseases, gaining insights for future clinical development
  • Optimizing novel administration routes to maximize biodistribution and target specific regions of the CNS for enhanced efficacy
  • Standardizing CNS Biomarkers to validate and utilize reliable biomarkers to accurately track disease progression and treatment responses

Uncover innovative approaches to determine optimal dosing for CNS gene therapy, ensuring effective translation from preclinical models to the clinic.

Join Your Peers to:

Fast-Track Your Clinical Development: Learn from the successes of uniQure and Biogen in gene therapy for neurodegenerative disorders. Gain insights into endpoint selection and navigate clinical development with greater confidence

Revolutionize CNS Targeting: Discover the latest advancements in CNS-tropic capsid development to achieve flood brain barrier penetration and reduce dosage with Affinia Therapeutics, Dyno Therapeutics, and The Broad Institute

Optimize Direct Brain Delivery and explore the groundbreaking clinical data on MRI-guided administration of gene therapy to the brain with AskBio

Overcome the limitations of allometric scaling and discover innovative approaches to determine optimal dosage in translational studies with Encoded Therapeutics and Biointaxis

Standardize and validate CNS Biomarkers to conduct robust longitudinal studies and establish reliable clinical outcome reference standards with latest insights from Grace Sciences

What's New for 2024:

Harness the opportunity for in-depth, small group discussions surrounding novel vector development and delivery, gene editing for CNS disorder, in vivo models, and regulatory framework to uncover novel technologies and propel your pipeline with Latus Bio, UCB, Arbor Bio and Regel Therapeutics

Discuss the role that gene therapy development will play in the future of CNS disease management and potential cures with Nysno Bio and EG427

updated audience breakdown

What Past Attendees Have Said:

Great content and wonderful representation of different pharma and biotechs in the field’

Jose Corleto, Astellas

'This was a fantastic meeting! Great line-up of industry speakers and networking opportunities’

Anindya Sen, Prevail Therapeutics

‘Great line-up of speakers and vibrant atmosphere!’

Fernando Aleman, Navega Therapeutics