WEBINARS AND VIDEOS

• What Is The DynaChrom™ Single-Use Chromatography System?

  This chromatography system offers a flexible solution, paired with an advanced software platform, allowing easy integration with downstream process automation.

• 2024 Regulatory Outlook With Dr. Peter Marks And Dr. Nicole Verdun | Audience Q&A

  Experts on the Cell & Gene Live event, 2024 Regulatory Outlook, answer questions about topics including pediatric development in rare diseases, diversity planning for ultra-rare disease, anticipated approvals by 2025, and more.

• Collaborating with the FDA: Dr. Peter Marks and Dr. Nicole Verdun Share Advice For Small And Emerging Biotechs

  In this segment, Dr. Peter Marks and Dr. Nicole Verdun share their advice and best practices for small and emerging biotechs when working with the FDA.

• Understanding AI And Machine Learning's Impact On CGT with Dr. Peter Marks and Dr. Nicole Verdun

  During this segment, Dr. Peter Marks and Dr. Nicole Verdun share their take on how AI and machine learning will impact FDA regulation in 2024.

• The Need for Speed: Dr. Peter Marks and Dr. Nicole Verdun Explain Meeting Unmet Need for Pediatric Patients

  How can sponsor companies accelerate development in pediatric patients for genetic diseases that affect both children and adults, but where gene therapy holds the greatest promise to address unmet needs in pediatric patients?

• Dr. Peter Marks on the Adoption of Novel Precision Analytics

  Hear advice for developers who are worried that the FDA will not accept changes in their process that include a new analytical method.

• CMC Comparability: Transitioning to Commercial Manufacturing with Dr. Nicole Verdun

  In this segment, Dr. Nicole Verdun shares some of the challenges demonstrating CMC comparability during late-stage product development (i.e., pivotal or Phase 3 clinical trial) that occur while transitioning to commercial manufacturing.

• Patient Data’s Impact On CMC Robustness

  When CGT products demonstrate dramatic improvements with minimal safety concerns in a Phase 2 study, sponsors may want to modify the protocol mid-study to increase patient enrollment.

• Dr. Peter Marks Talks a Standardized Playbook for Gene Therapy Manufacturing

  In this segment, Dr. Peter Marks shares developments in CMC for advanced therapies in the near future.

• Why Clinical Requirements Change: Dr. Peter Marks on Pre-IND Meeting Pathways

  Learn how clinical requirements change after the FDA and a company agree on a pathway during a pre-IND meeting and how the company might recover as a result.

• Race To The Regulatory Finish Line: Dr. Nicole Verdun Talks Efficiencies In Platform Technologies

  Could a platform approach help certain therapies make their way over the regulatory finish line more efficiently?

• Focal Points for 2024: Dr. Verdun and Dr. Marks Talk Regulatory Challenges for Cell-Based Therapies

  Dr. Verdun and Dr. Marks provide detailed feedback on the regulatory challenges facing cell therapy and explain the Agency’s most important focal points for 2024, patient accessibility, and more.

• How Past Gene Therapy Approvals Are Shaping the Future with Dr. Peter Marks

  In this segment of Cell & Gene Live, Dr. Peter Marks shared what he and his team have learned from current gene therapy approvals to accelerate future approvals.

• What's New for 2024: Dr. Nicole Verdun Offers a Look Inside The Office Of Therapeutic Products

  What is the Office of Therapeutic Products, where did it originate, and what's on its regulatory agenda for 2024?

• mRNA For Cancer Immunotherapy 2024 Outlook | Audience Q&A

  During our Cell & Gene Live, mRNA For Cancer Immunotherapy 2024 Outlook, our expert panelists answered audience questions regarding patient safety, foreseeable challenges in mRNA cancer immunotherapy for solid tumors, and much more.

• Supply Chain And Operational Challenges Facing mRNA

  Explore some of the biggest supply chain challenges facing mRNA cancer therapeutics and what short-term solutions may exist, starting with Chain of Custody/Chain of Identity.

• Bottlenecks And Manufacturing Challenges

  Expert panelists J. Andrew Case (Andy) and Daniel Getts cover the biggest bottlenecks facing mRNA cancer vaccine manufacturing today.

• Achieving Stability For mRNA

  Expert panelists for this Cell & Gene live event share some of things researchers are exploring to keep mRNA more stable.

• Major Advancements In mRNA

  Experts on this Cell & Gene live event discuss the major scientific biological advances in understanding the potential for mRNA-based immunotherapies vaccines for cancer over the last 2 years.

• Genentech, Myeloid’s Approaches To mRNA Technologies

  Experts at a Cell & Gene Live event discussed the use of mRNA technology in cancer immunotherapy, including a brief overview of the different approaches being used to leverage mRNA technology.

• Quality Roundtable: Optimizing Biologics Manufacturing Processes With Raw Materials

  Improve your commercial manufacturing process with advice from regulatory and raw materials experts on the development journey from pre-clinical to licensure.

• Upstream To Downstream: A Comprehensive Approach To AAV Production

  How can you face the challenges of AAV production, scale up your AAV manufacturing effectively, and enhance the recovery and purity in your downstream processing.

• Stability To Scalability: The Future Of Viral Vector Therapies | Audience Q&A

  Dave Maheu, VP, Head of Process & Analytical Development at Candel Therapeutics and Curran Simpson, Chief Operating Officer at REGENXBIO answer the audience questions in real time and cover the analytical testing systems used in formulation optimization and more.

• Scaling Viral Vectors

  Industry experts cover the high operational costs associated with viral vectors, along with the challenge presented by their shot shelf life and scalability.

• Overcoming Capacity Constraints

  How can CGT biotechs overcome issues such as building an in house facility and ultimately get their therapies to patients.

• Market Demand For AAV Vectors

  Here, Dave Maheu, VP, Head of Process & Analytical Development at Candel Therapeutics and Curran Simpson, Chief Operating Officer at REGENXBIO detail the key factors driving the adeno associated virus manufacturing market.

• An Active Release Mechanism For Magnetic Separation And Cell Isolation

  Discover an innovative active release solution that will serve autologous and allogeneic processes to the benefit of cell therapy patients.

• Regulatory Compliance And Viral Vectors

  Hear from experts who detail the specific regulatory challenges for advanced therapies, as well as how the regulatory requirements differ in early research and development in academia and in industrial vector production.

• Manufacturing Hurdles With Viral Vectors

  With a wide range of viral vector-based drugs already approved, viral vectors are expected to remain the primary delivery mechanism for the foreseeable future. However, as the demand for viral vectors increases, addressing the challenges related to their manufacture and scale-up is critical. In this segment, Dave Maheu, VP, Head of Process & Analytical Development at Candel Therapeutics and Curran Simpson, Chief Operating Officer at REGENXBIO share what they consider to be the most difficult challenges in vector development and manufacturing currently.

• Quality Control Assays

  Two company leaders detail not only the vectors their respective companies use and why but also the quality control assays performed on viral vectors.

• Optimize Cell Isolation And Activation For Flexibility And Scalability

  Learn about a first-of-its-kind platform technology that enables isolation and activation of T cells in one step using an active release mechanism for clinical trial or commercial manufacturing.

• Planning for Success: Supply Resiliency Strategies for Bench to Production

  Build scalable material sourcing strategies for R&D and early phases through to commercialization and learn how to accelerate the development process and ensure the scalability of your biologics.

• Driving Down COGS Audience Q&A

  Expert panelists provide detailed answers to questions submitted in real time by our registrants providing insightful responses regarding analytical challenges to automation, test automation, and more.

• Safety And Efficiency Of Source Material Collection

  Expert CTO's detail how automation improves safety and efficiency when collecting source material.

• Auto, Allo, And AI

  CTO experts debate autologous and allogeneic, and how automation improves the CAR-T cell process every step from collection to infusion. They also discuss the major ways AI optimizes automation.

• Pipeline Progress

  Experts working on robust pipelines that focus on various therapeutic areas explain how process automation looks different per therapeutic focus area.

• Mitigating Risk In Process Automation

  Two Chief Technical Officer's for therapeutic organizations cover the various ways in which automation helps to reduce risk and why closing systems and reducing the number of manual manipulations is so critical.

• Technology Needs For Automated Manufacturing

  Investigate the importance of technology as it relates to automation and standardization within cell therapy manufacturing. The panelists in this discussion cover solutions and services needed for process automation in CGT.

• Breaking Down Automated Manufacturing

  Experts explain the general benefits of automated manufacturing as well as the key areas that need improvement to scale up quality and to help reduce the costs of therapeutics.

• Mitigate Bioprocessing Limitations With A More Efficient And Sustainable Harvest Solution

  Subject matter experts in research and development, product management, and bioprocessing research introduce a single-use centrifuge, and its simplified, automated, intensified processing.

• Tackling Cell And Gene Therapies' Top 3 Regulatory Impediments: Audience Q&A

  During each Cell & Gene Live, our audience has the opportunity to submit real-time questions for our expert panelists. Watch and listen as Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio provide detailed, insightful responses to our audience questions on everything from regulatory timelines to successful and unsuccessful strategies needed to convince the regulators that current regulation requirements are either inappropriate or alternatively excessively burdensome for cell and gene-based therapies.

• Challenge #2: Rare Or Ultra-Rare Study Design Considerations And Use Of Existing Data

  There are more than 7,000 unique, rare diseases recognized in the United States, and 300 million people worldwide living with a rare disease; patients often experience a long journey to receive their diagnosis. Our expert panelists, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio, examine the regulatory requirements for safety and efficacy demonstration and identify the gaps in regulatory oversight that require clearer guidance.

• Challenge #1: The Changing Regulatory Landscape

  By 2030, 60 CGTs are expected to reach regulatory approval. One of the biopharmaceutical industry's most closely watched regulatory developments is the FDA’s reauthorization of the Prescription Drug User Fee Act (PDUFA) or PDUFA VII, as it provides resources for the FDA and CBER to have the capacity to provide feedback on development and review submitted applications, among other critical activities. During our Cell & Gene Live, Tackling Cell and Gene Therapies’ Top 3 Regulatory Impediments, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio break down how PDUFA VII, Fiscal Year 2023 Omnibus Appropriations Bill, and more will impact the cell and gene therapy field in the next few years.

• CGT’s Main Opportunities To Meet Regulatory Requirements

  Host and Moderator, Erin Harris, Chief Editor of Cell & Gene, began this Cell & Gene Live, Tackling Cell and Gene Therapies’ Top 3 Regulatory Impediments, featuring Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio, by asking the experts to highlight the main opportunities where the cell and gene therapy industry is working to meet regulatory requirements. They cover accelerated and expedited designation of regenerative medicines, navigating the regulatory requirements around the root causes of genetic disease by repairing or replacing a patient’s genes, and much more.

• Other CGT Regulatory Challenges

  During this segment of Cell & Gene Live, Tackling Cell and Gene Therapies’ Top 3 Regulatory Impediments, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio detail their own experiences with roadblocks with regulators and how they and their teams worked to overcome them.

• Challenge #3: Lack Of Animal Models

  Many cell and gene therapies are intended for diseases and conditions that primarily impact pediatric patients, requiring the prospect of direct benefit to individual subjects enrolled in the trial.  In the absence of good animal models, there may be challenges demonstrating the prospect of direct benefit.  During this segment of Cell & Gene Live, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO discusses novel approaches to consider going forward.

• Rapid Media And Feed Screening Options For Targeted CHO Clones

  Explore efficient and effective ways to identify a formulation for your clone – helping you streamline your process and achieve desired results.

• Accelerating mAb Development To Meet Increase In mAb Approvals

  To accelerate the delivery of life-saving therapeutics, it is essential to evaluate ways to maximize efficiencies, while minimizing risk, to stay up to speed with the growing demand.

• Enabling GMP Production with Enhanced Single-Use Fermentors

  As more products are moved into single use fermenters, there is greater need to improved oxygen delivery closer to or beyond the abilities of stainless-steel vessels.

• A Collaborative Path To Single-Use Supply Assurance

  Learn how leading biopharma companies such as Sanofi and single-use solutions provider Thermo Fisher Scientific are addressing supply challenges and developing collaborative processes.