STEM-CELL DERIVED THERAPIES: FROM SOURCE TO SITE

• Stem-Cell Derived Therapies: From Source To Site

  Stem cells are the backbone of many allogeneic, “off-the-shelf” cell therapies. Watch as industry experts discuss questions and considerations regarding stem-cell-derived therapies

• Starting Materials For iPSCs
• Umbilical Cord Blood As A Regulated Source Of Starting Material
• Donor Traceability And Consent As It Relates To Cord Blood
• How Often Do iPSCs Not Differentiate?
• Considerations When Using Autologous Stem Cells As A Starting Material
• The Importance Of Cryopreservation
• Site Storage And The Last Mile
• The Importance Of Freezing And Stability Studies
• Long-Term Cryopreservation And Storage Of Stem Cells
• The Need For Improved Cryoprotectants
• The Challenges With Shipping Cell Therapy Products
• Are There Genetic Risks To Patients When Using Pooled Donor Manufacturing?
• The Importance Of Potency Assays
• Selecting The Right Vendors When Building Your Supply Chain
• Mitigating Human Error In The Cell Therapy Supply Chain
• The Importance Of Mock Runs, Testing Your Processes
• Intermediate Storage For Cell Therapies
• Bringing CGTs To Middle Income, Developing Countries

HOW TO REDUCE BATCH-TO-BATCH VARIATION IN CELL THERAPY MANUFACTURING

• How To Reduce Batch-to-Batch Variation In Cell Therapy Manufacturing

  As the patient’s immune cells are the starting materials, inherent biological variability leads to CAR-T manufacturing.

• Defining The Root Cause Of Batch-To-Batch Variability
• The Biggest Challenges In Autologous Cell Therapy Manufacturing
• Operations Steps For Reducing Batch-To-Batch Variability
• Understanding Critical Quality Attributes (CQA) Impact On Variability
• The Biggest Challenges In Allogeneic Cell Therapy Manufacturing
• Analytical Challenges Facing Autologous Cell Therapies
• Reducing Variability For Apheresis Material For Autologous Cell Therapy
• Improving Data Collection And Tracking To Reduce Batch-to-Batch Variability
• Best Practices For Reducing Variability Without Increasing Regulatory Burden
• How Automation Reduces Batch-To-Batch Variability
• How To Reduce Batch-To-Batch Variation In Cell Therapy Manufacturing | Audience Q&A

2024 REGULATORY OUTLOOK WITH FDA'S DRS. PETER MARKS AND NICOLE VERDUN

• 2024 Regulatory Outlook With FDA's Drs. Peter Marks And Nicole Verdun

  Explore the evolving regulatory landscape for cell and gene therapies in 2024 including the increasing speed availability of cell and gene therapies by enabling manufacturing processes and standards development.

• What's New for 2024: Dr. Nicole Verdun Offers a Look Inside The Office Of Therapeutic Products
• How Past Gene Therapy Approvals Are Shaping the Future with Dr. Peter Marks
• Focal Points for 2024: Dr. Verdun and Dr. Marks Talk Regulatory Challenges for Cell-Based Therapies
• Race To The Regulatory Finish Line: Dr. Nicole Verdun Talks Efficiencies In Platform Technologies
• Why Clinical Requirements Change: Dr. Peter Marks on Pre-IND Meeting Pathways
• Dr. Peter Marks Talks a Standardized Playbook for Gene Therapy Manufacturing
• Patient Data’s Impact On CMC Robustness
• CMC Comparability: Transitioning to Commercial Manufacturing with Dr. Nicole Verdun
• The Need for Speed: Dr. Peter Marks and Dr. Nicole Verdun Explain Meeting Unmet Need for Pediatric Patients
• Dr. Peter Marks on the Adoption of Novel Precision Analytics
• Understanding AI And Machine Learning's Impact On CGT with Dr. Peter Marks and Dr. Nicole Verdun
• Collaborating with the FDA: Dr. Peter Marks and Dr. Nicole Verdun Share Advice For Small And Emerging Biotechs
• 2024 Regulatory Outlook With Dr. Peter Marks And Dr. Nicole Verdun | Audience Q&A

CONSIDERATIONS FOR INVESTING IN YOUR GENE THERAPY’S AUTOMATION FRAMEWORK

• Considerations For Investing In Your Gene Therapy’s Automation Framework

  Deciding which technology to incorporate into your biotech’s operations, such as automation, machine learning, or AI, requires consideration into what's scalable and relevant to your goals.

• Advancements In Automation And Computational Technology
• How AI And ML Can Support Automation
• Defining Automation Framework
• When To Start Thinking About The Use Of Automation
• The Inherent Risks Of Adopting New Technologies
• Regulatory Concerns Surrounding Automation
• The Cost Of Building An Automation Framework
• What To Look For In A Technology Vendor
• How Well-Supported Is The Current Technology Vendor Space?
• Embracing Automation And Addressing The Barriers To Adoption

MRNA FOR CANCER IMMUNOTHERAPY 2024 OUTLOOK

• mRNA For Cancer Immunotherapy: 2024 Outlook

  As additional mRNA-based cancer immunotherapies vaccines enter clinical development, operational and supply chain challenges must be addressed to reduce turnaround times and COGS.

• Genentech, Myeloid’s Approaches To mRNA Technologies
• Major Advancements In mRNA
• Achieving Stability For mRNA
• Bottlenecks And Manufacturing Challenges
• Supply Chain And Operational Challenges Facing mRNA
• mRNA For Cancer Immunotherapy 2024 Outlook | Audience Q&A

ARE WE THERE YET? ROADMAP(S) TO HIGHER QUALITY AAV

• Are We There Yet? Roadmap(s) To Higher Quality AAV

  Current limitations in AAV gene therapy manufacturing platforms often leave us making difficult tradeoffs. To learn how companies are evaluating and striving to overcome these tradeoffs in their technology and outsourcing partner selections, facility designs, and regulatory preparations, tune into this archived episode of Cell & Gene Live.

• What’s Keeping The AAV Space Up At Night?
• AAV Critical Quality Attributes: Advances In Analytical Characterization
• For The Love Of Mass Photometry: The Most Notable AAV Analytical Technology Advancements
• Beyond Full-Empty Capsids: Additional AAV Quality Concerns & Questions To Address
• "Space Invaders," Expression Systems, & Producer Cell Lines: AAV Upstream Process Development Innovations
• Greatest Advances & Unmet Needs In AAV Downstream Process Development
• AAV Outsourcing: Identifying A “High-Quality” CDMO Partner
• Where Are The Greatest Innovations Yet To Come For The AAV Field?

OPTIMIZING YOUR COLD CHAIN FOR CELL THERAPIES

• Optimizing Your Cold Chain For Cell Therapies

  Cell & Gene’s Chief Editor Erin Harris and expert panelists discuss practical ways to improve shipping systems, tracking, product monitoring, and more for both just-in-time delivery and cryogenic preservation.

• An Inside Look At Umoja Biopharma And Bristol Myers Squibb's Cold Chains
• Overcoming Cryopreservation Challenges
• Risk Mitigation Strategies
• Regulatory Considerations For An Optimized Cold Chain
• Inventory Management, Staff Training, And Timeline Optimization
• Courier Optimization
• Breaking Down Shipping
• Optimizing Your Cold Chain For Cell Therapies | Audience Questions

STABILITY TO SCALABILITY: THE FUTURE OF VIRAL VECTOR THERAPIES

• Stability To Scalability: The Future Of Viral Vector Therapies

  Despite advances that have contributed to improvements in viral vector yields, innovation is still needed in the CGT sector to impact product quality and accelerate scale for viral vector production.

• Quality Control Assays
• Manufacturing Hurdles With Viral Vectors
• Regulatory Compliance And Viral Vectors
• Market Demand For AAV Vectors
• Overcoming Capacity Constraints
• Scaling Viral Vectors
• Stability To Scalability: The Future Of Viral Vector Therapies | Audience Q&A

REGULATING THE CGT WORKFLOW TO IMPROVE PATIENT SAFETY

• Regulating The CGT Workflow To Improve Patient Safety

  As regulatory guidelines evolve, and the need to meet accelerated timelines increases, so too does the need for an efficient manufacturing workflow.

• Regulatory Considerations For Scale Up
• Late-Stage Comparability Of The Manufacturing Process
• Partnerships With CDMOs
• Policy Development
• Real-World Evidence And Registries
• COAs And DHTs And Post-Approval Monitoring
• Class-Specific Considerations
• Regulating The CGT Workflow To Improve Patient Safety: Audience Q&A

DRIVING DOWN COGS: AUTOMATION'S ROLE IN CELL THERAPY MANUFACTURING

• Driving Down COGS: Automation's Role In Cell Therapy Manufacturing

  Process automation, task automation, and test automation have become crucial components of cell therapy manufacturing. Improve your cell therapy manufacturing costs and scalability via standardization and automation.

• Breaking Down Automated Manufacturing
• Technology Needs For Automated Manufacturing
• Mitigating Risk In Process Automation
• Pipeline Progress
• Auto, Allo, And AI
• Safety And Efficiency Of Source Material Collection
• Driving Down COGS Audience Q&A

MAJOR TRENDS IN NEXT-GEN RNA ENGINEERING

• Major Trends In Next-Gen RNA Engineering

  From tissue specificity to potency and durability, major trends in RNA are developing as the space continues to evolve at record-breaking speed. In June 2023, Cell & Gene Live welcomed Nathaniel Wang, CEO and Co-Founder, Replicate Bioscience and Sam Deutch, EVP of Research and Early Development at Nutcracker Therapeutics for a deep dive into how RNA can accelerate drug development and how the evolution in the scale-up production of novel engineering technologies impacts near- and long-term deployment of new therapies.

• Tissue Specificity and Potency & Durability
• Overcoming Deployment Challenges
• “Plug and Play” Technologies: Lessons for RNA Technologies
• Major Trends In Next-Gen RNA Engineering: Audience Q&A

BUILDING AN IN-HOUSE CELL & GENE MANUFACTURING FACILITY BEST PRACTICES LESSONS LEARNED

• Building An In-House Cell & Gene Manufacturing Facility: Best Practices & Lessons Learned

  Listen in on a panel discussion where cell and gene therapy experts share their experiences in building state-of-the-art manufacturing facilities and offering detailed advice on how your company can do the same.

• The Why Behind Build In “Build Versus Buy”
• Cost Versus Labor Model
• Timelines And Milestones
• Building The Team
• Speed To Clinic
• The Role Of Automation In Manufacturing
• Audience Q&A

SCALING CELL THERAPIES PART 2: ESTABLISHING COMPARABILITY IN MANUFACTURING

• Scaling Cell Therapies Part 2: Establishing Comparability In Manufacturing

  Michael Blackton, SVP, Cell Therapy Manufacturing, DeTIL Development at Nurix Therapeutics and John Lee, SVP, Cell Therapy Manufacturing at Center for Breakthrough Medicines are back with Cell & Gene Live host Erin Harris for Part 2 of our Scaling Cell Therapies series. Check out the conversation as these experts focused on comparability - how establishing manufacturing, characterization, and controls can positively affect scalability. Available on-demand thanks to the support of Corning Life Sciences.

• Challenges Associated With Comparability For Cell-Based Products
• Increased Comparability Burden During Clinical Trials
• How The Process Influences The Product
• Donor-To-Donor Variability
• Design And Execution Of Comparability Studies
• Audience Q&A

TACKLING CELL & GENE THERAPIES’ TOP 3 REGULATORY IMPEDIMENTS

• Tackling Cell And Gene Therapies’ Top 3 Regulatory Impediments

  Listen in to this expert discussion on problems in the cell and gene therapy industry, specific FDA initiatives to overcome these, broader regulatory trends, and more.

• CGT’s Main Opportunities To Meet Regulatory Requirements
• Challenge #1: The Changing Regulatory Landscape
• Challenge #2: Rare Or Ultra-Rare Study Design Considerations And Use Of Existing Data
• Challenge #3: Lack Of Animal Models
• Other CGT Regulatory Challenges
• Tackling Cell And Gene Therapies' Top 3 Regulatory Impediments: Audience Q&A

EVALUATING TRANSLATIONAL RESEARCH TOOLS FOR CGTS

• Evaluating Translational Research Tools For CGTs

  Translational research tools, including biological assays (bioassays) and biological markers (biomarkers), are needed to bridge the gap from understanding the disease process to identifying therapeutic targets and testing potential therapies in preparation for clinical research in humans.

  On November 15, 2022 Gregory Opiteck, Ph.D., VP Head of Translational Sciences at Allogene Therapeutics and Douglas Sanders, Ph.D., VP Head of Translational Medicine at AVROBIO joined Cell & Gene Chief Editor Erin Harris to cover assay and vendor selection in support of allogenic CAR T therapy as well as the various modalities of AAV gene therapy.

• Defining Translational Research
• Challenges with Bioassays
• Informative Biomarkers to Drug Development
• The Importance of Vendor Selection
• Strategies for Development of Next-Gen AAVs
• AAV & Ex Vivo Gene Lentiviral Therapy
• New Tools for Translational Research
• Questions from the Cell & Gene Live Audience

SCALING CELL THERAPIES: AUTO VS. ALLO

• Scaling Cell Therapies: Auto vs. Allo

  To achieve commercial success, a developed process must be scalable and suitable for a manufacturing environment. The scaling strategy depends on the type of cell therapy.

• The Critical Differences Between Scale Up And Scale Out
• The Importance Of The Starting Materials And Donor Cells
• But What About Aseptic Processing?
• Single-Use Supply Chain Challenges & Opportunities

ADVANCEMENTS IN TARGETED MRNA DELIVERY

• Advancements In Targeted mRNA Delivery

  mRNA has broad potential as a therapeutic. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems.

• The Not-So-New Challenges Of mRNA Delivery
• The Cell As The Delivery Vehicle
• Innovations In mRNA Delivery
• The Wider Use Of mRNA To Treat Various Forms Of Cancer & Autoimmune Diseases
• Cell & Gene Live Audience Q&A

WIN THE CGT TALENT FIGHT: HOW TO FIND AND RETAIN EXPERTS

• Win The CGT Talent Fight: How To Find And Retain Experts

  The cell and gene therapy (CGT) market is expected to be worth $5.5 Billion by 2026. Yet, the sector struggles with recruiting and retaining enough experienced candidates needed to bring life-saving treatments to market. Why is that, and what can you do now to overcome the talent crunch and keep skilled professionals in highly sought-after roles? Now available On-Demand, Rosie Walker, Director of ARM’s GROW RegenMed Internship Program, Dr. Andrew M. Scharenberg, co-founder and CEO of Umoja Biopharma, Juliette Hilliard, Director of Talent Acquisition at Novartis Gene Therapies, and Dr. Ray Tabibiazar, Chairman and CEO at SalioGen Therapeutics joined Cell and Gene Live host Erin Harris to share their experiences as well as the specific takeaways that you, too, can use to source and retain the skilled experts you need to reach commercialization.

KITE PHARMA'S ULTIMATE SUPPLY CHAIN TEST

• Kite Pharma's Ultimate Supply Chain Test - A Q&A With Their 4-Exec Panel On Overcoming The Pandemic's Disruption

  Throughout the pandemic, Kite Pharma overcame constantly disrupted supply chains, including limited flights and border closings, all while ensuring that daily shipments of live cells remained on schedule and that the critical needs of people with cancer were met. Four of Kite's supply chain executives share their lessons learned and how those learnings will continue to evolve and shape their supply chain operations going forward.

THE THERAPEUTIC POTENTIAL OF EXOSOMES

• The Therapeutic Potential Of Exosomes

  In this Cell & Gene Live, Dr. Maria Mitrani, Co-Founder and Chief Scientific Officer at Organicell; Benny Sorensen, Scientific Advisory Board member at Codiak BioScience; and Nicolas Rousseau, Co-Founder and COO at EVerZom talk to Chief Editor Erin Harris about the sector’s understanding of extracellular vesicles (EV). Specifically, they cover the regulatory landscape for exosomes as well as appropriate characterization models and advancements in manufacturing needed to ensure scalability. The expert panelists also provided detailed responses to audience questions submitted during the live event. 

• What Is The Therapeutic Potential Of Exosomes?
• The Regulatory Pathway & The Road to FDA Approval
• Understanding Appropriate Characterization Methods
• Advancements In Manufacturing
• Exosomes And Scalability Challenges
• Audience Q&A

TACKLING THE CELL & GENE MANUFACTURING TALENT CRUNCH

• Tackling The Cell & Gene Manufacturing Talent Crunch

  Join Project Farma and Precision ADVANCE leaders Anshul Mangal and John Khoury for a discussion with BioProcess Online Chief Editor Matt Pillar to address solutions to the biotherapeutic and cell and gene therapy process and manufacturing talent gap.

CAN INNOVATION DRIVE DOWN CELL THERAPY MANUFACTURING COSTS?

• Can Innovation Drive Down Cell Therapy Manufacturing Costs?

  The high cost of producing new cell and gene therapies is one of the largest issues facing the industry. After launching Kymriah and Yescarta, Novartis’ price tags were $475,000 and $375,000, respectively. Advancements in manufacturing are driving costs down, but not at the required pace.

• The Why Behind The Expense Of Cell Therapy Manufacturing
• The Current Challenges Facing Cell Therapy Manufacturing
• Cell Therapy Manufacturing And Economies Of Scale
• How Automation Drives Down Cell Therapy Manufacturing Costs
• True Innovations In Driving Down Cell Therapy Manufacturing

THE FUTURE OF CRISPR-ENGINEERED STEM CELL THERAPIES

• The Future of CRISPR-Engineered Stem Cell Therapies

  To learn more about CRISPR gene editing, Cell and Gene Chief Editor, Erin Harris, sat down with Sadik Kassim, Chief Technical Officer at Vor Biopharma to discuss the benefits of CRISPR-engineered stem cell therapies, the challenges and feasible solutions, what’s next, and more.

• The History of CRISPR Gene Edited Therapies
• The Future of CRISPR-Engineered Stem Cell Therapies: 3 Clinical Trial Examples
• The Ethical Implications Of Editing A Genome
• The Challenges Associated With CRISPR Gene Editing
• The Patient Perspective Of CRISPR-Engineered Stem Cell Therapies
• The Future of CRISPR-Engineered Stem Cell Therapies: Adverse Effects In Clinical Trials
• The Future of CRISPR-Engineered Stem Cell Therapies: Technology & Platforms
• The Permanency Of Gene Therapy
• The Future of CRISPR-Engineered Stem Cell Therapies: Digestive Disease Studies
• The Misconceptions Of CRISPR Gene Edited Stem Cell Therapies
• Will CRISPR Change Modern Medicine?
• The Future of CRISPR-Engineered Stem Cell Therapies: Outsourcing vs. In-House
• What The Gene Therapy Sector Doesn't Understand About CRISPR?
• The Future of CRISPR-Engineered Stem Cell Therapies: Virtual Event Audience Q&A