*All times are shown in EST

HIGHLIGHTING THE LATEST UPDATES IN NEUROLOGICAL DISORDERS TRIALS TO INFORM FUTURE DEVELOPMENT

8:30 am Chair’s Opening Remarks

8:40 am Intracisternal Delivery to the CNS of an AAV Gene Therapy Candidate for the Treatment of GM1 Gangliosidosis

Synopsis

  • Routes of delivery of AAV vectors to the CNS
  • Intracisternal (ICM) administration: bioavailability and safety considerations
  • Preclinical evaluation of ICM-administered AAVrh10 vector carrying human GLB1
  • Status of clinical trial in GM1 gangliosidosis patients

9:00 am Lessons Learned from Bringing Gene Therapy for Huntington’s Disease to the Clinic

  • David Cooper Vice President, Clinical Development, CNS, uniQure

Synopsis

  • Translating non-clinical experience into clinical administration procedure
  • Executing on the first-in-human gene therapy trial in HD
  • Adapting rapidly to optimise trial design

9:20 am Q&A Panel Discussion: Highlighting Key Clinical Progress in Gene Therapies for Neurological Disorders

10:00 am Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

10:30 am Morning Break

INTRODUCING NOVEL COMPANIES & GENETIC APPROACHES TARGETING THE CNS

11:00 am Gene Therapy Targeting Ectopically Expressed Kainate Receptors in Refractory Temporal Lobe Epilepsy

Synopsis

  • Ectopically expressed kainate receptors (KARs) trigger epileptic activity in refractory temporal lobe epilepsy
  • Using an engineered miRNA approach knocking down ectopically expressed KARs suppresses spontaneous recurrent seizures

11:20 am The Challenges & Opportunities of Targeting CNS Disorders with Lentiviral-Based Gene Therapy

  • Chris Mason Founder & Chief Scientific Officer, Avrobio

Synopsis

  • Ex vivo lentiviral gene therapy enables the engraftment of microglia-like cells throughout the CNS
  • Platform has demonstrated meaningful clinical effects in CNS in MLD and C-ALD patients
  • AVROBIO preclinical data demonstrates therapeutic transgene expression in the brain across multiple indications

11:40 am Q&A Panel Discussion: Exploring Different Genetic Approaches & Novel Targets for CNS Disorders

12:20 pm Lunch & Networking

IMPROVING TRANSLATABILITY OF GENE THERAPY RESEARCH FOR CNS DISORDERS

1:20 pm Learning from Rare Neurological Disorders for Future Development in Common Disorders

  • Lucie Bruijn Senior Director, Translational Medicine, Novartis Gene Therapies

Synopsis

  • Talk details to follow

1:40 pm Targeted Gene Therapy: Building for Clinical & Commercial Success

Synopsis

• Rationale for CNS targeted Gene Therapy
• Update on PTC Therapeutics Gene Therapy programs
• Essentials of Gene Therapy Product Launch

2:10 pm Correction of the MPSIIIB Phenotype Following Delivery of NAGLU into the CNS with a Novel AAV Capsid Variant

2:25 pm Q&A Panel Discussion: Optimizing Translation & Moving into Other Indications

DISCUSSING CLINICAL TRIAL DESIGN & REGULATORY CHALLENGES

2:45 pm Short Break

3:00 pm Optimising Clinical Trial Design for Adult Patient Populations & Neurodegenerative Diseases

Synopsis

  • Disease-modifying AAV9-based gene therapies for genetically gefined patient sub-populations suffering from neurodegenerative disorders
  • Choosing the right biomarker-based and clinical endpoints for FIH trials
  • Incorporating patient experience when designing a clinical trial for genetic forms of PD and FTD
  • Executing global FIH AAV9-based trial: challenges and opportunities

3:20 pm Q&A Panel Discussion: Addressing Clinical Trial & Regulatory Challenges

4:00 pm Chair’s Closing Remarks