Conference Day One
Wednesday, March 26
8:00 am Registration & Coffee
8:25 am Chair’s Opening Remarks
PIONEERING THE FUTURE OF GENE THERAPY: UNLEASHING INNOVATION, OVERCOMING ROADBLOCKS & ACCELERATING MARKET ACCESS IN 2025
9:00 am Navigating New Horizons: Gene Therapy Breakthroughs & Challenges in the Rare Disease Landscape
Synopsis
• What emerging innovations does 2025 hold for rare disease treatment?
• What lessons have we learned from 2024 and how can we overcome these roadblocks in 2025 for gene therapy development?
• Understanding strategic collaborations by leveraging partnerships for accelerated progress
9:30 am A Simplified, Scalable AAV Manufacturing using a Process Intensified Adherent Platform
Synopsis
· Adopting process intensification to streamline operational efficiency and minimize complexity, leading to reduced manufacturing and compliance risks
· Insights on utilizing small footprint manufacturing combined with high-efficiency cell lines, cost-effective transfection techniques resulting in consistent high-yield viral vectors with minimal impurities to lower costs of goods (COGs)
10:00 am Regulatory Case Study: The Role of Clinical Biomarkers in the Development of Gene Therapies
Synopsis
• Biomarkers relevant to disease progression that support the differentiation of gene therapies
• Biomarkers as key endpoints for accelerated approval of gene therapies
• Innovative approaches in biomarker discovery for gene therapies
10:30 am Panel Discussion: Starting with the End in Mind – Patients & Payers, Building Trust in Gene Therapy
Synopsis
• Addressing concerns around the efficacy, safety, and cost of gene therapies among patients and payers
• Best practices for demonstrating the long-term value of gene therapies through outcome-based data and real-world evidence
• Patient advocacy groups can easily organize patients and provide opportunities for education
• Effective messaging and educational initiatives to ensure buy-in from patients and payers
11:00 am Morning Break & Speed Networking
Translation
Clinical
Manufacturing
Novel Platforms Beyond AAV:
Using Viral & Non-Viral Delivery for In
Vivo Gene Therapy
Moderator: Richard Wilson, Senior Vice President, Primary Focus Lead of Genetic Regulation, Astellas Pharma
Optimizing Clinical Trials for Gene Therapies Through Immunogenicity & Natural History Data
Moderator: Daniel Chung, Chief Medical Officer, SparingVision
12:00 pm Extending the Reach of Genetic Medicine Through Next-Generation Parvovirus Vector Technology
Synopsis
- A comparison of LNPs and AAVs reveals key differences in immunogenicity and toxicity, suggesting potential benefits of combined use
- Improved targeting and uptake strategies aim to mitigate immune responses and toxicity in metabolic liver diseases
- Overcoming delivery challenges, for LNPs to be optimized for more effective transport of therapies to liver cells
12:30 pm Addressing AAV Limitations with Ligand- Capsid Bioconjugation
Synopsis
- How the ligand-capsid bioconjugation works: chemistry and process
- Examples of ligands improving AAV characteristics
- Future applications of AAV bioconjugation
12:00 pm Machine-guided design and optimization of AAV gene therapies
Synopsis
- Using machine learning to solve gene delivery challenges to the eye, muscle, and CNS
- Demonstrating the capabilities of next-gen AAV vectors in NHP studies
- Emerging opportunities applying AI to general purpose multi-property optimization for in vivo effectiveness
12:30 pm Case Study: Building Natural History Data for Rare Disorders to Improve Existing Strategies for Gene Therapy Development
Synopsis
- Leveraging patient registries, electronic health records (EHRs), and patient-reported outcomes to gather natural history data quickly and cost-effectively
- Using natural history data to define clinical endpoints, establish baseline variability, and select appropriate patient populations for trials
- Identifying and validating surrogate endpoints that can predict clinical benefit, facilitate regulatory interactions, and enable faster market access
12:00 pm Next-Gen Cytogenetic Genomic Integrity Assays for C> Development
Synopsis
- KROMATID’s single cell cytogenetic platform provides advanced characterization and data analysis of genomic integrity for gene modified cell therapies
- Nationwide Children’s CARNK novel allogeneic therapy utilized KROMATID’s platform to assist in characterizing the safety profile of their therapy for hematologic malignancies
- Cytogenetic testing provides critical orthogonal data for characterization of whole genome assessment of transgene integration and on-and-off target genotoxicity
12:30 pm Towards Non-Viral DNA Based Gene Writing Therapy
Synopsis
- An overview of Full Circles’ non-viral gene writing platform technology
- Unpacking the successes in ex vivo gene editing with C4DNATM
- Unlocking non-viral adoptive cell therapy applications with C4DNATM mediated precise gene writing
1:00 pm Next-Gen Bioprocessing: IsoTag™ Affinity Reagents for LV and AAV Purification
Synopsis
- Novel affinity technology for complex biologics purification
- Cost competitive solution that consolidates unit operations
- Pursuing collaboration opportunities with clinically relevant therapeutics
1:10 pm Lunch & Networking
Pioneering Precision in Gene Editing by Advancing Platforms for Targeted & Scalable Therapeutic Solutions
Moderator: Richard Wilson, Senior Vie President, Primary Focus Lead of Genetic Regulation, Astellas Pharma
Innovations in Capsid Engineering for More Efficacious Therapies
Moderator: Daniel Chung, Chief Medical Officer, SparingVision
Optimizing CMC Pathways for Seamless Transition from Early Development to Commercial Readiness
Moderator: Toni Guiriba, Associate Director, Corporate Strategy, REGENXBIO
2:15 pm Starting with the End in Mind: Community Engagement Throughout the Drug Development Cycle
Synopsis
- Explore approaches to effectively engaging communities early in development
- Develop strategies for addressing challenges at each stage of development
2:45 pm Developing Gene Editing Therapies That Utilize Obsidian’s cytoDRiVE™ Platform to Provide Regulated & Tunable Expression of Proteins
Synopsis
- Modulating the activity of gene editing tools like CRISPR-Cas9 to increase the specificity and reduce off-target effects
- Determining the best small-molecule regulators to use with the platform and validating the system in various preclinical models
- Demonstrating proof of concept for the ability to modulate gene editing activities in a controlled and precise manner
2:15 pm Early AAV Manufacturing Strategic Decision for Phase 1&2: Alternatives Scenario & Strategic Consideration for Readiness to Phase 3 Commercial
Synopsis
- Strategies for incorporating market needs, pricing, and reimbursement considerations into early development stages
- What to consider for defining an optimal process approach for phase ½ while integrating phase 3 perspectives, from IP to COGs assumptions
- Cost and timelines under investor's objectives and competition and potential future-focused partners
- Common pitfalls and solutions from CDMO for ensuring a successful long-term vision to market launch
2:45 pm Streamlining Licensed Gene Therapy Integration: Abeona Therapeutics’ Approach to CMC Adaptations & Regulatory Success
Synopsis
- Explore Abeona’s strategies for modifying the CMC aspects of a licensed gene therapy to align with their proprietary manufacturing practices, ensuring consistency and efficiency
- Gain insights into Abeona’s approach to maintaining therapeutic integrity while implementing necessary process adjustments, including raw material sourcing, quality testing, and scalability
- Understand the regulatory landscape for manufacturing-modified licensed therapies, with lessons from Abeona’s experiences in compliance, submission requirements, and securing timely approvals
3:00 pm Afternoon Refreshments & Networking
NAVIGATING THE GENE THERAPY LANDSCAPE: MARKET STRATEGIES & BUILDING TRUST
4:00 pm Panel Discussion: Optimizing Gene Therapy Programs for Patient Communities of Any Size
Synopsis
• Multi-stakeholder discussion of friction points in the development lifecycle and how to overcome them: what have we tried, what has been successful, and under what circumstances
• Dissection of real-world examples over time and across different patient communities
• Analysis of recent trends and their impacts on key strategies and tactics
4:30 pm Panel Discussion: Navigating Global Regulatory Challenges in Cell & Gene Therapy
Synopsis
• Diverging Regulatory Pathways: Understanding key differences in regulatory requirements between the FDA, EMA, and other global regulators in cell and gene therapy
• Exploring ongoing initiatives for global regulatory convergence and the impact on innovation and patient access
• Perspectives from regulators in the U.S., EU, Asia, and other countries on navigating local challenges and fostering international collaboration
5:00 pm Next-Generation TEM Method for Quality Control of AAVs and Other Nanoparticles
Synopsis
- QuTEM’s latest AAV method offers an automated, robust, and objective approach for vector content assessment, determining %Filled, %Partially Filled, %Overfilled, and %Empty capsids, as well as genome integrity (in kilobases) for each subpopulation
- This platform-validated method provides unique insights into AAV transgene incorporation at different quality level (R&D, Non-GMP, and GMP)
- QuTEM delivers comprehensive TEM-based nanoparticle characterization, enabling quality monitoring throughout the entire manufacturing process
5:30 pm An Overview of the Gene Therapy Landscape
Synopsis
- Diseases of interest: preclinical vs clinical
- Immunogenicity and the rise of non-viral vectors
- An overview of the financial landscape, focusing on enabling technology deals