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