Conference Day One
7:30 am Check-In & Light Breakfast
8:25 am Chair’s Opening Remarks
Spotlighting Key Advances in Genome Editing Platforms
8:30 am DeepXE: A Deep Learning Approach to Modeling & Designing Efficient CRISPR-CasX Guide RNAs for Therapeutics
Synopsis
- Showcase the first predictive model approach for the CasX-based genome editing platform, helping to unlock its therapeutic potential
- Demonstrate how combining high-throughput screening with deep learning accelerates the identification of potent and precise CRISPR edits across multiple targets
- Highlight the growing role of predictive modeling to advance next generation CRISPR-based therapeutics
9:00 am Session Reserved for Aldevron
9:30 am Gene Editing Case Study: Accelerating Therapeutic Development Through nChroma Bio’s Platform & Delivery Innovations
Synopsis
- Understand how epigenetic editing and base editing can facilitate durable silencing with no off-target changes in expression
- Assess how designing the platform alongside the delivery vehicle is essential to streamline development
- Appreciate the benefits of focusing on in vivo editing compared to ex vivo
10:00 am Platform Overview: Introduction to INDUCE-seq®, a PCR-free Method for Genome-wide Mapping of Double-strand DNA Breaks in Days, Not Months
Synopsis
Therapeutic Development:
- Data on confirming gene editing efficiency via on-target break detection Applications in iPSC and T-cell Therapies:
- Evaluation of base editor tools using INDUCE-seq®
Preclinical Safety Assessment:
- Results from the NIST genome editing program and collaboration with HESI
- Data from studies with the Innovative Genomics Institute (IGI) supporting gene editing INDs
Guide RNA Screening:
- Progress on SafeGuide AI, a predictive tool to differentiate safe versus high-risk gRNA designs
10:30 am Morning Refreshment Break & Speed Networking
Synopsis
Put a face to a name: This session is the perfect opportunity to get face-to-face time with key opinion leaders, leading companies, and dedicated researchers pioneering genome editing. Establish meaningful connections and gain individual insight beyond the papers and press releases into innovative platforms and shared challenges.
Discovery & Novel Technologies
Highlighting Innovations & Technology Enhancing Genome Editing Therapy Development
11:30 am Utilizing AI to Engineer CRISPR Chassis to Effectively Reach Specific Target Sites
Synopsis
- Discuss the potential for AI to cut the time and cost of developing genome editing therapeutics
- Stipulate how AI optimized proteins can facilitate desirable modifications in cell models
- Underscore the value of unique data sets in unlocking part of the design space with genAI that is not otherwise accessible
12:00 pm Optimizing CasPhi for MB-111: A Multi-Component Engineering Problem
Synopsis
- Explore how MB-111, a single-dose, in vivo gene editing therapy to treat Familial Chylomicronemia Syndrome, Persistent Chylomicronemia, and Severe Hypertriglyceridemia, inactivates APOC3, resulting in durable lowering of triglycerides
- Understand that at the core of MB-111 is Mammoth Biosciences’ proprietary engineered CasPhi system, a hypercompact CRISPR nuclease derived from phage
- Investigate how engineering CasPhi required balancing an array of parameters including nuclease and guide RNA potency, fidelity and stability to result in saturating editing at a clinically relevant dose
12:30 pm Lunch Break & Networking
Exploring Delivery Vehicles to Target the Liver & Beyond
1:30 pm Investigating Approaches for Hepatic & Extra-Hepatic Delivery
Synopsis
- Outline the key challenges facing tissue-specific delivery of genome editing platforms
- Explore non-viral delivery approaches to improve the targeting efficiency of hepatic tissue
- Understand how passive and active ligand-based targeting strategies can be leveraged to reach extra-hepatic tissues, enabling a broader range of indications to be treated
2:00 pm Utilizing Ionizable & Non-Ionizable Lipids to Optimize LNP Extra-Hepatic Delivery
Synopsis
- Discuss the advantages of LNPs including the ability for mRNA, transient expression, lack of integration and reduced immunogenicity
- Understand how ionizable lipids are essential for the proper packaging of the LNP, endosomal escape and subsequently for the importance of developing novel ionizable and nonionizable lipids that can improve targeting outside the liver
- Highlight how the development of novel LNP formulations found to be more potent and more efficacious, are crucial for the future of genome editing therapeutics
2:30 pm Developing Delivery Technology to Precisely Target Lymphocyte Subsets
Synopsis
- Engineer delivery technology that can precisely target subsets of different lymphocytes, including specific autoreactive T-cells
- Emphasize how targeting autoreactive T-cells is important in tackling T-cell mediated autoimmune diseases such as Multiple Sclerosis and Type 1 Diabetes
- Outline how strong targeting is important to spare non-target T-cells, and thus enhance therapeutics safety
Preclinical & Clinical Development
Reviewing Delivery Strategies to Facilitate Preclinical & Clinical Success
11:30 am Exploring Methodologies to Characterize Lipid Nanoparticle (LNP) Final Product
Synopsis
- Compare Development Challenges: Understand unique manufacturing and analytical challenges for LNP versus AAV vehicles
- Analytical Methodologies: Explore advanced strategies for comprehensive characterization and quality assessment of LNPs
- Therapeutic Impact: Discuss critical factors to ensure high quality LNPs to improve therapeutic efficacy and patient safety
12:00 pm Utilizing Virus-Like Particles to Efficiently Facilitate In Vivo HSPC Gene Editing
Synopsis
- Underpin the advantages of moving away from ex vivo editing and towards in vivo editing
- Understand the approaches needed to make efficacious edits in vivo using virus-like particles
- Explain how virus-like particles can allow the delivery of a variety of macromolecules and have cell-specific targeting capabilities
12:30 pm Private Lunch with Edilytics
Evaluating Strategies & Approaches Key to Driving Clinical Success in a Time & Cost-Effective Manner
1:30 pm International Considerations: Evaluating the Impact of Obtaining Data from Outside the US
Synopsis
- Explore how obtaining initial data outside the US can expedite your process of moving into a later stage clinical trial in the US
- Reflect on how demonstrating safety and efficacy is crucial to de-risking scaling-up manufacturing and development, and for getting investment for other programs with similar gene editing technology
- Understand how trials outside the US may be essential to find more appropriate patient groups
2:00 pm Roundtable: Reviewing Clinical Strategies to Facilitate Gene Therapies for Rare Disease & Beyond
Synopsis
- Key strategies to optimize clinical trial efficiency to accelerate time and cost-effectiveness
- Adjust approaches to account for population sizes to enhance therapeutic efficacy and safety
- Discuss emerging trends and future directions to optimize clinical strategy
- Leverage gene editing to expand into large indications in oncology and autoimmune diseases through allogeneic CAR-T platforms
2:30 pm Evolution of Clinical Efficacy Endpoints of Lovotibeglogene Autotemcel (Lovo-cel) in HGB-206
Synopsis
- Navigate what strategies constitute clinical success
- Explore which key regulatory interactions helped to build this strategy
- Understand which holdbacks arose in trials and what learnings can be extrapolated from this to improve future programs
3:00 pm Afternoon Break & Poster Session
Translating Learning from Ex Vivo to Turbocharge In Vivo Gene Editing
4:00 pm Discussing Shifts in the Gene Editing Landscape to Transition fromEx Vivo to In Vivo Gene Editing
Synopsis
- Review the benefits of in vivo genome editing approaches from a patient perspective
- Explore the need to refocus the safety guidelines for in vivo treatments compared to ex vivo treatments
- Evaluate what approaches and strategies can be harnessed from ex vivo therapies to enhance in vivo therapy development
4:45 pm Optimizing Clinically Validated Ex Vivo Approaches to be Utilized In Vivo to Enhance Cost-Effectiveness & Efficacy
Synopsis
- Reflect on the advantage of moving to in vivo treatments
- Understand how clinically validated ex vivo approaches can be amended for in vitro contexts
- Explain how transitioning to an in vivo approach makes treatment more economically viable and affordable, and thus more attractive from an investor standpoint