Pre-Conference Workshop Day

Monday 11th July, 2022

11.00am -1.00pm

WORKSHOP A: Seeking Translatable Biomarkers of Neurological Disorders

Beyond rare diseases, neurological gene therapy pipelines are now turning to more common disorders. This workshop will explore the latest research in fluid biomarkers of neurological disease to detect treatment effects in gene therapy of neurodegenerative and neurodevelopmental disorders.

Attend this workshop to:

  • Discover the latest research in fluid neurological biomarkers for reducing invasiveness for patients
  • Discuss neuroinflammatory biomarkers to detect CNS immunogenic responses
  • Review the detection of gene therapy improvement

Workshop Leaders:

Charlie

Charlie Arber
Senior Research Fellow
University College London

11.00am -1.00pm

WORKSHOP B: Gene Therapy for Novel Indications

With gene therapy pipelines expanding into new target areas within neurology such as neuropathic pain and inner ear disorders, this workshop will showcase the challenges and opportunities of using gene therapy as an approach for these newer indications.

Attend this workshop to:

  • Discover the huge unmet clinical need of patients suffering from chronic pain conditions as the development of selective and efficacious NaV1.7 inhibitors have failed to demonstrate clinical benefit in multiple trials
  • Understand the biology behind neuropathic pain and the mechanism of action by which genetic therapies can intervene
  • Explore gene-based therapeutic approaches as a potential strategy to treat chronic pain conditions using in vivo data
  • Explore the hearing disorders suitable for gene therapy approaches
  • Discover intracochlear administration
  • Discuss the challenges and opportunities for gene therapies targeting the inner ear

Workshop Leaders:

Laurent

Laurent Desire
Director, Preclinical Development
Sensorion

Thomas

Thomas Chalberg
Founder & Chief Executive
Officer
Genascence Corp.

Workshop Leaders:

Charlie

Charlie Arber
Senior Research Fellow
University College London

Workshop Leaders:

Laurent

Laurent Desire
Director, Preclinical Development
Sensorion

Thomas

Thomas Chalberg
Founder & Chief Executive
Officer
Genascence Corp.

1.30 - 3.30pm

WORKSHOP C: Advancing Preclinical Models of Neurodegeneration for Gene Therapy Research

CNS drug development is a huge area of pharmaceutical research, and the main challenge still remains to recapitulate the human disease in preclinical models. With a combination of models being used to prove safety and efficacy before translating into the clinic, this workshop will explore the latest models available to ensure the most accurate predications possible. Both in vitro human iPSCderived neuron models, and in vivo disease models are critical tools for testing gene therapy modalities.

Workshop Leaders:

Jose

José Lanciego
Staff Scientist, Functional Neuroanatomy of the
Basal Ganglia Lab
University of Navarra

Ariana

Adriana Galvan
Associate Professor, Department of Neurology
Emory University School of Medicine

1.30 - 3.30pm

WORKSHOP D: Overcoming Gene Therapy Pricing & Reimbursement Challenges in Europe

As more gene therapy developers run clinical trials in Europe, the field must discuss the anticipated challenges ahead. Showcasing case studies of European approvals, and discussing current payer opinions, this workshop will enable you to improve your understanding of the European landscape as you advance your programs.

Workshop Leaders:

vICTOR

Victor Maertens
Government Affairs Manager
European Confederation of Pharmaceutical
Entrepreneurs (EUCOPE)

Workshop Leaders:

Jose

José Lanciego
Staff Scientist, Functional Neuroanatomy of the
Basal Ganglia Lab
University of Navarra

Ariana

Adriana Galvan
Associate Professor, Department of Neurology
Emory University School of Medicine

Workshop Leaders:

vICTOR

Victor Maertens
Government Affairs Manager
European Confederation of Pharmaceutical
Entrepreneurs (EUCOPE)

4.00 - 6.00pm

Gene Therapy Strategies for the CNS

Join this workshop to:

  • Evaluate the identification of therapeutic targets in neurology
  • Assess the delivery approaches available for CNS programs: ex vivo and in vivo gene therapy
  • Optimise the translational steps, from preclinical proofs of concepts to clinical trials
  • Discuss rare diseases to complex pathologies and… back

Workshop Leaders:

Nathalie

Nathalie Cartier-Lacave
CNS
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