8:45 am Coffee & Networking
9:15 am Chairman’s Opening Remarks
Accelerating the Next Frontier of Cell Therapy with Next Generation Gene Editing
9:30 am Cell-Specific In Vivo Gene Delivery & Genome Editing via Retargetable Fusogens
Synopsis
• Highlight the in vivo delivery platform that Sana has developed for gene therapy
• Discuss progress in targeting T Cells and HSCs
• Provide examples of pre-clinical studies with delivery of integrating vectors and Gene editing machinery
10:00 am AAV-Mediated In Vivo Base Editing Prevents Cardiomyopathy in Mice
Synopsis
• One dose of AAV encoding base editors could modify the disease-causing genomic nucleotide in ~80% of left ventricular cardiomyocytes
• This editing prevented the onset of hypertrophic cardiomyopathy, even in mice that were just two months away from developing symptoms
• Delivery of Cas9 nuclease to destroy the dominant mutant allele could also prevent cardiomyopathy but appeared to have a more narrow therapeutic window
10:30 am Roundtable Discussion- Transforming the Cell Therapy Space Using Precision Delivery Platforms with Next Generation Editing Machinery
Synopsis
• Developing safer and more precise genetic modifications with complex gene editing strategies such as base
editing/prime editing
• Overcoming the challenge of unpredictable off-target edits
• Improving cellular viability with reduced possibility of double-strand breaks
11:00 am Morning Break & Refreshment
Evaluating Therapeutic Payloads Being Utilized for In Vivo Delivery
11:30 am Development and Evaluation of Non-Viral RNA Delivery Platforms
Synopsis
• Commentary on the advantages and applications of RNA based medicines
• Commentary on the challenges associated with delivering RNA based medicines to the correct location in the body for the right amount of time and at the proper dose
• Commentary on synthetic non-viral delivery vectors for controlled RNA delivery to cell populations in vitro and in vivo
12:00 pm Panel Discussion: War of the Payloads – Addressing the Advantages & Disadvantages of Transient VS Direct Integration
Synopsis
• Discussing the use of non-stable integration with a DNA payload for greater safety and control
• Discussing the challenges associated with stable integration, including lack of control in the context of proliferated cells
• Re-thinking re-dosing strategies for enhanced efficacy
• Evaluating the implications of removing lymphodepletion for in-vivo approaches, and associated effects on persistence/durability
12:45 pm Lunch & Networking
Unveiling the Full Potential of In Vivo Engineering Therapeutics to Treat Patients in Oncology and Beyond
1:45 pm Delivering Genetic Cargoes to Cells Of Choice In Vivo Using Non-Viral Delivery Vectors
Synopsis
- Development of non-viral delivery vectors, and the delivery of multiple different payloads to cells.
- In vivo delivery to immune cells
- Exploring the use of these vectors for diverse indications.
2:15 pm Mastermind Session: Discussing the Translatable Applications of In- Vivo Therapeutics in Oncology, Autoimmunity, Infectious Disease & More
Synopsis
• Deep dive session comparing and contrasting in vivo engineering of therapeutic cells to treat a wider range of disease indications including solid tumor, cardiovascular condition, IBD lupus, HIV and more
• Discussing the blockbuster potential for in vivo approaches to bring lost lasting treatments to areas of high unmet need