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