3 Top Sessions

  1. ”NHS Perspective on the Implementation of Gene Therapy for SMA" Fiona Marley, Head of Highly Specialised Commissioning, NHS England
  2. “Gene Therapy for a Metachromatic Leukodystrophy (MLD)- A European Registration Case Study”
    David Uguen, Vice President, Regulatory Sciences, Orchard Therapeutics
  3. Workshop on “Utilising Focused Ultrasound Technology to Enhance Gene Therapy Delivery Across the BBB”, Nathalie Cartier-Lacave, CNS, AskBio
    Miroslaw Zabek, Professor of Neurosurgery, Interventional Neuro Center, Neurosurgery Clinic CMKP, Mazowiecki Szpital Brodnowski 

Accelerating Europe's Progress in CNS Gene Therapy

11 - 13 July | PARIS, FRANCE

”Really great presentations together with the convenience of remote attendance. The conference was a really great way to get up to speed on the latest in gene therapy of neurological disorders."
Johanne Kaplan, Chief Development Officer, ProMIS Neurosciences

“Overall great content and great ability to connect with relevant partners in the space. Excellent content, ample opportunity for learning and and the ability to connect with current and new partners in the GT space.”
Zach Carr, Biologics & Drug Delivery Program Manager, ClearPoint Neuro Inc.

“Phenomenal speakers and very intuitive, user-friendly software. Great opportunity to learn about cutting-edge gene therapy research in various neurological disorders.”
Jennifer Franks, Scientist, Bridgebio

Accelerating Europe's Progress in CNS Gene Therapy

Navigating Europe-Specific Challenges & European Regulators Requirements; Optimizing Preclinical Research in Neurological Models & Revolutionizing Gene Therapy Delivery to CNS Targets in the Clinic

With systemic toxicity challenges plaguing the AAV field, low-dose CNS indications are an ideal target for gene therapy. The plethora of neurological indications dominating gene therapy pipelines vary from rare neurological disorders to more common diseases including Parkinson’s, epilepsy and neurodevelopmental disorders.

As programs advance globally, the 3rd Annual Gene Therapy for Neurological Disorders Europe meeting is the field’s definitive forum to showcase the European landscape’s progress and specific regulatory challenges. Gene therapy developers from all of the world, including Europe’s brightest minds in CNS gene therapy, will unite in Paris to share their research progress and host an industry-dedicated discussion.

View the world-class speaker faculty and topics for discussion here

Your Expert Speakers Include:

Elena Gargaun

Clinical Research Director, R&D, Neurology Development


Fatima Bosch

Professor & Director, Center of Animal Biotechnology & Gene Therapy

Universitat Autònoma de Barcelona, Spain

Fiona Marley

Head of Highly Specialised Commissioning

NHS England

Nathalie Cartier-Lacave

Research Director


Stéphane Palfi

Chef de Service de Neurochirurgie, Directeur Equipe de recherche, Hôpital Henri Mondor

Université Paris-Est Créteil (UPEC)

Clarice GTx - Buttons

Agenda Highlights Include:

  • Utilizing NHPs and novel gene therapy tools to assess efficacy and biodistribution with different CNS administration routes
  • Highlighting the specific needs of MHRA and EMA and the specific challenges of running trials to optimize your European strategy
  • Optimizing preclinical model research with devices to improve CNS delivery translation into the clinic
  • Hearing from neurosurgeons at the forefront of CNS gene therapy delivery to anticipate future scale up challenges
  • Discussing the progress in brain targeting of technologies beyond AAV, and their ability to mitigate CNS toxicity
  • Discussing novel vector research for improved selectivity and distribution to the CNS
  • Evaluating the payer perspectives and addressing the commercial challenges of neurological gene therapies in Europe
  • Addressing the bottleneck of NHP availability and CNS-specific CROs in Europe

Join over 100 fellow preclinical, translational, clinical and commercial gene therapy experts from European-based companies including AviadoBio, Orchard Therapeutics, UniQure, UCB, VectorY, Lysogene, AskBio Europe, Roche, Spark Therapeutics, Sanofi and Janssen for improved competitive intelligence of the new and established European companies leading the gene therapy field for neurological disorders.

Who Attended in 2021:

2022 Partners:

Other Events In The Gene Therapy Series: