Gene Therapies: Critical Considerations on Developing the Transformative Genetic Medicine of the Future

07 Sep 2023
Stream 3

15:30am Chair Introduction 

Chairperson: Luís Pereira de Almeida, Professor & Group Leader, University of Coimbra


15:35am Presentation: Developing the next generation gene therapy for the treatment of Geographic Atrophy (GA)

  • The burden and challenge of treating GA including current therapeutic options
  • Complement Therapeutics innovative asset CTx001, an effective and durable gene therapy
  • In vitro and in vivo data demonstrating the potential of CTx001 as a best in class treatment for GA

Rafiq Hasan, Chief Executive Officer, Complement Therapeutics  


15:50am Presentation: Next Generation Bicistronic Gene Therapies for Chronic Ophthalmic Disorders

  • Ikarovec’s bicistronic AAV constructs simultaneously target multiple clinically relevant disease pathways, thereby improving efficacy and offering improved long-term sight preservation.
  • Ikarovec has fully equipped laboratories and established, in-house in vivo ophthalmic disease model capabilities for rapid, flexible, and cost-effective development.
  • Two lead candidates for the treatment of age-related macular degeneration and geographic atrophy have demonstrated improved efficacy over mono-therapies in key proof-of-concept studies, and are primed for clinical development.

Emily Warner, Principal Scientist, Ikarovac


16:05am Presentation: Prometheus – Novel Low-Cost Gene Therapy Platform with Dose Adjustment Capability

  • Remedium’s novel dose-adjustable gene therapy platform allows for the delivery of a broad range of subcutaneously administered biologics as a single injection gene therapy.
  • The system provides an ultra-low-cost, safe, and durable means of delivering genes to subcutaneous tissues for the expression of a broad range of therapeutic transgenes.
  • The platform technology for the first time enables safe and effective delivery of biologics as a single injection gene therapy with post-treatment dose up- or down-titration capability.

Frank Luppino, President & Chief Executive Officer, Remedium Bio


16:20am Gene Therapy for Brain Disorders 

  • The case of the SCA3 neurodegenerative disorder  
  • Gene addition, gene silencing and gene editing 
  • Delivery to the brain: AAV vectors and Extracellular vesicles 
  • The challenges 

Luís Pereira de Almeida, Professor & Group Leader, University of Coimbra

16:35 Closing Panel with Q&A  


Luís Pereira de Almeida
Professor & Group Leader
University of Coimbra
Rafiq Hasan
Chief Executive Officer
Complement Therapeutics
Emily Warner
Principal Scientist
Frank Luppino
President & Chief Executive Officer
Remedium Bio