Pre-Conference Workshop Discussions

9.00 AM - 12.00 PM | Workshop A: Gene Therapy: Exploring Avenues for Targeted Therapeutic Intervention in ALS

Impeded by limited therapeutic targets and difficulty in delivery to the brain and spinal cord, gene-therapy-based approaches to treating ALS have been historically challenging. However, recent advances in interrogating disease mechanisms and identifying causative genes combined with progress made in CNS targeting, gene delivery, and gene editing techniques have opened new doors of therapeutic possibility. Join this workshop to keep up-to-date with the latest progress in gene-therapybased approaches to targeting ALS.

Highlights include:

  • Examine mutations occurring in C9orf72, SOD1, TARDP, and FUS to illuminate opportunities for therapeutic intervention
  • Navigate novel strategies to overcome biological barriers with targeted delivery methods
  • Explore the landscape of current gene therapy trials to delineate the best therapeutic strategies
  • Discuss how progress in targeting genetically defined populations can be translated to sporadic disease

Workshop Leader:

Mansuo Hayashi

Mansuo Hayashi
Chief Scientific Officer
Prevail Therapeutics

1.00 PM - 4.00 PM | Workshop B: Digital Biomarkers: Integrating More Frequent, Sensitive & Objective Monitoring in Clinical Trials

As technology develops, it is clear that digital biomarkers have the potential to transform approaches to early diagnosis of ALS and could be used as a sensitive outcome measure to accurately evaluate the efficacy of novel therapeutics. Substantial heterogeneity in clinical presentation remains a challenge in monitoring patients with ALS, but could digital biomarkers represent a move towards a decentralized, patient-centric, and effective measurement of efficacy?

Highlights include:

  • Exploring suitable digital biomarkers for clinical presentation of ALS
  • Examining feasibility and reliability of digital biomarkers as indicators of efficacy and disease progression
  • Could digital biomarkers be used for early diagnosis of ALS? Investigating practical opportunities to employ non-invasive digital biomarkers

Workshop Leader:

James Berry Harvard
MicrosoftTeams-image (15)
Ram Miller Novartis

James Berry
Chief, Division of ALS & Motor Neuron Diseases
Massachusetts General Hospital

Kelley Erb
Director, Digital & Quantitative Medicine

Ram Miller
Director Translational Medicine