In Vivo Therapies: Current Directions and Future Development

Chair

David Peritt, Founder and Chief Scientific Officer, Lupagen

9am Presentation Title: In Vivo Therapies: Current Directions and Future Development

• Non-viral, Non-Lipid based In-Vivo gene editing
• Engineering Gene Editing

Philip Roche, Chief Executive Officer, Jenthera Therapeutics

9.15am Presentation: From NEXT to NICE: from Nanoparticles-based Ex vivo Therapeutics to Nanoparticles-based In vivo Cell engineering

• Hybrid/polymeric nanoparticles as a solution to improve CAR T-cell manufacturing process and CAR T-cell product attributes.
• Key performance criteria for an ideal in vivo CAR re-infusable product
• Translational hurdles from ex vivo to in vivo CARs

Cécile Bauche, Founder & Chief Scientific Officer, Alaya Bio

9.30am In Vivo CAR-T. Liberating Cell Therapy From The “Out Of Scope” Penalty Box.

• EXUMA is advancing autologous cell therapies enabled by CD3 directed lentivectors and a proprietary FITNESS DRIVER synthetic intracellular signaling construct for lymphodepletion free cell therapy (rPOC platform)

• Clinical POC from EXUMA’s ongoing rPOC CD19 SC clinical study will inform the safety and efficacy of the novel T and NK effector cell phenotype generated by the rPOC process

• EXUMA is advancing a cost-effective mRNA therapeutic encoding a synthetic car ligand (SCL) to provide the physician control of cell therapy pharmacokinetics with the potential to increase Cmax, AUC and retreat/re-expose patients to CAR therapy without the need for a subsequent infusion of cell therapy or treatment with LV

Gregory Frost, Chairman & Chief Executive Officer, EXUMA Biotech

9.45am Q&A