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.

11:50 am Chair’s Opening Remarks

HIGHLIGHTING PROGRESS IN GENE THERAPY FOR NEURODEGENERATIVE DISEASES

12:30 pm Demonstrating Efficacy & Neuroprotection in Early Stage Neurodegenerative Patients – Parkinson’s

  • Stéphane Palfi Chef de Service de Neurochirurgie, Directeur Equipe de recherche, Hôpital Henri Mondor, Université Paris-Est Créteil (UPEC)

Synopsis

  • Talk details to follow

1:00 pm Initiating Clinical Trials with Gene Therapies Targeting Neurodegenerative Disorders

  • Paulo Falabella Vice President - Clinical Development & Operations, REGENXBIO

Synopsis

  • Clinical Experience with AAV9-based Gene Therapies Targeting
    Neurodegenerative Disorders
  • Interim analysis of the RGX-121 gene therapy first in human study for the treatment of severe Mucopolysaccharidosis Type II
  • Interim analysis of the RGX-111 gene therapy first in human study and a single patient IND for the treatment of severe Mucopolysaccharidosis Type I

1:30 pm Lunch Break

EARLY CLINICAL CONSIDERATIONS: CLINICIAN & NEUROSURGEON PERSPECTIVES

3:00 pm Devices in the Clinic: Catheters, Combinations & Surgical Strategies

  • Tim Goble Senior Director, Device Development & Product Delivery, Sio Gene Therapies

Synopsis

  • Exploring different catheter technology including SmartFlow and MEMS and evaluating the current landscape of surgical devices (Frame, Clearpoint, Starfix)
  • Discussing recent modifications to 3D printable customized frames for burr hole/ twist drill approaches
  • Reviewing surgical strategies – starting early in TPP process, intra op and post operative imaging and changing surgical strategies during a clinical trial

3:30 pm Current Clinical Challenges of Delivery: Simultaneous Injection & Technology Implementation Novel – Personal Experience

  • Miroslaw Zabek Professor of Neurosurgery, Interventional Neuro Center, Neurosurgery Clinic CMKP, Mazowiecki Szpital Brodnowski, Mazowiecki Szpital Brodnowski; UniQure Huntington’s Program

Synopsis

  • Sharing experience in gene therapy of Multiple System Atrophy, Parkinson’s disease, AADC deficiency and glioblastoma multiforme
  • In small treated groups for MSA and PD, discussing only the basics of gene therapy of these diseases and the surgical technique without discussing the results, and results larger groups of treated patients will be presented
  • Discussing the challenges in brain gene therapy relating to the methodology, surgical techniques, methods of infusion, infusion rates and the time which the patient has to spend in the MR gantry

 

4:00 pm Panel Discussion: Improving Clinical Trials in Anticipation of Scaling Up

  • Miroslaw Zabek Professor of Neurosurgery, Interventional Neuro Center, Neurosurgery Clinic CMKP, Mazowiecki Szpital Brodnowski, Mazowiecki Szpital Brodnowski; UniQure Huntington’s Program
  • Tim Goble Senior Director, Device Development & Product Delivery, Sio Gene Therapies
  • Stéphane Palfi Chef de Service de Neurochirurgie, Directeur Equipe de recherche, Hôpital Henri Mondor, Université Paris-Est Créteil (UPEC)

Synopsis

  • Addressing the scalability and integration of gene therapy delivery devices – specialist equipment, training and centres
  • Highlighting current clinical accuracy of delivery
  • Predictability of the infusion pattern with preclinical modelling

4:45 pm Short Break

TARGETING YOUNGER PATIENT POPULATIONS: GENE THERAPY FOR NEURODEVELOPMENTAL DISORDERS & EPILEPSY

5:15 pm Exploring Gene Therapy for Epilepsy

  • Annahita Keravala Senior Vice President, Head of Gene Therapy, CODA Biotherapeutics

Synopsis

  • Targeted CODA-receptor expression for mTLE using next-gen AAV vectors
  • Highlighting AAV capsid selection criteria for epilepsy target cells
  • Optimising the AAV expression cassette
  • Sharing preliminary data in two models of focal epilepsy

5:45 pm Panel Discussion: Targeting Younger Patient Populations

  • Richard Porter General Manager, Epilepsy - Corlieve Subsidiary, UniQure
  • Annahita Keravala Senior Vice President, Head of Gene Therapy, CODA Biotherapeutics
  • Mitra Tavakkoli Head of Neurodevelopmental Disorders & Genetic Epilepsies, Vice President, Clinical Development, Taysha Gene Therapies

Synopsis

  • Discussing ethical considerations around informed consent
  • Addressing appropriate outcomes and emergent secondary pathologies
  • Access and pricing issues for gene therapies targeting rare neurological disorders
  • Clinical translation of neurodevelopmental products – determining transgene expression

6:30 pm Chairs Closing Remarks

End of Conference