TACKLING CELL & GENE THERAPIES’ TOP 3 REGULATORY IMPEDIMENTS

• Tackling Cell And Gene Therapies’ Top 3 Regulatory Impediments

  Cell & Gene Live's first event of 2023 welcomed Dr. Adora Ndu, Chief Regulatory Affairs Officer at BridgeBio and Dr. Nina Hunter, Vice President, Corporate Strategy at REGENXBIO joining host Erin Harris to discuss the top three regulatory issues stymying the cell and gene therapy sector. Despite the promise of cell and gene therapies, the regulations governing them seem to lag, hindering the clinical translation of these novel medicines. During the hour, they discussed problems in the 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
• 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