FOCUSED ULTRASOUND
08:00 A Non-Invasive Delivery Platform for Gene and Cell Therapy to the Brain and Beyond
- Overview of focused ultrasound (FUS) as a modality to non-invasively and transiently open the blood-brain barrier (BBB) for therapeutic delivery —including clinical data
- Recent advances in preclinical studies using FUS to deliver gene and cell therapies to the brain in small and large animal models
- Toward first-in-human translation: ongoing efforts to advance FUS-mediated delivery of gene therapy to the clinic.
- Emerging preclinical efforts expanding FUS-mediated gene therapy delivery to large organs (e.g., liver, heart) Challenges and opportunities for clinical translation: a framework in development to guide indication selection and align with regulatory, manufacturing, and access considerations.
Frederic Padilla Director Gene and Cell Therapy Program Focused Ultrasound Foundation
08:30 Unlocking CNS Access Through Transferrin Receptor-Targeted Capsids
- Optimizing for translation in the clinic should start with capsids interacting with well-characterized human receptors.
- Regulatory guidance in gene therapy and direct FDA feedback across multiple programs outlines preclinical path for human-specific transferrin receptor-binding capsid and use of humanized animal models.
- Surrogate mouse BBB-crossing capsids can be used in efficacy studies in parallel with distribution studies enabling dose translation between surrogate and clinical capsids targeting human receptors.
Christopher Davis, PhD Director, Nonclinical and Translational Research Apertura Gene Therapy
09:00 Reducing Host Responses While Enhancing Delivery In Viral Gene Therapy Platforms
Viral gene therapy has emerged as a promising avenue for the treatment of a variety of genetic disorders. However, the success of these therapies often hinges on two critical challenges: the immune responses of the host and the efficiency of viral delivery systems. This talk will delve into innovative strategies designed to reduce host immune responses to viral vectors while simultaneously enhancing their ability to effectively deliver genetic material to targeted cells. Key areas of focus include:
- Exploration of genetic modifications and novel viral platform designs aimed at minimizing immune recognition and improving vector stability and specificity.
- Discussion on advancements in vector delivery techniques, such as tissue-specific targeting and controlled release, to increase the therapeutic efficacy of gene therapies.
- Examination of current clinical trials and preclinical research that showcase these advancements, along with a forward-looking perspective on overcoming remaining obstacles.
Niren Murthy Professor UC Berkeley
09:30 From Donor to Dose: Building a Robust Foundation for Allogeneic Manufacturing
- The Foundational Role of Donor Selection
- Key Donor Attributes and Selection Criteria
- Operational and Logistical Considerations
- Managing Donor Variability
- Strategic Alignment with Development Stage
- Collaborative and Ethical Dimensions
Raluca Dumitru, MD PhD VP Scientific Affairs, CGT Discovery Life Sciences
10:00 Integrating cutting-edge technologies in cell and gene therapy manufacturing to optimize speed, safety, and environmental sustainability in biotech.
- Current challenges in gene therapy viral vector manufacturing.
- Recent technical innovations that significantly improve AAV titer, purity and vector potency with dramatically reduced manufacturing cost and turnaround time.
- AAVPCR, a next-generation AAV manufacturing platform that features in cellulo plasmid DNA replication for superior rAAV production.
- Potential future directions towards economical and robust off-the-shelf AAV manufacturing.
Hao Liu Viral Vector Core Research Associate III Umass Gene Therapy Center
10:30 Refreshment Break
11: 00 From capsid design to scalable production: enabling BBB-crossing AAV with Apertura’s TfR1 CapX
- Apertura Gene Therapy has engineered systemically delivered AAV capsids designed to traverse the blood–brain barrier.
- We embed manufacturability and scale considerations at the start of capsid design, enabling smoother translation into AAV production workflows.
- TfR1 CapX has been produced at clinically relevant scale to demonstrate readiness for downstream clinical manufacturing requirements.
Von Wiltman Associate Director, Technical Operations Apertura Gene Therapy
11:30 Overcoming Clinical Trial Design Challenges in Rare Disease Populations: Innovative Approaches to Endpoint Definition, Statistical Significance, and Regulatory Standards in Small Patient Cohorts
12:00 Lunch Break
13:00 Standardizing Gene Therapies to Ensure Consistent Clinical Outcomes via Robust Analytical Strategies
In this presentation, we address the critical role of gene therapy development, focusing on analytical strategies to ensure consistency, reliability.
Yan Zhi Director, Cell & Gene Therapy Product Owner CSL Behring
13:15 Overcoming Process and Scale-Up Challenges in Viral Vector Manufacturing to Enable Commercial-Ready Cell and Gene Therapies
In this session, we examine key strategies for scalable and robust viral vector manufacturing, including upstream and downstream optimization, platform technologies, and quality control. Attendees will gain insights into process intensification, automation, and regulatory expectations, with real-world case studies illustrating successful tech transfers.
13:30 Addressing Critical Product and Process Challenges in Cell & Gene Therapy Manufacturing
This presentation delves into innovative strategies and technologies that streamline manufacturing workflows, reduce variability, and improve batch consistency, ultimately enabling more reliable and scalable production of life-changing therapies.
Chaozhong Zou Executive Director(Head), Applied Gene and Cell Therapy Center Cincinnati Children's
13:45 Explore how engineering allogeneic cell therapies enables scalable, off-the-shelf manufacturing strategies that enhance access, lower costs, and accelerate commercial readiness for global impact.
14:00 Interactive Discussion: Redefining Manufacturing Excellence in Scalable, Automated, and Consistent Cell & Gene Therapy Production
Panelist:
- ·Yan Zhi Director, Cell & Gene Therapy Product Owner CSL Behring
- ·Chaozhong Zou Executive Director(Head), Applied Gene and Cell Therapy Center Cincinnati Children's
- ·Hao Liu Viral Vector Core Research Associate III Umass Gene Therapy Center
- ·Vladimir P. Bermudez, Ph.D., PMP Associate Director, Columbia Initiative in Cell Engineering and Therapy Columbia University Irving Medical Center
14:30 Refreshment Break
15:00 Reimbursement Challenges and Strategies of Cell and Gene Therapies in Fee-for-Service Medicaid
- Medicaid programs have encountered challenges with cell and gene therapies, particularly in terms of coverage policies and reimbursement.
- These challenges have necessitated creative approaches by Medicaid Pharmacy Directors to ensure patients have access to these novel therapies and hospitals receive appropriate reimbursement.
- Cell and gene therapies are evolving at a rapid pace compared to reimbursement systems, prompting innovative solutions.
- States have been creative to find equitable reimbursement policies to not only ensure sustainability but also capture available rebates Carveouts from Managed Medicaid Plans and traditional reimbursement methodologies have grown as more products come to market
- How can value based agreements help states address the financial hardships posed by the numerous cell and gene therapies
Chad M. Bissell, PharmD, MBA Vice President, Drug Launch Services Artia Solutions
15:30 Strategic Stakeholder Engagement to Navigate Payer Skepticism in Cell and Gene Therapy Reimbursement
This session will explore how early, strategic engagement with key stakeholders, including payers, providers, regulators, and patient advocacy groups can help mitigate uncertainty and support more sustainable reimbursement pathways. Attendees will gain insights into best practices for evidence generation, real-world data integration, and value communication tailored to payer concerns. The session will also highlight successful models and collaborative approaches that have improved market access and reimbursement success for CGT innovators.
Jodie Wehling Vice President, Market Access Head of Market Access, Trade & Distribution, Contracting Mesoblast Limited
15:45 Unlocking Payer Value: Aligning Cell and Gene Therapy Pricing Strategies with Long-Term Outcomes and Real-World Evidence for Market Access Success
This session explores innovative strategies to align CGT pricing with long-term clinical outcomes and real-world evidence (RWE), addressing payer concerns around value, sustainability, and budget impact. Attendees will gain insights into outcome-based agreements, data-driven value assessments, and collaborative approaches to market access that can unlock payer confidence and ensure timely patient access. Learn how to position therapies for reimbursement success in a value-based healthcare environment.
16:00 Interactive Discussion: Redefining Market Access, Evidence, and Reimbursement Strategies in Cell & Gene Therapy
Panelist:
- Chad M. Bissell, PharmD, MBA Vice President, Drug Launch Services Artia Solutions
- Jodie Wehling Vice President, Market Access Head of Market Access, Trade & Distribution, Contracting Mesoblast Limited
17:00 End of conference
Cell & Gene Therapy Summit 2026
19 March 2026.(Pacific Time ) | Virtual Congress