VIDEOS

• Unlocking The Full Potential Of AAV-Mediated Gene Delivery | Audience Q&A

  In this final segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO addressed audience questions on re-administration, stable producer cell lines, and manufacturing challenges, and more.

• Addressing Regulatory Challenges Associated With AAVs

  In this segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO, the panelists cover the regulatory landscape, including working with regulatory agencies, addressing regulatory challenges for common diseases, long-term follow-up studies, beneficial regulatory pathways, establishing appropriate endpoints, vector shedding studies, anticipated regulatory guidance, and the cost burden of co-developing companion diagnostics.

• Key Considerations In Safety Monitoring And Manufacturing For AAV Vector Production

  In this segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO, the panelists address key safety considerations for AAV gene therapy clinical trials and novel safety monitoring approaches.

• Clinical Development Strategies And Prioritization For AAVs

  In this segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO, the panelists discuss the overall clinical development strategy for AAV based gene therapies.

• Emerging Technologies In AAV Vector Design And CNS Delivery

  In this segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO, the panelists discuss promising emerging technologies or approaches in AAV vector design that could significantly impact the field in the next five years, focusing on regulatable gene expression.

• Addressing Preexisting Immunity And Expanding To Common Diseases

  This segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO addresses strategies for dealing with the challenge of preexisting immunity to AAV vectors.

• Critical Factors For Early Phase Clinical Trials, Dose Selection, And AAV Capsid Engineering

  In this first segment of the Cell & Gene Live, Unlocking the Full Potential of AAV-Mediated Gene Delivery, David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics and Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO address the most critical factors to consider when designing early phase clinical trials for AAV gene therapies, including limitations of animal models, dose selection, target patient population, and safety.

• Introducing EndoCleave, A New GMP-Grade Endonuclease For VV Manufacturing

  Explore how a reliable endonuclease, produced under GMP Grade standards, ensures consistent, efficient nucleic acid removal, and supports your regulatory compliance needs.

• Audience Q&A | From Bottleneck To Breakthrough: Reimagining The mRNA Therapeutics Supply Chain

  During every Cell & Gene Live, our expert panelists provide actionable responses to real-time questions from the audience. Here, panelists share information on categorizing raw materials, linear DNA, the future of the mRNA supply chain, and more.

• Tips For Scaling mRNA Therapeutics

  In this segment, Joseph Barberio of Strand Therapeutics and Christian Moreno of Nutcracker Therapeutics share their insight on what’s needed to achieve success in scaling mRNA therapeutics.

• Is Cold Chain Really A Challenge Facing mRNA Therapeutics?

  mRNA product storage can be challenging due to extremely low temperature requirements. Here, Joseph Barberio of Strand Therapeutics and Christian Moreno of Nutcracker Therapeutics share their take on existing cold chain challenges.

• Tackling The Standardization Issue For mRNA Therapeutics

  This segment features a discussion around the obstacles to establishing a standardized production pipeline for mRNA therapeutics across the industry as well as some the approaches being considered to standardize cold chain management protocols for mRNA products.

• Regulatory Considerations For The mRNA Platform

  Joseph Barberio of Strand Therapeutics and Christian Moreno of Nutcracker Therapeutics share how their companies ensure data integrity and privacy compliance throughout R&D and manufacturing.

• Top Bottlenecks Facing mRNA Supply Chain

  In this first segment from Cell & Gene Live, we explore the state of the mRNA therapeutics supply chain and the major bottlenecks that exist today, including non-GMP grade reagents to sourcing DNA.

• Solutions For Cell Analysis Metabolite Testing

  Learn about a family of instruments that performs precise measurements and enables informed decisions and fast time response.

• Develop Rapid, Accurate Point Of Care Assays Quickly and Efficiently

  Explore Lyophilization-ready (lyo-ready) reagents, a unique portfolio of high-performance enzymes that streamline the development and production of diagnostic assays intended for PoC.