Harness Technology Innovation Across Capsid Engineering & Delivery Routes to the CNS to Improve Biodistribution and Translate Gene Therapies for Neurological Diseases into and Through the Clinic

6th Annual Gene Therapy for CNS Summit

Maximize Gene Therapy’s Therapeutic Potential in Neurology

The 6th Gene Therapy for CNS Summit is returning to Boston with a refreshed agenda to match exactly with the priorities of the gene therapy field in 2024. With more companies than ever submitting INDs for neurological diseases, preparing for first-in-human trials, and novel capsids proving their worth in positive data readouts showing greater transduction, there’s a clear shift as the innovative promise becomes a reality.

Bringing in-depth content on how novel AAVs are performing in preclinical models, maximizing biodistribution through a variety of administration routes, and applying the significant progress and successes from the first approvals in rare, genetic diseases to more complex, larger patient populations like Alzheimer’s and Parkinson’s.

Join neurology experts who are leveraging gene therapy as the premier modality to tackle neurological disease, across translational, preclinical, R&D, and clinical teams who are dedicating their day-to-day to advance their neurological assets into and through the clinic.

CNS Gene Therapy Heats Up in 2024: Breakthroughs and What’s Next?

With ongoing investment, collaborative efforts, and exciting discoveries, it seems like there's groundbreaking news in CNS gene therapy every week. Here's a glimpse at some of the biggest stories shaping the future of neurological treatments:

  • Huntington's Disease Success: uniQure's AMT-130 trial shows an impressive 80% slowdown in disease progression. This therapy also received the first-ever FDA RMAT designation for Huntington's. Learn more from David Margolin, VP of Clinical Development at uniQure, on day 2 of the conference.
  • AskBio Initiates Recruitment to its Phase 2 Parkinson’s Disease Trail: AskBio's Phase 2 trial (REGENERATE-PD) is underway, building on the success of their Phase 1b study. This AAV2 GDNF gene therapy holds promise for treating moderate-stage Parkinson's Disease. Don't miss Krystof Banckiewicz, Chair of CNS Gene Therapy at AskBio, discuss the latest advancements in MRI-mediated delivery for this program.
  • Crossing the Blood-Brain Barrier: Researchers at the Broad Institute achieved a major breakthrough by engineering an AAV that can cross the BBB, paving the way for broader CNS gene therapy applications. Be sure to catch Ben Deverman's talk (Day 1) to learn more about this revolutionary technology.
  • Sangamo's Cutting-Edge Capsids: Sangamo's STAC-BBB capsid is leading the way in efficient BBB penetration, with CTA submission expected in Q4 2025. David Ojala, Director of AAV Engineering at Sangamo, will be presenting on "Developing Capsids and Cargo for Brain-Wide Epigenetic Regulation" (Day 1).

World-Class Speaker Faculty Includes:

Previously Attending Companies Include:

Photo of previously attending companies - Gene Therapy for CNS


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