ENHANCING SPEED & QUALITY IN CELL & GENE THERAPY MANUFACTURING FOR THE DACH REGION

Commercializing Cell & Gene Therapies in global markets

• Navigating global regulatory landscapes for successful CGT commercialization across diverse markets

• Developing scalable manufacturing and distribution strategies to meet global demand

• Building strategic partnerships to enhance market access and accelerate therapy adoption worldwide

Considerations for the development and commercialization of autologous CAR-T cell products

• Balancing personalized production with cost-effective scaling for broader patient access

• Ensuring efficient vein-to-vein delivery while maintaining product quality & regulatory compliance

• Overcoming challenges related to pricing, reimbursement, for commercial success

Leadership Panel Discussion: How to achieve convergence of viral vector manufacturing technologies towards robust platform processes?

• Streamlining viral vector production through standardized platform technologies

• Enhancing scalability and efficiency in viral vector manufacturing

• Overcoming challenges in process consistency and regulatory compliance

Synthetic biology for forward cell reprogramming

• Understanding synthetic gene circuits to precisely control cell fate transitions and reprogram cells into desired types.

• Scalable production of reprogrammed cells for regenerative medicine and personalized therapies.

• Simplifying the reprogramming process by integrating synthetic biology tools for more efficient, targeted cell conversions.

Optimizing Upstream Processes for Enhanced Cell Quality and Viability in Dendritic Cell-Based Allogeneic AML Immunotherapy

• Dendritic cells derived from leukemic progenitors hold promise for novel cancer immunotherapies.

• Mendus has developed DCOne, a proprietary platform using a unique cell line to produce off-the-shelf leukemic-derived dendritic cells.

• Our lead program, vididencel, delivered positive Phase 2 results as a maintenance therapy for AML.

Industrialization of a novel vector for gene therapy – Arenavirus

• Developing efficient processes for large-scale production of Arenavirus vectors

• Ensuring adherence to regulatory standards for clinical and commercial applications

• Expanding the use of Arenavirus vectors in various gene therapy treatments

Advancements in Viral Vector Design for Repeated Administration in Chronic and Intractable Diseases

• Curigin’s innovative approach involves genetically modifying the capsid surface of viral vectors to prevent exposure to antibodies, allowing for repeated administration in patients with existing antibodies.

• This breakthrough technology enables effective and continuous treatment administration, crucial for achieving therapeutic outcomes in chronic and intractable diseases.

The CMC Challenges of the Downstream Process Development and Scale Up

• Novel engineered Fc-fusion proteins for cell therapy.

• The CMC complexities of cell therapy product characterization.

• Addressing the hurdles in scaling up cell therapy manufacturing, from batch-to-batch consistency to the application of automated systems.

A Platform Technology for Downstream Reprogramming of Cell Fate

• Introducing a platform to identify lineage control switches that enhance downstream cell differentiation processes.

• Understanding how these switches optimize cell quality, stability, and functionality during reprogramming.

• Exploring applications for improving cell-based products in regenerative and personalized medicine.

Innovative Adsorptive Separation Strategies for Viral Gene Therapy Vectors

• Exploring advanced methods to enhance the purity and yield of viral gene therapy vectors in downstream processing.

• Optimizing adsorptive separation techniques to improve efficiency and scalability in the isolation of viral vectors.

• Discussing the impact of these innovations on the delivery and efficacy of therapeutic genes, advancing treatment outcomes.

Twin-Column Continuous Chromatography for Improving Viral Vector based Gene Therapies

• Enhancing purification efficiency for viral vectors in gene therapies through twin-column continuous chromatography.

• Reducing production costs and time by streamlining the purification process.

• Improving product yield and consistency for large-scale viral vector manufacturing.

Industrialization of cell therapies: Platform Approaches

• Standardizing production processes to enhance scalability and reduce costs

• Leveraging automation and data analytics to optimize efficiency and maintain quality.

• Developing adaptable platforms that support multiple cell therapy types for faster development and commercialization

Streamlined GMP Manufacturing and Innovative Tracking System for TCR-T Cell Therapy

• Streamlined TCR-T therapy production with an automated 6-day GMP process.

• Optimized 3rd generation TCR-T therapy for treating multiple solid tumor indications

• UniTope & TraCR: An innovative and universal tool for tagging and tracking of TCR-T cells

Strategies for Intensification: Achieving Continuous Processing

• Strategies for Achieving Continuous Processing Intensification

• Methods to Enhance Continuous Processing

• Intensification Techniques for Seamless Operations

Programmable and auto-regulated viral vectors for new applications of gene therapy

• Viral vectors are evolving to offer more precise gene delivery for treating genetic disorders, with greater control over gene expression.

• New advancements allow vectors to be auto-regulated in response to physiological cues, ensuring more targeted gene expression.

• These programmable, vector-agnostic systems increase safety and on-target efficacy, expanding potential therapeutic applications.

Navigating CMC and Regulatory Hurdles for CAR T Cell Therapy: Strategies for Post-Approval Changes in Autologous Cell Therapies

• Key considerations for maintaining product consistency and safety in CAR T therapies.

• Navigating evolving standards and ensuring alignment with regulatory expectations.

• Best practices for implementing post-approval changes without compromising quality or efficacy.

Patient-Centric Manufacturing and Safety

• Ensuring patient safety throughout the manufacturing and delivery of cell and gene therapies.

• Integrating patient safety science into manufacturing practices to enhance product safety.

• Addressing challenges and solutions in maintaining patient-centric approaches during production.

Advancing Red Blood Cell Production from Stem Cells: Enhancing Yield and Quality

• Developed a high-yield method to produce red blood cells from CD34+ stem cells.

• Achieved longer survival and superior functionality of ex vivo generated RBCs compared to standard RBCs.

• Potential to reduce transfusions for patients with blood disorders like β-thalassemia and sickle cell disease

Adopting a Risk-Based Approach to CGT Production for Enhanced Quality and Compliance

• Implement risk assessment tools to identify and mitigate potential hazards in CGT production.

• Optimize manufacturing processes by prioritizing critical control points for product safety and efficacy.

• Align production strategies with regulatory requirements, ensuring continuous risk management throughout the product life cycle.

Efficiency and Scalability in CRISPR Manufacturing

• Addressing the challenges of scaling up CRISPR-based therapies.

• Automation and standardized production processes to meet clinical and commercial demand.

• Case Studies on CRISPR manufacturing through efficient gene modification.

Overcoming Bottlenecks in CRISPR Manufacturing for Gene Therapy

• Recent innovations and improvements in CRISPR technology and manufacturing processes.

• Practical strategies and best practices for optimizing the workflow.

Advancing Regulatory T Cell Therapies: Applications & Innovations in Cell Therapy

• Breakthroughs in enhancing the efficacy and specificity of Tregs in cell-based therapies.

• Ongoing clinical trials and real-world applications for treating autoimmune and inflammatory diseases.

• Exploring new indications and the broader impact of Treg cell therapies in immunotherapy

Advancing Autologous Cell Therapies for Muscle Regeneration

• Exploring cutting-edge techniques in developing and optimizing autologous cell-based therapies for muscle diseases..

• Addressing common challenges in the development and implementation of autologous cell therapies, including scalability and regulatory considerations.

Manufacturing Stromal Cell Therapeutics for Type 1 Diabetes

• Streamlining the production of stromal cell-based therapeutics aimed at treating and potentially curing type 1 diabetes.

• Key breakthroughs in the scalable manufacturing of stromal cell therapies for diabetic patients.

• Ensuring quality and consistency in stromal cell manufacturing to improve patient outcomes and disease management.

Gene Therapy Manufacturing in the AI era

• Current landscape of AI used in gene therapy manufacturing.

• Challenges and enablers in adoption.

• Standardizing genome-wide off-target prediction methods.

Development of Digitalization and Automation in Viral and Non-Viral Vector Production.

• Recent advancements in automation technologies specifically designed for vector production.

• Use of robotic platforms and automated assays to rapidly test and refine vector designs.

• Strategies to reduce the potential for errors during the formulation process.

Scaling Out vs. Scaling Up: Best Practices for CAR-T Cell Therapy Production

• Expanding capacity by replicating smaller production units to meet demand.

• Increasing the size and efficiency of existing production processes for higher output.

• Balancing cost, efficiency, and quality while scaling CAR-T cell therapy production.

Rapid Analytical Methods for Real-Time CAR-T Product Release  

• Deploying technologies for immediate assessment of CAR-T product quality.

• Rapid assays to ensure potency and purity without delays.

• Streamlining quality control with automation for consistent real-time results.

Innovations in Lipid Nanoparticles for Gene Delivery  

• Latest advancements in lipid nanoparticle formulations.

• Improvements in lipid composition, surface modifications for enhanced cellular uptake.

• Strategies for targeted delivery to specific tissues or cells in precision therapy.

Clinical Applications and Future Directions of Non-Viral Vectors in Gene Delivery  

• Examination of recent clinical applications of non-viral gene delivery methods.

• Discussion on vector design and techniques.

• Future trends and potential innovations that could further advance the field.

CMC Challenges in Gene Therapy Development: Adding Sustainability to a Complex Equation – A Biotech Case Study

• Addressing CMC complexities while integrating sustainable practices.

• Reducing waste and energy consumption in gene therapy production.

• Real-world lessons from a biotech company’s sustainable approach.

Sustainable Practices in Viral Vector Manufacturing

• Advances in waste minimization and recycling in vector production.

• Low-impact methods for scaling up viral vector manufacturing.

• Integration of sustainability metrics in production planning.