10:00 am Networking Morning

Synopsis

Join your fellow gene therapy experts for a coffee morning to make new connections and catch up with friendly faces before the presentations start.

11:30 am Chair’s Opening Remarks

NAVIGATING EUROPEAN REGULATORS & PAYERS WITH NEUROLOGICAL GENE THERAPIES

12:00 pm Addressing the Regulatory Challenges of Clinical Trials in Europe with Gene Therapy for Neurological Disorders

  • Maria Beatrice Panico Leading Senior Medical Assessor, Clinical Investigations & Trials, Science, Research & Innovation Group, Medicines & Healthcare Products Regulatory Agency

Synopsis

  • Addressing routes of administration including intrathecal, intra cisterna magna, intra parenchymal
  • Discussing the recruitment challenges for rare diseases and new small subtypes created by precision medicine, etc
  • Highlighting the opportunities and challenges of decentralised trials

12:30 pm Gene Therapy for a Metachromatic Leukodystrophy (MLD)- A European Registration Case Study

  • David Uguen Vice President, Regulatory Sciences, Orchard Therapeutics

Synopsis

  • OTL-200 is an ex vivo autologous hematopoietic stem cell gene therapy approved by the European Medicines Agency (EMA) in 2020 and is currently the only
    approved treatment for MLD
  • Appreciate the details of a successful EU registration procedure for a gene therapy to treat metachromatic leukodystrophy
  • Hear about the various types of interactions with EMA before, during and after the registration

1:00 pm NHS Perspective on the Implementation of Gene Therapy for SMA

  • Fiona Marley Head of Highly Specialised Commissioning, NHS England

Synopsis

  • Lessons learnt from commissioning gene therapies in England
  • End-to-end process from horizon-scanning to service delivery
  • Practical considerations
  • The pivotal role of patient groups

1:30 pm Lunch Break

3:00 pm Panel Discussion: Navigating Regulatory Challenges in Europe

  • Fiona Marley Head of Highly Specialised Commissioning, NHS England
  • Maria Beatrice Panico Leading Senior Medical Assessor, Clinical Investigations & Trials, Science, Research & Innovation Group, Medicines & Healthcare Products Regulatory Agency

Synopsis

  • Targeting neurological disorders with gene therapy – rare to common disorders patient populations
  • Comparing the regulatory challenges between Europe and the US
  • What are the variances in payer opinions in Europe?

ASSESSING ADMINISTRATION ROUTE & BIODISTRIBUTION IN PRECLINICAL MODELS

3:30 pm Reverse Translation of CNS Gene Therapy – Using Clinical Insights to Inform Preclinical Biodistribution Studies in NHPs

  • Elena Gargaun Clinical Research Director, R&D, Neurology Development, Sanofi

Synopsis

  • Addressing the challenges in choosing a preclinical model
  • Interplay between preclinical results and clinical trial design
  • Ensuring valuable interpretation of results

4:00 pm Assessing CNS Gene Therapy Technology in Various Preclinical Models

Synopsis

  • Exploring novel molecular strategies for targeting CNS disorders
  • Showcasing dose-finding and bio-distribution studies in preclinical models
  • Assessment of gene therapies in patient-derived cells

4:30 pm Short Break

OPTIMIZING CNS CELL TARGETING WITH NOVEL VECTORS

5:00 pm Utilising AAV for High Gene Editing in Brain Circuits

  • Nicole Déglon Professor, Département des Neurosciences Cliniques (DNC), Laboratoires des Neurothérapies Cellulaires et Moléculaires, Centre Hospitalier Universitaire Vaudois

Synopsis

  • Improving gene editing safety with different transient Cas9 nuclease expression systems
  • Showing preclinical data for novel AAV-KamiCas9 for in vivo applications in the brain with intrastriatal injection
  • Utilising advanced light sheet microscope to image optically clear specimens according to the CLARITY protocol and multimodal image registration and connectivity analysis (MIRACL) to integrate the Allen brain atlas

5:30 pm Discussing AAV-ligand Conjugates for Improved Efficacy & Distribution in CNS

Synopsis

  • CNS delivery of gene therapy products remains challenging, in particular for achieving deep brain distribution of genetic materials
  • Chemical modification of AAV by new conjugation chemistry enables a rapid generation of rationally designed capsids
  • Generation of several conjugated AAV (coAAV) with improved in vivo transduction and biodistribution properties

6:00 pm Evaluating Gene Therapy Distribution & Delivery to the CNS

  • Ronald Crystal Professor & Chair, Department of Genetic Medicine, Weill Cornell Medical College

6:30 pm Panel Discussion: Discovering Novel Technology for CNS Disorders

  • Rodolphe Clerval Chief Executive Officer, COAVE THERAPEUTICS
  • Nicole Déglon Professor, Département des Neurosciences Cliniques (DNC), Laboratoires des Neurothérapies Cellulaires et Moléculaires, Centre Hospitalier Universitaire Vaudois
  • Maria Grazia Biferi CNS Preclinical Research Lead, Spark Therapeutics
  • Ronald Crystal Professor & Chair, Department of Genetic Medicine, Weill Cornell Medical College

Synopsis

  • What are the newer procedures for capsid screening platforms for tissue and cell tropism?
  • Which vectors are showing clinical progress?
  • Are there novel vectors showing CNS tropism and selectivity in NHPs?
  • What are the respective challenges for different vector types?

7:00 pm Chairs Closing Remarks

7:15 pm Scientific Poster & Pipeline Session

Synopsis

Celebrating being able to connect in-person at the first physical meeting in Europe, and Showcasing the latest CNS research in the gene therapy field to provide an opportunity to share expanding pipeline progress for companies and research centres pursuing neurological disorders.

End of Conference Day One