Conference Day Two
8:00 am Check-In & Light Breakfast
7:55 am Chair’s Opening Remarks
Cultivating Academic, Biotech & Pharma Collaborations to Revolutionize Genome Editing Therapeutic Development
8:00 am Panel Discussion: Driving Collaborations to Unlock New Therapeutic Opportunities
Synopsis
- Evaluate partnerships between pharma and biotech companies and the impact academia can have on the development of genome editing therapies
- Discuss the ideal criteria for company collaborations and investment to improve discovery efficiency
- Highlight key strategies from successful collaborations to direct and refine future collaborative efforts
8:30 am Promoting How Gene Editing Has Fast-Tracked Novel Therapies for Complement-Driven Diseases
Synopsis
- Explore the rationale behind developing gene therapy strategies including durability and convenience
- Understand the strategies available to develop gene therapies ranging from AAVmediated gene editing, first to third generation CRISPR gene editing therapies and epigenetic editors
- Emphasize how compared to these other available gene therapy strategies, why base editing technology is important and how base editing strategies have been guided through working in a collaboration with Beam Therapeutics
Evaluating Strategies & Approaches Key to Driving Clinical Success in a Time & Cost-Effective Manner
9:00 am 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
9:30 am 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
10:00am Morning Break & Networking
Reviewing Delivery Strategies to Facilitate Preclinical & Clinical Success
11:00 am Exploring Methodologies to Characterize Lipid Nanoparticle (LNP) Drug 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
11:30 am Highlighting How Designing Compact Epigenome Editors Can Facilitate Clearance for Clinical Trials
Synopsis
- Focus in on the importance of designing gene editors with delivery in mind
- Showcase strategies required to build a compact epigenetic gene editing platform
- Highlight that AAVs are well-studied in clinical trials so provide a good way forward, but a condensed gene editing platform opens the opportunity to use alternative delivery methods
12:00 pm Movement Towards the Clinic: Preclinical Development of Gene Activation Programs with CRISPR-GNDM® Platform
Synopsis
- Demonstrate the efficiency and durability of gene activation by Cas-9 mediated in vivo epigenome editing
- Understand the impact of using a muscle tropic AAV capsid delivery system
- Highlight that despite sustained Cas-9 expression and limited immunogenicity, favorable toxicology data support the therapeutic safety profile
12:30 pm Emphasizing How the TEMPO Platform Presents Several Benefits Over Nuclease-Based Editing Platforms
Synopsis
- Conduct an overview of the mechanism of action of TEMPO
- Showcase the importance of this platform in its ability to control gene function in a tunable and reversible way, and how this subsequently allows the bypassing of technical and clinical barriers
- Highlight key safety and regulatory considerations needed to progress to the pre-clinic and beyond
Evaluating Clinical & Commercial Approaches to Genome Editing
2:00 pm Fireside Chat: Enhancing Investment & Commercialization of Gene Editing Therapies
Synopsis
- Reflect on the current genome editing therapeutics field and key successes and learnings
- Discuss the need to make gene therapies more economical and accessible to a broader number of patients
- Understand strategies and perspectives to increase investment
2:30 pm Applying CRISPR-based Therapeutic Technology to Treat Viral Infectious Diseases
Synopsis
- Explore the advantages of applying gene editing therapies outside of rare disease
- Define how Excision’s CRISPR approach allows for the excision of large sections of viral DNA to eliminate multiple viral genes
- Understand the strategies and approaches which allow for the EBT-101 program to reach clinical trials
3:00 pm Identifying the Role of Clinical Biomarkers in Genome Editing Therapeutics for Sickle Cell Disease
Synopsis
- Biomarkers to support efficacy of gene editing therapies to treat sickle cell disease
- Specialized biomarker discovery to assess disease pathophysiology
- Importance and opportunity biomarkers bring to accelerate clinical development
3:00 pm Afternoon Break & Networking
Thinking Ahead: Reviewing Strategies to Streamline Translatability & Scalability
4:00 pm Innovating Manufacturing Processes to Enhance Quality, Quantity & Scalability
Synopsis
- Explore how the quality threshold needed for in vivo and ex vivo therapies differs and the subsequent early manufacturing considerations that this requires
- Emphasize the importance of characterization of delivery vehicles when manufacturing, to demonstrate efficacy and safety
- Promote the early evaluation of what aspects of the workflow of gene editing therapeutic development will be challenging to scale up to ensure a more time and cost-effective transition to the clinic
4:30 pm Roundtable: Limiting the Immunogenicity & Off-Target Effects of Editing Platforms to Enhance Therapeutic Safety & Efficacy
Synopsis
- Discuss the immunogenicity risks of bacteria-derived genome editing proteins alongside the delivery capsid
- Comprehend design strategies for building a genome editing platform to reduce immunogenicity and off-target effects
- Reinstate how addressing immunogenicity is key to maintaining a platform that is safe and efficacious
5:00 pm Exploring How Novel Nucleases & Appropriate Animal Models Are Key to Clinical Success
Synopsis
- Understand how engineering nucleases to improve on target efficacy and decrease off target effects was crucial to speeding up pre-clinical progression
- Evaluate how selecting mouse models and NHPs provide appropriate models to showcase efficacy and safety
- Reflect on how close collaboration between R&D and pre-clinic, as well as matching gene editing technology to appropriate indications, is essential to cut time and cost of therapeutic development